Clinical trials and hemophilia

Clinical Trials 101

Learn how a clinical trial works and the benefits to participating
Author: Heather Boerner

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Disclaimer: This article is intended to be educational only and not a promotion of any particular clinical trial or product.

Travis Tussing is doing something very small that could have a big impact. He is participating in a clinical trial. The 30-year-old law student in Lansing, Michigan, has severe hemophilia A. Since October 2009, he has been infusing three times a week and has visited his local hemophilia treatment center (HTC) for blood draws more often than usual. It takes only about an hour out of his week. But at the end of the year, he’ll have contributed to research into a new, longer-acting product that could replace what he uses now.

“If there were a longer-lasting product, everyone would use it,” he says. “Donating a small amount of time is worth it for the bigger benefit that we could all experience if this works.”


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Clinical trials are studies involving patients that test a new drug, device or treatment. More than 200 clinical trials involving hemophilia and more than 50 involving von Willebrand disease (VWD) are under way worldwide, according to the National Institutes of Health’s Web site. The site also lists clinical trials looking at rare factor deficiencies and platelet disorders. Because the bleeding disorders community is so small, it will take many people to volunteer to participate in clinical trials for the entire community to gain more knowledge and develop new treatments.

“Persons with hemophilia should expect state-of-the-art care from their providers,” says Donna DiMichele, MD, professor of pediatrics at Weill Cornell Medical College and former director of the New York Comprehensive Center for Hemophilia and Coagulation Disorders in New York City. “But that state-of-the-art care is really only as good as the evidence that supports it. The way we get that evidence is through clinical trials.”

If you think clinical trials are inconvenient, unsafe or not confidential enough, think again. Clinical trials can be simple and easy. Consider this primer on what they are, what to ask and how to get involved.

The Phases of Clinical Trials

There are four stages of a clinical trial, and you may be asked to participate in any of them.

Phase 1
Phase 1 trials are usually small studies designed to test a theory or product. There can be more risk in this phase because the product or treatment may be untested except on animals.

Phase 2
If phase 1 is successful, the trial will proceed to a second phase, in which researchers will seek to confirm the findings of the first trial but with a larger group of subjects. The risks are typically lower in this case.

Phase 3
Phase 3 trials are done on an even larger group of subjects, seeking to test for side effects, confirm previous findings and compare the treatment to other more traditional treatments.

Phase 4
If the other phases go well, the treatment or product will be put out in the market. Phase 4 studies track the long-term effect of the treatment or product, as well as any side effects, on specific groups of people, which can sometimes be different than the groups studied in previous phases.

Are You a Control?
The gold standard of clinical trials is the double-blind placebo study. This type of study divides participants into two groups: people receiving the new treatment or product, and people who go on with their regular treatment or receive a harmless placebo version, like a sugar pill.

If you’re participating in a double-blind placebo study, you—and even the researchers—may not know which group you’re in. This “double-blind” aspect makes it more likely that the results will be impartial and more accurate.

Safety First

The first concern of any researcher—and anyone considering participating in a clinical trial—is safety. Most studies are conducted through HTCs and universities and are first reviewed by human subjects protection committees, also called institutional review boards (IRBs). These groups are made up of a broad cross-section of men and women in the scientific, nonscientific and bleeding disorders communities. The boards verify safety before the trial can begin and monitor the trials once under way. The studies adhere to federal safety standards mandated by Congress and enforced by NIH.

“There are a lot of safeguards put in place in advance of asking patients to participate in a clinical trial,” says DiMichele, who has conducted many clinical trials on people with bleeding disorders. “Studies are set up to maximize the benefits and minimize the risk to the person participating in the trial.”

For instance, in trials of new products, tests have usually already been done on animals, and the properties of proteins in the treatments are carefully studied, says Mark Weinstein, PhD, associate deputy director for scientific affairs at the US Food and Drug Administration’s Center for Biologics Evaluation and Research.

“The researchers conduct clinical trials because we’re not certain about the efficacy and safety of products, so there is a certain degree of risk,” Weinstein says. “But much work is done pre-clinical trial to make sure the study is as safe as possible.”

Safety levels for studies that will involve children are even more stringent. The Office for Human Research Protections at the US Department of Health and Human Services requires IRBs to consider a child’s ability to understand what’s happening to him. In many studies, children—not just their parents—are required to consent. Often, researchers are required to show that the procedures children will be exposed to are not much different than they would experience in the regular course of treatment.

There are protections in place for all study participants that they may not even be aware of. Craig Kessler, MD, chair of the National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council (MASAC), says researchers are going beyond ensuring the safety of the patient during the trial to ensure that any tissue collected from the patient is stored safely and confidentially long after the trial is completed.

Participating in a Clinical Trial

Because NIH and academic institutions have stringent standards, the best way to sign up for a clinical trial is to talk to your HTC and look for a trial on Any study published in an academic journal must first be listed at

Once you find a study, you’ll want to make sure you qualify. Not everyone will. Every trial has people who are allowed to participate and those who aren’t, says DiMichele. That’s because researchers are trying to eliminate variables that might throw off the results and invalidate the study.

Unfortunately, that often means people with co-morbidities—that is, one or more conditions beyond the medical condition targeted by the trial—can be excluded from clinical trials that would otherwise benefit them.

“It’s an uphill and selective battle,” Kessler says. “One thing we’re trying to do in MASAC is broaden inclusion definitions so people with bleeding disorders can participate. Many new drugs—for example, for hepatitis C—would benefit people with hemophilia, but the studies exclude them because of their hemophilia.” (See MASAC Recommendation #193, “Regarding Availability of HCV Treatment Trials.”) You can also sign a citizen petition to the FDA to make investigational drugs available for people with hemophilia and hepatitis C.

Children with bleeding disorders are also excluded from many clinical trials, even though researchers need to understand how possible treatments may work for them.

Questions to Ask

When you speak to the study coordinator (probably a nurse) about joining a trial, here are some of the questions you should ask:

  • What are the inclusion criteria (age, medical conditions, etc., required for participation in the study)?
  • What might exclude me from participating?
  • What will be asked of me in the trial?
  • How will the treatment be taken—at a doctor’s office or at home?
  • How will this care be different than my usual care? (This is especially important for parents to ask for children because it will affect your day-to-day life and your child’s schedule.)
  • What else will I be asked to do, such as keeping a drug journal?
  • What are the potential benefits of the treatment?
  • Will I be allowed to leave the trial if I become uncomfortable with it at any time? The answer to this question should always be “yes.” If not, avoid that trial, Kessler says.
  • What are the side effects and potential risks of the trial?

This last question is particularly important for your well-being. Wisconsin resident Shelly Mattson, 24, is an administrative assistant for the Great Lakes Hemophilia Foundation and is a past chair of the NHF’s National Youth Leadership Institute (NYLI). She has moderate VWD and participated in a clinical trial three years ago for a new treatment. Instead of her usual treatment, she was given Wilate for six weeks.

“I got a big packet with all the information about risks and benefits,” she says. “If you don’t feel like they’re giving you enough information, I would be suspicious.” So, read all the forms. If you don’t agree to what researchers want you to do or you don’t feel safe, don’t sign the forms.

Benefits of Participating in Clinical Trials

Mattson received a stipend of $500 for her participation in the trial, but such financial rewards are becoming more rare, Kessler says. Some studies will reimburse you for parking, but medical ethics rules prohibit researchers from paying people to participate. If you decide to do a trial, you may get to try a new treatment before it’s released to the public, and you may get free treatment or increased monitoring of your disorder without interference from insurance.

But the main benefit is knowing that you’re helping other members of the bleeding disorders community, Mattson says.

Not long ago, she read that Wilate would soon be available to consumers. Mattson hasn’t switched her treatment yet, but she said a thrill ran through her to know that some people will get better treatment because she took a few weeks out of her life to test out the drug.

“I got very excited,” she says. “I remember thinking, ‘That’s very cool. I was part of that.’”