Cerebellar ataxia is a form of ataxia originating in the cerebellum and is most often caused by neurodegenerative disorders of the cerebellum, either hereditary or sporadic. The leading clinical symptoms of cerebellar ataxia are disturbances of stance/gait (> 85%) with recurrent falls, limb ataxia with severe functional impairment of arm and hand movements, dysarthrophonia with impaired oral communication abilities and ocular motor disturbances with impaired vision.
Conditions
Interventions
Trade Name: Tanganil
Pharmaceutical Form: Tablet
INN or Proposed INN: ACETYLLEUCINE
Other descriptive name: Tanganil
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number:
Sponsors
Hospital of the University of Munich
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects will only be included in the study if they meet all of the following criteria: 1) Clinically confirmed cerebellar ataxia (CA) with a total SARA-Score = 3 (range 0-40) of hereditary or non-hereditary degenerative type 2) Patient did not receive any of the following prohibited medication within 4 weeks prior to randomization: aminopyridines, Acetyl-DL-Leucine, Riluzole, Gabapentin, Varenicline, Chlorzoxazone 3) The ability to follow study instructions and likely to attend and complete all required visits 4) Written informed consent of the subject prior to any study specific intervention 5) Age = 18 years Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 80 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 28
Exclusion criteria
Exclusion criteria: Subjects will not be included in the study if any of the following criteria applies: 1) Subject is not able to give consent 2) Onset of ataxia in association with stroke, encephalitis, sepsis, hyperthermia or heat stroke 3) Toxic causes for ataxia of cerebellar type 4) Rapid progression of ataxia (development of severe ataxia in less than 12 weeks) 5) Subject suffers from any of the following: o chronic diarrhea o unexplained visual loss o malignancies o insulin-dependent diabetes mellitus 6) Ataxia due to multiple sclerosis, ischemia, hemorrhage or tumor of the posterior fossa as confirmed by imaging 7) Ataxia due to clinical likely multisystem atrophy type C (MSA-C) 8) Diagnosis of clinical likely Friedreich ataxia 9) Known history of hypersensitivity to the investigational drug or derivates 10) Liver failure defined as AST/ALT > 300 U/l 11) 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 12) 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 13) Known or persistent abuse of medication, drugs or alcohol 14) 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 15) Current or planned pregnancy or nursing women 16) Patient has received any of the following prohibited medication within 4 weeks prior to randomization o Aminopyridines (including substained-release form) o Acetyl-DL-Leucine o Riluzole o Gabapentin o Varenicline o Chlorzoxazone
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary aim of the ALCAT trial is to determine the effect of Acetyl-DL-Leucine compared to placebo intervention on improving motor function measured by the total score of the Scale for the Assessment and Rating of Ataxia (SARA).;Secondary Objective: To demonstrate that Acetyl-DL-Leucine is efficacious in 1. improving motor function measured by the Spinocerebellar Ataxia Functional Index (SCAFI) and SARA subscore items 2. improving quality of life (EQ-5D-5L) as well as depression (Beck Depression Inventory, BDI-II) and fatigue (Fatigue Severity Score, FSS) 3. to check for the occurrence of adverse effects of the trial drug. 4. improving gait patterns measured by gait analysis (examination will be perfomed in the sub group of patients being enrolled in the trial center DSGZ) ;Primary end point(s): Primary efficacy endpoint: The primary efficacy endpoint is defined as the absolute changes in SARA total score from period-level baseline to week 2 after start of treatment start and to the end of the 6-weeks treatment period, i.e. the difference between post-treatment values and the corresponding period-level baseline values: Delta (SARAtotal) = SARAtotal (post-treatment) – SARAtotal (period baseline) ;Timepoint(s) of evaluation of this end point: after 2 and 6 weeks of treatment with IMP (for each treatment period) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Key secondary endpoint(s): SCAFI and subscores of SARA, QoL (EQ-5D-5L), depression (BDI-II), fatigue (FSS) Assessment of safety: Documentation and reporting of side effects (AEs, SAEs, SUSARs) ;Timepoint(s) of evaluation of this end point: 6 weeks of treatment with IMP (for each treatment period) as well as in follow-up 4 weeks after taking the last IMP. Absolute changes at the end of the 6-week treatment period compared to the period-level baseline defined as score at visit 2 (treatment period 1) and at visit 5 (treatment period 2) as well as 4 weeks after taking the last IMP (follow-up). Absolute changes in the coefficient of variation gait parameter of gait analysis (examination will be perfomed in the sub group of patients being enrolled in the trial center DSGZ). | — |
Countries
Austria, Germany
Contacts
Public ContactDSGZ Studienzentrale
Hospital of the University of Munich
Outcome results
None listed