How Research Turns into Treatments for HD
You can become involved in finding treatments for Huntington’s disease (HD) that may lead to improvements and potentially a cure by volunteering to help doctors and researchers learn more about HD
Types of Studies
| Clinical Trials (Interventional studies) | studies the effectiveness and safety of potential treatments on people |
| Observational Studies | Observes data or collects information from participants |
Stages of Research
| Pre-clinical | researchers investigate a new idea in their lab usually using animals or cell models. If promising, they apply to the FDA for an Investigational New Drug Application. |
| Clinical | 3 Phases of trials to understand the effectiveness and safety of treatments on volunteers. |
| FDA New Drug Application | reviewing and approval leads to the next stage. |
| After FDA Approval | researchers continue to monitor research volunteers for side effects and they submit their safety reports to the FDA. |
Phases of Clinical Trials
| Phase 1 | involves healthy volunteers finding out if a drug or therapy is safe and exploring different ways to administer treatments such as pills or injections. |
| Phase 2 | involves volunteers with HD to test if the experimental treatment is safe and effective. Some volunteers receive a placebo. |
| Phase 3 | given to a larger group of volunteers to learn about safety, effectiveness, and test for different dosage amounts. |
Key Terms
| Informed Consent | explains the purpose of the study, how long it’s expected to last, tests or procedures being done, risks and benefits, and whom to contact for further information. |
| Inclusion/Exclusion Criteria | are the reasons a person can/cannot participate in a trial. Each trial uses different criteria. |
| Open-Label Extension (OLE) | allows eligible participants to take the active drug instead of a placebo if a drug is found to have the potential for benefit. |
| Compassionate Use (Expanded Access) | allows the use of the treatment without FDA approval and outside of the clinical trial if an individual has a life-threatening condition. |
Key Questions to Ask
- What expenses do you cover?
- Do you provide a stipend for travel/food/lodging?
- Do you provide a stipend for my partner?
- Can I bring my children? What accommodations do you have for them? Do you provide a stipend for childcare?
- Do you have a stipend for pet care expenses?
- Do you offer a concierge service?
- What is your policy on open-label extension (OLE)?
- What is your policy on compassionate use?
- If I participate, will I be able to participate in other studies or future studies?
- Are you flexible on dates and times of visits?
- How many visits should I anticipate each month (or year)?
- When will the results from the study be available?
- How long will it take to administer the drug/therapy?
- Can I work/do other activities while receiving the drug/therapy?
- Who can I call between visits if I have questions or concerns?
- Do you provide any professional support such as a social worker or therapist?
- What other questions have other participants asked?
