IB1001-202
Research type
Research Study
Full title
Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multicenter, open-label, rater-blinded Phase II study
IRAS ID
260774
Contact name
Reena Sharma
Contact email
Sponsor organisation
IntraBio Ltd
Eudract number
2018-004406-25
ISRCTN Number
ISRCTN99999999
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 7 months, 17 days
Research summary
The primary purpose of the study is to evaluate the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and investigating the efficacy in terms of improving symptoms, functioning, and quality of life against the defined endpoints in patients with GM2.
GM2 is a rare, distressing, neurovisceral autosomal-recessive inherited metabolic, lysosomal storage disorder (LSD) that predominantly affects paediatric patients.
Patients with neurological onset early in life have more severe symptoms and deteriorate faster. GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) is estimated to affect 1:320,000 live births.
Currently there are limited, and no curative treatments approved for GM2 worldwide,
therefore, there is a strong need for the development of novel and more effective therapies to treat these intractable diseases.This Extension Phase will enroll patients who have completed Visit 6 of the parent study phase of IB1001-202. Patients will be offered the opportunity to participate in this Extension Phase if the safety and tolerability during the 42-day (+7 days) treatment period of the parent study are considered to be acceptable by their Principal Investigator (PI) and the PI determines further treatment with IB1001 to be in the patient’s best interest.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
19/NW/0157
Date of REC Opinion
20 Jun 2019
REC opinion
Further Information Favourable Opinion