Background
Acetyl-DL-leucine
Trial rationale
Methods/Design
Trial design and setting
Patient population and eligibility criteria
Inclusion criteria | Exclusion criteria |
---|---|
• Clinically confirmed CA with a SARA total score ≥ 3 (range 0-40) (CA (hereditary or non-hereditary, non-acquired) • Patient did not receive any of the following prohibited medication within 4 weeks prior to randomization: o Aminopyridineso Acetyl-DL-leucineo Riluzoleo Gabapentino Vareniclineo Chlorzoxazone• 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 | • 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: o chronic diarrheao unexplained visual losso malignancieso insulin-dependent diabetes mellitus• Ataxia due to multiple sclerosis, ischemia, hemorrhage or tumor of the posterior fossa as confirmed by imaging • Ataxia due to clinically likely multisystem atrophy type C (MSA-C) • Diagnosis of clinically likely Friedreich’s ataxia • Known history of hypersensitivity to the investigational drug or derivatives • 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, in the opinion of the investigator, 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 • Patient has received any of the following prohibited medication within 4 weeks prior to randomization o Aminopyridines (including sustained-release form)o Acetyl-DL-leucineo Riluzoleo Gabapentino Vareniclineo Chlorzoxazone |
Recruitment and patient involvement
Randomization, concealment, and blinding
Trial procedures and interventions
Enrollment | Treatment period 1 | Treatment period 2 | Close-out | |||||||
---|---|---|---|---|---|---|---|---|---|---|
Before | Eligibility Screening Visit 1c
| Visit 2 (Baseline) | Visit 3 2 weeks after V2 | Visit 4 6 weeks after V2 | Wash-out 4 weeks | Visit 5 | Visit 6 2 weeks after V5 | Visit 7 6 weeks after V5 | Follow-up Visit 8 4 weeks after V7 | |
Timeline (days) | 0/-28 | 0 | 14 | 42 | 70 | 70 | 84 | 112 | 140 | |
Informed consenta
| X | |||||||||
Inclusion/exclusion criteria | X | |||||||||
Medical history, including demographics & medications | X | |||||||||
Documentation of physiotherapy/speech therapy | X | Xb
| X | X | X | X | X | X | ||
Neurological examination | X | |||||||||
Blood tests | Xd
| Xd
| Xd
| Xd
| ||||||
Randomization | X | |||||||||
Dispensing of trial drug | X | X | ||||||||
Return of trial drug | X | X | ||||||||
Compliance check | X | X | X | X | ||||||
Patient questionnaires (EQ-5D-5 L, BDI-II, FSS) | X | X | X | X | X | X | X | |||
Ataxia rating scale: SARA | X | Xb
| X | X | X | X | X | X | ||
Ataxia rating scale: SCAFI | X | X | X | X | X | X | X | |||
Documentation of (S)AEs | X | Xb
| X | X | X | X | X | X | ||
Documentation of concomitant medication (drug history) | X | X | X | X | X | X | X |