Identify candidates for gene therapy
- Trial ID
- NCT06151002
- Official Title
- PD Frontline, Part of RAPSODI GD (Remote Assessment of Parkinsonism Supporting Ongoing Development of Interventions in Gaucher's Disease) to Build an Online Trial-ready Cohort of GBA Genotyped People With Parkinson's
- Goal
- Identify candidates for gene therapy
- Status
- RECRUITING
- Sponsor
- University College London Hospitals
- Study Type
- OBSERVATIONAL
- Enrollment
- 4800 participants
- Conditions
- Parkinson Disease
Summary For Families
The team is building an online, trial-ready group of people with Parkinson's whose GBA gene status is known, so future treatments aimed at GBA-related Parkinson's can find the right participants faster. They enroll people with a formal Parkinson's diagnosis and use remote assessments plus genetic testing to determine GBA variants, keeping participants in a registry for quick referral to GBA-focused studies; this is observational, not a treatment. Adults aged 18 to 90 with Parkinson's can join, but anyone already confirmed GBA-positive will be referred to the sister RAPSODI study instead. The study excludes other types of parkinsonism, other neurological disorders, and people taking medications known to cause parkinsonism.
Locations
- University College London (UCL), London, United Kingdom
Frequently Asked Questions
- What is this trial testing?
- This trial is studying an experimental treatment. The team is building an online, trial-ready group of people with Parkinson's whose GBA gene status is known, so future treatments aimed at GBA-related Parkinson's can find the right participants faster. They enroll people with a formal Parkinson's diagnosis and use remote assessments plus genetic testing to determine GBA variants, keeping participants in a registry for quick referral to GBA-focused studies; this is observational, not a treatment. Adults aged 18 to 90 with Parkinson's can join, but anyone already confirmed GBA-positive will be referred to the sister RAPSODI study instead. The study excludes other types of parkinsonism, other neurological disorders, and people taking medications known to cause parkinsonism.
- Who can participate?
- Participants must be between 18 Years and 90 Years.
- Where is this trial located?
- This trial is recruiting at 1 location.
- Does it cost anything to join?
- No. There is no cost to participate. Study-related care and treatment are provided at no charge.
- How long does the trial last?
- This trial is estimated to last approximately 5 years.
- Will I get the real drug or a placebo?
- It depends on the trial design. Some Parkinson's trials are placebo controlled, meaning a portion of participants get an inactive comparison while others get the experimental treatment. Some use crossover designs so everyone eventually receives the active treatment at some point. Observational trials do not use a placebo at all because they are not testing a new treatment. The consent form for any specific trial spells out the design and your odds of being assigned to placebo before you enroll.
- How often will I need to visit the study site?
- Visit frequency varies by trial. Many Parkinson's studies require an in person visit every 4 to 12 weeks during the active treatment phase, with shorter or longer gaps depending on the design. The site coordinator can give you the full visit schedule before you sign anything, so you know what the time commitment looks like.
- Can I leave the trial if I change my mind?
- Yes. You can withdraw from any clinical trial at any time, for any reason, without affecting your standard medical care. Trials are voluntary by law. The team may ask if you are willing to do a brief exit visit so they can collect safety information, but you are not obligated.
- Will travel or parking be reimbursed?
- Many trials reimburse for parking, mileage, and sometimes lodging if the site is far from your home. Reimbursement policies vary by sponsor and site. When you contact the trial team, ask specifically what is covered and how reimbursement is processed.
- Can my spouse or care partner come with me to visits?
- In most cases yes, and it is often encouraged. Care partners can help with notes, questions, and getting home safely after a long visit. Some study assessments do need to happen one on one, but care partners are usually welcome for the rest of the appointment.