Clinical Trial Seeks First New Alzheimer’s Treatment in a Decade

April 8th, 2015

Alzheimer’s disease is a debilitating, progressive neurodegenerative disease affecting close to five million Americans. The Alzheimer’s Association estimates the disease is responsible for at least 60%, and perhaps as much as 80%, of all dementia cases, and afflicts more than 10% of Americans over age 65, and one-third of those over age 85. Worldwide, some estimates put the number of people with Alzheimer’s as high as 40 million.

While not observable in the living, autopsies show microscopic growth of abnormal clumps of beta-amyloid protein plaque and neurofibrillary tangles within and between brain cells of Alzheimer’s patients. While these are associated with the disease, it is still not known whether they cause it, or result from some other cause.

Since family history is the second greatest risk factor, trailing only age, Alzheimer’s also appears to have a genetic factor. Alzheimer’s is usually first identified in patients age 65and up – most commonly by difficulties in assimilating or remembering new information, impaired reasoning, personality changes or other mental or physical irregularities. In less than 5% of cases, onset comes much earlier, and a specific genetic link has been identified for this form of the disease.

Some current treatments can bring temporary easing of symptoms, but thus far no treatment has been discovered that reverses or stops the progression of Alzheimer’s. The disease’s prevalence, virulence and costs – the annual costs of treating an American with Alzheimer’s is somewhere around $50,000 — has drawn $565 million in government research this year seeking its causes and better treatments, and numerous private companies are seeking to develop effective remedies.

In hopes of discovering the first new treatment in a decade, a Phase II clinical study, bearing the label NOBLE, is now underway at about 50 U.S. hospitals hopes to enroll 450 people between the ages of 55 and 85 with mild to moderate Alzheimer’s. It aims to assess whether a new experimental drug, T-817MA, helps prevent or slow the loss of brain nerve cells.

The NOBLE trial is funded entirely by the drug’s developer, Toyama Chemical Ltd., part of Japan’s Fujifilm Group. Running the clinical trial is the Alzheimer’s Disease Cooperative Study, a National Institutes of Health-supported clinical trials consortium based at the University of California San Diego. The lead researcher is Dr. Paul Aiser, a leading Alzheimer’s authority.

All participants in the trial are already taking the leading current treatment drug (donepezil, also known by the trade name Aricept), and some are also taking memantine (trade name Namenda), another treatment of a different type. The trial will randomly divide participants into three groups. One group will receive a placebo; the other two groups will receive the experimental drug, in one of two dosage levels. All participants will be tested for memory, other mental functions and overall ability to function, and will receive regular physical exams, blood tests and brain scans.

Toyama has been working on the drug since 2005. In 2008-2011, a more limited trial in the U.S. and Canada using only one dosage level suggested T-817MA has the potential to slow down cognitive and functional decline in Alzheimer’s patients.

Begun in March 2014, the NOBLE trial is scheduled to last until March 2016. New participants are still being sought.

Clinical Trials Bring New Immunotherapy Treatment for Lung Cancer, Melanoma

March 16th, 2015

Successful clinical trials have led to Food and Drug Administration approval on March 4 of an immunotherapy drug seen as a breakthrough for hard-to-treat cases of the most common form of lung cancer.

The new remedy, nivolumab (tradenamed Opdivo by developer Bristol-Myers Squibb), is the first approved treatment using the body’s immune system, rather than chemotherapy, to fight squamous cell non-small cell cancer (NSCLC), found in seven out of every eight lung cancer patients. Lung cancer causes the most cancer deaths in the U.S. (nearly 160,000 last year), and about 225,000 new lung cancer diagnoses are made each year.

A randomized clinical trial included 272 people with advanced NSCLC who had previously been treated with platinum-based chemotherapy drugs. After starting treatment, the 135 patients who received Opdivo had an average survival time 3.2 months longer than the 137 patients who received docetaxel, a leading taxane-type chemotherapy drug also marketed under the names Taxotere and Docecad.

Tumors shrank or disappeared in about 15% of patients given the new drug, and among them, 59% saw the improvement continue for more than six months. In contrast, chemotherapy typically brings such results in 10% or less of NSCLC patients, and the trial participants had already taken chemotherapy treatments without success.

The trial, planned to continue through June this year, was stopped more than three months ahead of schedule, since its results showed a clear survival advantage for patients given the new immunotherapy treatment.

The new drug works by attacking the PD-1 protein, which some cancer cells make as a shield against the immune system. By lowering that shield, the drug enables the patient’s immune system to fight the spread of cancer cells.

Dr. Rachel Sanborn, an oncologist with the Providence Cancer Center in Portland, Oregon, which participated in the SNCLC trial, hailed the FDA’s approval of the first-ever immunotherapy treatment for lung cancer an exciting development, calling it “the beginning point, not the end point.” While all participants in the clinical trial had previously received chemotherapy, future trials are planned to examine whether the drug could be a first-line treatment.

Opdivo had qualified for priority review by the FDA, a program that speeds review of drugs designed to treat serious conditions with the potential of bringing significant improvement in either or both safety or effectiveness.

The drug had won FDA approval last December for treating melanoma, responsible for almost 10,000 deaths in this country annually, making it the nation’s fifth most prevalent form of cancer. That trial involved patients who could not be treated by surgery or whose cancer had metastasized and did not respond to chemotherapy.

Like the case with the lung cancer trial, the melanoma trial finished three months early, and qualified for priority review at the FDA. The agency also designated it as a potential breakthrough therapy and accorded it special orphan-drug procedures.

That clinical trial found almost a third of 120 participants with inoperable or metastatic melanoma experienced tumor shrinkage; the shrinkage persisted for over six months in about a third of those patients.

Encouraged by the drug’s performance with two forms of cancer, researchers plan to test whether it can show similar results against other forms of the disease.

FDA Eyes Easier Access to Experimental Drugs for Seriously Ill Patients

March 3rd, 2015

The U.S. Food and Drug Administration (FDA) has proposed simplifying the procedures by which seriously ill patients can be allowed “compassionate use” of experimental drugs. The agency on February 4 issued a draft guidance document entitled Individual Patient Expanded Access Applications: Form FDA 3926, and will take public comments for 60 days.

Saying it wants to create a “streamlined alternative” to its current process of applying for what the agency calls “expanded access” to investigational drugs (which are being reviewed but have not yet been approved by the FDA). The agency’s Expanded Access Program aims to provide patients with serious or immediately life-threatening conditions for which there are no other comparable or satisfactory treatments an alternative avenue for getting access to drugs that may help.

The FDA says it already approves nearly all the expanded access applications it receives – last year, for instance, it rejected only nine of the 1,873 applications it received, and agency records show the FDA turned down just 33 (or about one-half of one percent) of the 5,995 expanded access applications it got over the last five years.

But the agency has been criticized for how complex its expanded access applications process is. The current system requires all expanded access submissions to file a summary form used for investigational new drug applications (IND 1571), which pharmaceutical firms submit when seeking FDA approval to run clinical trials for a drug under development. Since the IND 1571 is designed for drug developers and summarizes extensive information – one senior agency official says it calls for 26 distinct categories of information and seven attachments — the FDA estimates it takes 100 hours to complete.

But the overwhelming share of expanded access applications come not from drug makers trying to expand clinical trials, but from physicians filing on behalf of a single patient. In fact, 97% of expanded access applications the agency received in 2014 covered either an emergency or regular single-patient use. The FDA says it’s concerned the daunting form and its administrative burdens may be too much in such cases. In fact, unwieldy FDA expanded access procedures may have helped fuel the “right to try” movement, which has already led several states to enact laws allowing patients to take non-FDA approved drugs.

The agency’s proposed solution is to create the new Form 3926, for use by doctors seeking to gain access to an experimental drug for a single patient. The FDA says this simplified form could be completed in less than an hour, not the 100 hours for an IND 1571.

The new form would ask for eight fairly straightforward data categories. These include: the patient (for privacy reasons, only the patient’s initials are used), plus: the patient’s clinical data; treatment data on the drug and planned usage; a letter from the drug maker authorizing product use; information on the supervising physician and his or her qualifications; and an agreement that, absent an emergency triggering different procedures, the expended access treatment won’t start for 30 days – the period FDA has to review INDs).

As at present, under the new guidance a physician seeking emergency approval for expanded access could call FDA for authorization to start treatments; in such cases, a written form must be filed within 15 days of getting authorization.
Besides the new guidance, the FDA has recently created a new webpage to summarize for patients their expanded access options.

Expanded Access to Clinical Trial Data Gains Ground

February 3rd, 2015

One of the greatest frustrations for medical researchers is knowing they have access to only a fraction of data from completed clinical trials. With limited exceptions, such as some government-funded research, clinical trial sponsors determine whether data from those trials ever see the light of day. Limited data-sharing has drawn criticism from many academics and researchers, and helped spur the open-data movement, which calls for wider availability of clinical trial data.

Several recent developments, however, suggest researchers are making significant headway in gaining greater access to data from a much wider range of clinical tests.

In a new report released January 14, the National Academy of Arts and Sciences-affiliated Institute of Medicine (IOM) calls for requiring clinical researchers to set a timetable for sharing trial data as part of registering a clinical trial. Calling for a research culture where data sharing is the “expected norm,” the non-profit, non-governmental advisory group’s preliminary report, Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk, recommends specific timelines for making clinical trial data available.

Clinical trial researchers should have a maximum of 18 months after a trial has been completed to release data, the IOM report says. The trial sponsors and researchers should disclose full analyzable data sets, along with metadata (describing this as “data about the data,” including such items as trial protocols, statistical analysis plans, and analytic coding). Even earlier, the preliminary IOM report calls for summaries of trial results (including adverse events) within a year after a trial’s end, and access to analytic data sets supporting published results within six months of publication. The IOM report recommends making exceptions, however, for studies done in support of regulatory applications, however.

New England Journal of Medicine editor-in-chief Dr. Jeffrey Danzen, one of the 12 members on IOM’s Committee on Strategies for Responsible Sharing of Clinical Trial Data, the authors of the new report on ways to make clinical trial data more widely available for other researchers, recently asserted in his publication clinical researchers “need to change how we think about data.” He advocates it be seen as a community resource, rather than as personal property.

While greater sharing of clinical test data would benefit research and public health, at the same time the IOM report notes the need to minimize foreseeable risks by protecting a variety of interests. These include: trial participants’ privacy; sponsors’ stakes in intellectual property rights and commercial confidentiality; proper recognition for researchers for their discoveries; discouraging invalid secondary analyses of released data (which could invite litigation and make clinical trials less trusted by the public;, and reassuring research institutions data-sharing rules do not amount to new unfunded mandates.

The report contains an extensive slate of recommendations, including trial sponsors insisting on data release plans for all studies they fund, and a multi-stakeholder group creating data-sharing principles and an infrastructure to enforce wider access to trial data.

The IOM report was sponsored by a group of 23 industry, federal agencies (the FDA and NIH), charitable and medical organizations. One of those sponsors, Johnson & Johnson, recently gave another boost to the open-data movement, by expanding its data-sharing under the Yale University Open Data Access Project. The firm, which earlier said it would share its pharmaceutical data with the Yale project (which as an intermediary responds to requests for the data), became the first company to agree to provide data on trials of its medical devices and diagnostics.

If You Suffer From Chronic Lung Infection This Clinical Research Study May Be For You

January 17th, 2015

clinical trials for chronic lung infectionsPulmonologists from major medical centers across the world are recruiting patients with non-cystic fibrosis bronchiectasis (BE) to participate in two clinical trials with Pulmaquin.

Patients with BE who have chronic respiratory infections with the microorganism Pseudomonas aeruginosa have a severe form of the disease that can lead to poor quality of life and untimely death.  BE is a condition characterized by abnormal dilatation of the airways, often associated with chronic infection.  The patient’s lung function is often irreversibly reduced compared to that found in healthy individuals.  BE is frequently observed in patients with cystic fibrosis  (CF). However, it is a condition that affects over 100,000 people without CF in the United States.  Many of these patients are non-smokers and the origin of their BE is unknown.

Two international clinical research studies are underway to evaluate the safety and effectivness of Pulmaquin®, an investigational drug that is inhaled once daily.  The trials compare Pulmaquin to placebo in the management of chronic lung infections with P. aeruginosa in patients with non-cystic fibrosis BE.  Investigators will evaluate this by determining how long it takes for participants to experience their first pulmonary exacerbation.  A pulmonary exacerbation is the new appearance or worsening of respiratory signs and symptoms such as cough, wheezing, chest congestion or shortness of breath, fever, or fatigue. Other important clinical endpoints, including the patients’ quality of life will also be evaluated.

If you or someone you know is interested in learning more please visit The Clinical Trial For Chronic Lung Infections.

Study Seeks Model Care for Chronic Kidney Disease Combined with Other Conditions

January 7th, 2015

clinical trialsA recently announced NIH-supported national multicenter project plans a five-year study of individuals diagnosed with three common chronic diseases: chronic kidney disease, hypertension and diabetes.

Each of those conditions affects millions of Americans. About a third of the nation’s adults experience hypertension, while about one in seven adults in the U.S. has chronic kidney disease, and one in ten is diabetic. The three conditions are frequently found in combination; about half of adults with chronic kidney disease also suffer from diabetes, and many also have high blood pressure.

The national study’s principal investigator, University of Texas Southwestern Medical Center nephrologist Dr. Miguel Vasquez, says these common correlations make studying each chronic condition by itself “not the real world for clinicians.” Instead, the new research project, the first of its size and type, aims to study evidence for which treatments are most effective for patients with this frequent combination of conditions.

First, the study plans first to identify patients with these multiple conditions in a wide variety of treatment settings. In addition to Dr. Vasquez’s institution, other participating research centers include Dallas-based Parkland Health and Hospital System, the Arlington-based non-profit Texas Health Resources, the Veterans Administration’s North Texas Health Care System, and Connecticut integrated primary-care group ProHealth Physicians, Inc.

Representing veterans’ hospitals, private hospital systems, safety-net hospitals and accountable care organizations, these providers will provide data from diverse healthcare settings and patient populations. For example, of the VA healthcare system’s 8.4 million enrollees nationwide, over two-thirds have hypertension, 34% have diabetes and 20% chronic kidney disease. Similarly, the roughly 350,000 patients cared for by ProHealth Physicians includes about 90,000 with high blood pressure, 26,000 with diabetes and 25,000 with chronic kidney disease.

After identifying a broad group of patients with all three conditions, the project will then examine data on which treatments most reduce such negative outcomes as hospitalization, readmission, cardiovascular events, and death for such patients. Although effective treatments are available separately for each of the three conditions, the study will look for which clinical practices most reduce unfavorable outcomes among patients with all three conditions.

Ultimately, the project hopes to develop an integrated care model for such patients, to be shared widely with primary-care providers, showing which early interventions may slow the progression of chronic kidney disease to end-stage renal disease, and thus not only improve patient health but also hold down healthcare costs.

Two parts of the National Institutes of Health, the National Institute of Diabetes and Digestive and Kidney Diseases and the National Heart, Lung, and Blood Institute, will oversee the study, known as “Improving Chronic Disease Management with PIECES” or “ICD Pieces.”

PIECES is an information technology platform developed by co-investigator Dr. Ruben Amarasingham, the president and CEO of the Parkland Center for Clinical Innovation. The platform will pull data from electronic health records to identify high-risk patients, then evaluate early detection strategies, and identify real-time methods for bringing the most useful care and resources to high-risk patients.

The large-scale project is one of three recent NIH-funded research projects studying multiple chronic conditions, all supported by the NIH Health Care Research Collaboratory, with research awards totaling over $19 million over a five-year period.

New NIH Website Aims to Make Global Clinical Trials Easier

December 18th, 2014

global clinical trialsA new National Institutes of Health website aims to make it easier for clinical trials around the world to comply with relevant regulations. Recently launched by NIH’s National Institute for Allergy and Infectious Diseases (NIAID), the new ClinRegs website is intended to assist clinical researchers by providing them a database showing the specifics of each country’s rules on clinical research.

Officials at the NAIAD say the website can serve as a central resource for international regulatory issues, which should save time and streamline compliance efforts for clinical trial planners. In fact, project officials said the website was developed after finding that regulatory compliance was a frequent, common hurdle for researchers and sponsors trying to plan clinical trials.

At the time the beta version of ClinRegs was launched in early September, it included clinical trial rules in 12 of the most active nations in clinical research (besides the US, the UK, China and India, these also include Brazil, Kenya, Malawi, Peru, South Africa, Tanzania, Thailand and Uganda).

NIH officials say more nations will be added in the future. Before the end of this year, regulatory information is expected to be added for Sierra Leone and Liberia, likely sites for clinical research on Ebola fever vaccine and remedies. The number of countries covered by the new website is anticipated to more than double over the next three to five years. Haiti, Mexico and Vietnam are among likely additions in the near future.

The new website displays information on seven key clinical trial regulatory topics – the national regulatory agencies (including links to the national authority), oversight of ethical committees, clinical trial approval and management, research sponsorship, informed consent of trial participants, and rules on biospecimens and investigational products.

In what the agency touts as a particularly useful feature, ClinRegs also allows users to bring up side-by-side comparisons of any two nations in the database.

NIH says the website’s summary of each nation’s clinical research regulations will be reviewed at least annually, and more frequently if it finds major regulatory revisions or additions. The date of last revision will be shown on the website. The ClinRegs team says it hopes to attract input from research and regulatory professionals worldwide, to keep its information current and accurate.

While ClinRegs is primarily intended to aid individuals and organizations involved in planning, conducting and overseeing clinical research, it is also open to members of the public, and the input of all users is being sought.

Visitors to the website are greeted by a short pop-up survey, in which NIH asks how often they visit the site, how they found it, their purpose in visiting, and specific research topic. The survey also asks visitors to rate the site’s design, helpfulness, and ease of use, and to identify additional countries they would like to see added to ClinRegs.

NIH also invites further comments and suggestions for improvements to be sent to: NIH, Project Clearance Branch, 6705 Rockledge Drive, MSC 7974, Bethesda, MD 20892-7974, ATTN: PRA# 0925-0668.

“Big Data” Analysis May Aid Selection in Clinical Trials and Aid Treatment Decisions

December 4th, 2014

clinical trials for patientsThe growing ability of scientists to analyze ever-increasing mountains of health data has drawn increased attention to clinical research uses of “big data.”

While public and private efforts to sequence human genetics took over a decade and cost $3 billion, today analyzing an individual’s genetic makeup takes one week and costs $1,000. As data volume and analytical tool sophistication grow, clinical researchers – supported by research centers and agencies — are increasingly drawing on larger, more diverse data sources.

The National Institutes of Health, for example, in 2012 launched an agency-wide Big Data to Knowledge initiative, and last year committed $100 million to BD2K research; NIH wants to see big data more standardized and widely used and shared. Proliferating electronic medical record systems offer broader data on larger groups.

Big data not only lets trial enrollers cast a wider recruitment net, but also find genetic or other factors affecting susceptibility to the disease or therapies, thus identifying enrollees more likely to benefit and less likely to suffer side-effects which might drive them out of trials. Access to wider databases can also help spot new correlations, such as previously unsuspected drug interactions.

A recent article in the New York Times Sunday magazine, with the provocative title, “Can Big Data Tell Us What Clinical Trials Don’t?,” showed how medical practitioners can tap big data to aid treatment decisions. It recounted the case of a 13-year-old girl with symptoms of kidney failure who’d been airlifted to Packard Children’s Hospital at Stanford University, and quickly diagnosed with lupus, along with several complications.

The on-call rheumatologist, recalling similar cases, wanted to give the girl anti-coagulant medication, fearing a potentially fatal blood clot, but other doctors disagreed. Finding no relevant medical literature, the medical team resolved the deadlock by running a statistical analysis of pediatric patients with lupus who had been admitted to the hospital during the past five years.

Their statistical search found blood clots had developed in 10 of 98 patients with similar symptoms who did not get anti-coagulants. Satisfied, the medical team administered the anti-clotting medication to the girl, who did not develop thrombosis or side-effects from the medication.

Obstacles remain to such real-time use of data-mining medical databases for research purposes, notably privacy protections under the Health Insurance Portability and Accountability Act (or HIPPA). A quick illustration: After the Stanford rheumatologist wrote up the data-mining analysis for the New England Journal of Medicine, hospital officials cautioned against further efforts of the type until they devised a patient privacy framework.

Many see additional data sources capable of modifying clinical trials or practice. At the 2013 Forbes Healthcare Summit, Glen de Vries, president of clinical trial database provider Medidata, advocated use of such devices like wearable monitors, sensors and even smartphone apps to track trial participants’ activities and reactions. Rather than measuring how far a cardiac drug tester can walk by bringing him to a doctor’s office for a treadmill test, de Vries suggested furnishing enrollees with devices like FitBits or Nike Fuel bands.

To Battle Ebola, Drug Makers Press for Cure, Vaccine

November 18th, 2014

clinical trials

With the Ebola virus ravaging West Africa and causing growing anxiety over the potential for outbreaks in Europe, America and elsewhere, drug developers are pressing to discover an effective treatment and develop a vaccine against the lethal hemorrhagic fever.

No drug is yet approved as safe and effective for treating or preventing Ebola. Current treatment for those stricken by the disease is to balance fluids and electrolytes, maintain blood pressure and oxygen, and treat complications. In Guinea, Liberia and Sierra Leone, where the disease has attained epidemic proportions, the death rate is about 70% of those infected.

As an emergency measure, ZMapp, developed by San Diego-based Mapp Biopharmaceutical, is being administered in some cases. For example, ZMapp was also given to two U.S. medical missionaries who recovered after contracting the disease in Liberia, where the disease has already claimed thousands of lives.

The experimental therapy is a cocktail drug combining three monoclonal antibodies. The company’s current molecular biology process uses tobacco plants to produce each antibody separately, and then must get a certificate of analysis for each before combining them into the combination drug ZMapp.

The company is working to boost yields from its plant-based production method, and ecently joined with another producer to see if yields be would boosted by using an animal-based biotech production method, which employs genetically-modified CHO cells from Chinese hamster ovaries cultivated in sterile tanks. The plant production method, carried out by the Reynolds tobacco company’s Kentucky BioProcessing subsidiary, is faster and cheaper, but not as suitable for large-scale production as more conventional biotechnology methods.

ZMapp is being tested in animal experiments done by the US Army Medical Research Institute of Infectious Disease, with assistance from the Bill and Melinda Gates Foundation. The animal tests are designed to see if the drug has the same effects at lower doses, since supplies of ZMapp are very limited.

On September 2, Mapp Biopharmaceutical received a $24.9 million contract from the U.S. Department of Health and Human Services to assist the company’s manufacturing and non-clinical activities. Part of the 18-month contract will also bring HHS technical support for the drug company’s effort to manufacture enough of the drug to support human safety clinical studies. The company had planned to start clinical studies in 2015, and is now working to accelerate its efforts to win FDA approval.

In addition, two Ebola-preventing vaccines have started clinical trials in humans. Even if early results from those tests are positive, no vaccine is expected to receive FDA approval and reach the marker until next year at the earliest.

At the National Institutes of Health (NIH), Phase 1 safety and effectiveness clinical trials in healthy adults have begun for an investigational vaccine jointly developed by the National Institute of Allergy and Infectious Diseases (NIAID) and GlaxoSmithKline, and for an experimental Ebola vaccine from the Public Health Agency of Canada. The NIAID-GSK vaccine will also receive clinical trials in the UK and several African nations. Further anti-Ebola products are likely to follow in starting clinical trials, perhaps as early as next year.

Questions to Ask about a Clinical Trial You’re Considering

November 1st, 2014

While trying to decide whether to sign onto a clinical trial, you’re likely to have lots of uncertainties and gaps in your knowledge. You may even be unsure on how to go about reaching this important decision.

The enrollment process — especially the requirement that you get detailed information from the researchers and have a chance to get your questions answered – should eventually equip you to your choice. (Note: the U.S. Food and Drug Administration, which wrote the rules on the informed consent requirement and oversees compliance with the mandate, earlier this year proposed revised guidance on the process and is taking public comment until October 27).

You should inform yourself as fully as possible about the clinical trial before deciding on whether to take part. But to give you a head start, here’s a fairly comprehensive summary of the issues you may want to consider.

Questions on Study Design and Administration

What’s the purpose of the study?

It could be to explore whether a therapy would be safe and effective, to probe whether it has significant side effects, to establish dosage levels, to gauge the comparative effects of two or more competing treatments.

How many people will participate in the study? How will they be selected? How long is the study supposed to last, and what will participants be asked to do?

Clinical test sizes vary, usually with small groups studied in the early stages, moving to larger groups as it moves nearer to approval (and, sometimes, clinical trials study treatments already on the market). While some trials seek healthy volunteers, participants are often selected from patients with a condition being studied, or a predisposition toward it.

Why does the research believe the treatment being studied might be an improvement? Has the treatment been tested previously? If so, what were the results? What process is being used to monitor participant safety and study results? Has the proposed study been reviewed and approved?

Since there can be numerous levels in the process – an Institutional Review Board and possibly an independent science review board, and perhaps additional levels, such as an independent safety review panel or Data and Safety Monitoring Board, you might also ask about any approvals that are still awaited.

Questions on Treatments and Possible Risks and Benefits

What are the study’s possible short-term and long-term benefits for participants? The possible short-term and long-term risks? How do these compare with those for any other available treatments?

During a trial, participants will receive medical treatment and screening; randomized tests give some participants a treatment being tested, but give a non-active placebo to other participants. Some treatments may bring improved health in some participants, but others may experience adverse reactions or side effects.

What tests, treatments and procedures will the trial include? Can I take my regular medicines during the trial? Where will I be treated, on what schedule, and who will be in charge?

Other Questions

How is the trial funded, and will I have to pay any costs? Will any of the treatments or tests be painful or affect my everyday life? Will I have other expenses (e.g. travel or childcare) from being in the trial?

If I have problems or need more information, who should I contact?