Edmond J. Safra Accelerating Clinical Trials in Parkinson's Disease: A Multiarm Multi-stage Platform Trial

Summary

Parkinson's disease (PD) is currently the fastest-growing neurological condition globally. It is projected to affect 172,000 people in the UK by 2030,with the current annual cost to the country being \~£3.6 billion. The disease progressively impairs physical abilities, leading to increased disability, falls, and difficulties with speech, swallowing, mood, thinking, and memory. While existing treatments can alleviate some symptoms, their effectiveness diminishes over time, and they can cause severe side effects. This trial uses a Multi-Arm,Multi-Stage (MAMS) design where multiple treatments are tested simultaneously in separate groups, called "arms." Each treatment is compared against a placebo, a dummy treatment with no active ingredients, to evaluate its effectiveness and safety. Throughout the trial, each treatment undergoes periodic reviews, known as interim analyses, to assess its safety and potential benefits. If a treatment shows promise, it continues in the trial until a final assessment determines its overall effectiveness. Treatments that do not show positive results are discontinued and replaced with new candidates. This approach reduces the number of participants needed to obtain reliable results and is more cost-effective and faster than conducting separate trials for each treatment. The treatments selected for this trial were chosen based on careful consideration of existing evidence regarding their safety and effectiveness. To choose the treatments we want to test, we carefully considered evidence for safety and effectiveness. The trial will start with two treatment arms (telmisartan and terazosin) and one placebo arm, with a third treatment arm added after one year. We can identify new treatments to add to the trial each year. Participants will be followed up for up to 36 months. After an in-person screening visit, all remaining visits at 3 months,6 months and then every 6 months after, for a total of up to 36 months can be completed remotely. The visits will include questionnaires, assessment of Parkinson's symptoms and discussions about any side effects. Participants will informed of trial progress. Results will be shared via the trial website and published in a medical journal.

Conditions

• Parkinsons Disease (PD)

Interventions

DRUG

Placebo

An over-encapsulated placebo capsule taken once daily to match the treatment arms following a 5-week titration phase.

DRUG

Telmisartan

Over-encapsulated telmisartan 40mg per day for 36 months (plus their usual SoC) following a 5-week titration phase. Titration phase for telmisartan: Week 1-3: one 20 mg capsule per day; Week 4-5: one 40 mg capsule per day

DRUG

Terazosin (Hytrin)

Over-encapsulated terazosin 5mg per day for 36 months (plus their usual SoC) following a 5-week titration phase. Titration phase for terazosin: Week 1: one 1 mg capsule per day; Week 2: one 2 mg capsule per day; Week 3: one 3 mg capsule per day; Week 4: one 4 mg capsule per day; Week 5: one 5 mg capsule per day

Sponsors & Collaborators

• Cure Parkinson's

  collaborator OTHER

• Parkinson's UK

  collaborator OTHER

• National Institute for Health Research, United Kingdom

  collaborator OTHER_GOV

• John Black Charitable Foundation

  collaborator UNKNOWN

• Medical Research Council

  collaborator OTHER_GOV

• Newcastle University

  collaborator OTHER

• Van Andel Research Institute

  collaborator OTHER

• Michael J. Fox Foundation for Parkinson's Research

  collaborator OTHER

• Gatsby Foundation

  collaborator UNKNOWN

• University College, London

  lead OTHER

Principal Investigators

• Prof Thomas Foltynie · University College, London

• Prof Camille Carroll · Newcastle University

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

30 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-09-12

Primary Completion

2031-05-31

Completion

2031-07-31

Countries

• United Kingdom

Study Locations