Effects of Acetyl-DL-Leucine on cerebellar ataxia - a multinational, multicenter, randomized, double-blind, placebo-controlled, 2-way crossover phase III trial

Phase 3

• Conditions: G11.0; G11.1; G11.2; G11.8; G11.9; Congenital nonprogressive ataxia; Early-onset cerebellar ataxia; Late-onset cerebellar ataxia; Other hereditary ataxias; Hereditary ataxia, unspecified

• Registration Number: DRKS00009733
• Lead Sponsor: Klinikum der Universität München, Campus Großhadern

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-01-15
• Study Completion: 2017-07-07
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 108

Inclusion Criteria

Subjects will only be included in the study if they meet all of the following criteria:
•Clinically confirmed cerebellar ataxia (CA) with a total SARA-Score = 3 (range 0-40) of hereditary or non-hereditary degenerative type
•Patient did not receive any of the following prohibited medication within 4 weeks prior to randomization:
oAminopyridines (including substained-release form)
oAcetyl-DL-Leucine
oRiluzole
oGabapentin
oVarenicline
oChlorzoxazone
•The ability to follow study instructions and likely to attend and complete all required visits
•Written informed consent of the subject prior to any study specific intervention
•Age = 18 years

Exclusion Criteria

Subjects will not be included in the study if any of the following criteria applies:
•Subject is not able to give consent
•Onset of ataxia in association with stroke, encephalitis, sepsis, hyperthermia or heat stroke
•Toxic causes for ataxia of cerebellar type
•Rapid progression of ataxia (development of severe ataxia in less than 12 weeks)
•Subject suffers from any of the following:
ochronic diarrhea
ounexplained visual loss
omalignancies
oinsulin-dependent diabetes mellitus
•Ataxia due to multiple sclerosis, ischemia, hemorrhage or tumor of the posterior fossa as confirmed by imaging
•Ataxia due to clinical likely multisystem atrophy type C (MSA-C)
•Diagnosis of clinical likely Friedreich ataxia
•Known history of hypersensitivity to the investigational drug or derivates
•Liver failure defined as AST/ALT > 300 U/l
•Simultaneous participation in another clinical trial or participation in any clinical trial involving administration of an investigational medical product within 30 days prior to the beginning of the clinical trial
•Subjects with a physical or psychiatric condition which at the investigator’s discretion may put the subject at risk, may confound the trial results, or may interfere with the subject’s participation in this clinical trial
•Known or persistent abuse of medication, drugs or alcohol
•Females of childbearing potential, who are not using and not willing to use medically reliable methods of contraception for the entire study duration as listed in the patient informed consent form
•Current or planned pregnancy or nursing women
•Patients has received any of the following prohibited medication within 4 weeks prior to randomization
oAminopyridines (including substained-release form)
oAcetyl-DL-Leucine
oRiluzole
oGabapentin
oVarenicline
oChlorzoxazone

After a wash-out period of at least 4 weeks these patients can be included in the study.

Study & Design

• Study Type: interventional
• Study Design: Not specified