Intrabio Limited

Address: Summit House, 170 Finchley Road

Sponsor Overview

assignment

Specializations

Rare neurological disorders

Intrabio Limited focuses on rare neurodegenerative and movement disorders, with research activity centred on conditions that affect coordination, balance, and motor control. Its clinical programme includes studies in Ataxia Telangiectasia, CACNA1A disorders, and Niemann-Pick disease type C.

Ataxia Telangiectasia
CACNA1A disorders
Niemann-Pick disease type C

The sponsor’s research portfolio is concentrated on neurological conditions with limited treatment options and overlapping features of ataxia and impaired motor function.

Ataxia-focused clinical research

Clinical trials supported by Intrabio Limited place strong emphasis on ataxia as a core therapeutic domain, using outcome measures relevant to gait, coordination, and functional mobility. This includes work across both inherited cerebellar disorders and broader syndromes presenting with progressive movement impairment.

Ataxia assessment
Cerebellar dysfunction
Motor coordination impairment

Research activity in this area reflects a clear interest in neurological symptom control and functional improvement in chronic rare disease settings.

Inherited metabolic and lysosomal disease

The sponsor is also active in inherited metabolic disorders, particularly lysosomal storage disease research involving Niemann-Pick disease type C. This area addresses neurological and systemic disease manifestations linked to abnormal cellular processing and progressive disability.

Lysosomal storage disorders
Niemann-Pick disease type C
Neurovisceral disease

These studies position the sponsor within a therapeutic field that combines neurology, metabolism, and rare disease medicine.

Therapeutic interest in N-Acetyl-L-Leucine

Intrabio Limited’s clinical research centres on N-Acetyl-L-Leucine as a treatment candidate across multiple rare neurological diseases. The trials examine its role in managing chronic symptoms associated with ataxia and related neurological impairment.

N-Acetyl-L-Leucine
Chronic neurological symptom management
Rare disease therapeutics

The sponsor’s funded studies are directed toward conditions where preserving mobility, coordination, and neurological function is a major clinical concern.

Trial Name	Country	Status
A Phase III, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of N-Acetyl-L-Leucine in Patients with CACNA1A Disorders	Austria Germany Greece Italy	Recruiting
Evaluation of N-Acetyl-L-Leucine Efficacy in Ataxia-Telangiectasia: A Phase III Randomized, Placebo-Controlled, Double-Blind, Crossover Study	Germany Slovakia Spain	Recruiting
Phase III Randomized Double-Blind Crossover Study of N-Acetyl-L-Leucine in Niemann-Pick Disease Type C	Czechia Germany Slovakia The Netherlands	Recruiting