PR006A safety and effects on progranulin in fronto-temporal dementia
Research type
Research Study
Full title
A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of PR006A in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM)
IRAS ID
1003419
Contact name
Jonathan Rohrer
Contact email
Sponsor organisation
Prevail Therapeutics, A Wholly Owned Subsidiary of Eli Lilly & Company
Eudract number
2019-003159-12
Clinicaltrials.gov Identifier
Research summary
Fronto-temporal dementia (FTD) is caused by cell loss in the brain's frontal lobes (areas behind the forehead) or temporal lobes (areas behind the ears). FTD probably accounts for less than 5% of dementias but it is the second or third most common type presenting in those under 65. FTD is mostly inherited and mutations in the progranulin gene (GRN) which encodes the PGRN protein are a common cause.
No disease modifying treatments are available for patients with FTD. Current standard of care involves off-label use of existing drugs which only modestly address disease symptoms. PR006A is a gene therapy product, which involves adding genetic material into the cells to make a beneficial protein.
This is the first time that PR006A will be given to humans. The purpose of this study is to assess the safety and effects of increasing doses of PR006A on PGRN levels in patients with FTD-GRN.
Fifteen patients (3 cohorts of 5 patients each) will be administered a one-time dose of PR006A via an injection through the base of the skull. It is expected that up to 4 patients will be treated in the UK at the National Hospital of Neurology and Neurosurgery in London.
Patients will remain in the study for approximately 5 years. After screening, patients will attend study visits the day before the PR006A injection, they will undergo the PR006A injection and stay overnight, then they will visit the clinic on Day 7, 14, 21, Month 1, 2 and 3, every 3 months for the first year and then every 6 months. Study procedures include physical and neurological examinations, vital signs, brain MRI and MRA, DEXA scan, ECG, dementia assessments, blood and urine testing, lumbar puncture and CSF collection.
The study is sponsored by Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company.REC name
South Central - Oxford A Research Ethics Committee
REC reference
21/SC/0126
Date of REC Opinion
20 Jul 2021
REC opinion
Further Information Favourable Opinion