PK Study of MR Amifampridine Formulations (QSC202075)
Research type
Research Study
Full title
A Phase 1, Single-Site, Open-Label, Partially Randomised, 2 Part Study Designed to Evaluate the Pharmacokinetic Profile of Amifampridine following Administration of Modified Release Formulations and an Immediate Release Reference Formulation in Healthy Subjects
IRAS ID
281305
Contact name
Brian Elsbernd
Contact email
Sponsor organisation
Catalyst Pharmaceuticals, Inc.
Eudract number
2020-001360-28
Duration of Study in the UK
0 years, 1 months, 1 days
Research summary
The sponsor is developing the test medicine, Amifampridine, to treat Lambert-Eaton Myasthenic Syndrome (LEMS). LEMS is a rare disease caused by the body’s immune system attacking its own healthy cells, resulting in muscle weakness and tiredness. The test medicine is already marketed under the name FIRDAPSE for treatment of LEMS, however the sponsor are developing a new formulation (recipe) of the test medicine which would require fewer administrations per day.\n\nThe study will try to evaluate how the test medicine and its main metabolite (breakdown product) are taken up by the body compared to the reference product. It will also try to determine the proportion of the test medicine that enters the bloodstream compared to the reference product. The safety and tolerability of the test medicine will be assessed. \n\nThe study consists of up to two parts. Part one will enrol 13 healthy male and female volunteers. The volunteers will each receive three doses of the test medicine and one dose of the reference product. Volunteers will attend the clinical unit for one study period, receiving all four doses in this visit, with 48 hour washout periods between each dose. Volunteers will receive a follow-up call 5-7 days post-final dose. \n\nOptional Part two will enrol 20 healthy male and female volunteers. Volunteers will be split into two groups dependent on results of a genetic test. Each volunteer will receive one dose of the test medicine in up to three study periods, and receive the reference product as multiple doses on one study period. Volunteers will attend the clinical unit for up to three study visits. Volunteers will receive a follow-up call 5-7 days post-final dose. \n
REC name
HSC REC B
REC reference
20/NI/0063
Date of REC Opinion
14 Jul 2020
REC opinion
Further Information Favourable Opinion