IB1001-203
Research type
Research Study
Full title
Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A multinational, multicenter, open-label, rater-blinded Phase II study.
IRAS ID
260363
Contact name
Anke Hensiek
Contact email
Sponsor organisation
IntraBio Ltd.
Eudract number
2018-004407-39
ISRCTN Number
ISRCTN99999999
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
not applicable, Not applicable
Duration of Study in the UK
0 years, 9 months, 18 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 Ataxia-Telangiectasia (A-T) and investigating the efficacy in terms of improving symptoms, functioning, and quality of life against the defined endpoints in patients with Ataxia-Telangiectasia (A-T).
A-T is a rare, devastating, autosomal-recessive cerebellar ataxia that causes progressive degeneration to the cerebellum, central nervous system (CNS), and immune system, resulting in cognitive and physical decline and premature death.
Patients with neurological onset early in life have more severe symptoms, deteriorate faster, and die sooner.
A-T is estimated to affect 1:40,000 to 1:100,000 live births. No medication has been proven effective for the treatment of inherited cerebellar ataxias, like A-T and there are no authorized treatments approved for A-T. Therefore, there is a strong need for the development of novel and more effective therapies to treat these intractable diseases.REC name
HSC REC A
REC reference
19/NI/0162
Date of REC Opinion
18 Sep 2019
REC opinion
Further Information Favourable Opinion