Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study
- Conditions
- Niemann-Pick disease type C
- Registration Number
- 2023-510278-14-00
- Lead Sponsor
- Intrabio Limited
- Brief Summary
The primary objective is to evaluate the efficacy of N-Acetyl-L-Leucine based on the Scale for the Assessment and Rating of Ataxia (SARA) for the chronic treatment of NPC
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Ongoing, recruiting
- Sex
- Not specified
- Target Recruitment
- 60
Written informed consent signed by the patient and/or their legal representative/ parent/ impartial witness
Male or female aged ≥4 years with a confirmed diagnosis of NPC at the time of signing informed consent
Patients must fall within: a) A SARA score of 7 ≤ X ≤ 34 points (out of 40) AND b) Either: i. Within the 2-7 range (0-8 range) of the Gait subtest of the SARA scale OR ii. Be able to perform the 9-Hole Peg Test with Dominant Hand (9HPT-D) (SCAFI subtest) in 20 ≤ X ≤150 seconds.
Weight ≥15 kg at screening
Patients who have any known hypersensitivity or history of hypersensitivity to Acetyl leucine (DL, L, D) or derivatives, and or Excipients in the IB1001 or placebo sachets
Simultaneous participation in another clinical study or participation in any clinical study involving administration of an investigational medicinal product
Patients with a physical or psychiatric condition which, at the investigator’s discretion and in consultation with the Medical Monitor and Sponsor (as applicable), may put the patient at risk, may confound the study results, or may interfere with the patient’s participation in the clinical study, i.e. reliably perform study assessments
Patients who have been diagnosed with arthritis or other musculoskeletal disorders affecting joints, muscles, ligaments, and/or nerves that by themselves affects patient’s mobility and, at the investigator’s discretion, interferes with their ability to perform study assessments
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The primary endpoint for the study is of the Scale for the Assessment and Rating of Ataxia (SARA). In the US, the Modified SARA is the primary endpoint. The primary endpoint for the study is of the Scale for the Assessment and Rating of Ataxia (SARA). In the US, the Modified SARA is the primary endpoint.
- Secondary Outcome Measures
Name Time Method Modified Disability Rating Scale (mDRS) Modified Disability Rating Scale (mDRS)
Quality of Life EQ-5D-5L for patients aged ≥18; EQ-5D-Y for patients aged <18 years Quality of Life EQ-5D-5L for patients aged ≥18; EQ-5D-Y for patients aged <18 years
Spinocerebellar Ataxia Functional Index (SCAFI) Spinocerebellar Ataxia Functional Index (SCAFI)
Physician’s, Caregiver’s (if applicable), and Patient’s (if able) Clinical Global Impression of Improvement (CGI-I) comparing end of period I (Visit 4) to baseline (Visit 2), and end of period II (Visit 6) to end of period I (Visit 4 Physician’s, Caregiver’s (if applicable), and Patient’s (if able) Clinical Global Impression of Improvement (CGI-I) comparing end of period I (Visit 4) to baseline (Visit 2), and end of period II (Visit 6) to end of period I (Visit 4
Trial Locations
- Locations (6)
Vseobecna Fakultni Nemocnice V Praze
🇨🇿Prague, Czechia
Academisch Medisch Centrum
🇳🇱Amsterdam, Netherlands
Comenius University Bratislava
🇸🇰Bratislava, Slovakia
SphinCS GmbH
🇩🇪Hochheim Am Main, Germany
Justus-Liebig-Universitaet Giessen
🇩🇪Giessen, Germany
Universitaet Muenster
🇩🇪Muenster, Germany
Vseobecna Fakultni Nemocnice V Praze🇨🇿Prague, CzechiaStella ReichmannovaSite contact00420224967794Stella.Reichmannova@vfn.cz