Clinical Trials and NF
Clinical trials can be an important option for some individuals living with neurofibromatosis (NF). They are research studies designed to evaluate new treatments, new uses for existing treatments, or new approaches to care.
Participation is always voluntary. Deciding whether to join a clinical trial is a personal decision that should be made in consultation with your healthcare provider.
You do not have to make this decision alone.
What Is a Clinical Trial?
A clinical trial is a carefully designed research study in which volunteers receive investigational treatments under the supervision of physicians and research professionals.
These treatments may include medications, medical devices, surgical procedures, behavioural therapies, or supportive interventions.
Clinical trials help researchers determine whether a treatment is safe, effective, and appropriate for wider use. Almost every approved medical treatment has gone through clinical trials.
Clinical Trials in NF
In NF, clinical trials may involve testing new medications to slow or stop tumour growth, evaluating treatments for plexiform neurofibromas or schwannomas, comparing surgical approaches, or investigating therapies previously tested in other conditions.
Some treatments being studied may have been used before in other disorders but are new for NF. Others may be entirely new therapies.
Clinical Trials in NF
In NF, clinical trials may involve testing new medications to slow or stop tumour growth, evaluating treatments for plexiform neurofibromas or schwannomas, comparing surgical approaches, or investigating therapies previously tested in other conditions.
Some treatments being studied may have been used before in other disorders but are new for NF. Others may be entirely new therapies.
Who Is Eligible?
Having NF does not automatically make someone eligible for a clinical trial. Each study has specific inclusion and exclusion criteria designed to protect participant safety and ensure accurate study results. If you are interested in participating, speak with your physician first.
The Phases of Clinical Trials
Phase I: The first time a new treatment is tested in people. Researchers evaluate safety, dosage, and side effects. Usually involves a small number of participants.
Phase II: Continues safety evaluation and examines how well the treatment works.
Phase III: Compares the new treatment to current standard therapy. Often involves many participants across multiple centres and may last several years.
Phase IV: Occurs after a treatment is approved and on the market. Researchers gather long-term safety and effectiveness data.
Informed Decision-Making
Before enrolling, understand the purpose of the study, potential risks and benefits, time commitments, sponsorship, and what happens if you withdraw. You should never feel pressured to participate. Take your time and ask questions.
Questions to Ask Before Participating
- How will my information be stored and protected?
- What is the purpose of the study?
- Why might this treatment work for NF?
- What are the risks and potential benefits?
- How does it compare to my current treatment?
- Who is sponsoring the trial?
- Will travel or other expenses be reimbursed?
- What tests and follow-up visits are required?
- What happens after the trial ends?
Travel, Time and Financial Considerations
Participation may require travel within or outside British Columbia, time away from work or school, and multiple appointments over months or years. Ask whether travel, accommodation, or meal expenses are reimbursed before enrolling.
Your Rights as a Participant
Participation is voluntary. You may withdraw at any time. You must receive clear information about risks and benefits, and your personal health information must be protected.
Clinical Trials Involving Children
When children participate, parents or guardians provide consent. Whenever possible, children should also provide assent appropriate to their age and understanding. It may be helpful to consult a physician unaffiliated with the trial before making a decision.
Clinical Trials and Drug Approval in Canada
After clinical trials are completed, Health Canada reviews the evidence to determine safety and effectiveness. The Canada Drug Agency evaluates cost-effectiveness, and provincial governments decide on public funding. This process can take time.
Where to Find Active Clinical Trials
Search for recruiting clinical trials in Canada at ClinivalTrials.gov – search using “neurofibromatosis” and filter by location.
Final Thoughts
Clinical trials can bring hope and uncertainty. There is no single correct decision.
If you are considering participation, speak with your healthcare provider and take the time you need to make an informed choice.