Spinocerebellar Ataxia Type 3 Clinical Trial
Official title:
Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3
DESIGN: Pilot, Phase II, double-blind, placebo-controlled study
JUSTIFICATION: In the literature one does not find a pharmacological treatment that changes
the natural history of Spinocerebellar ataxtia type 3 (SCA3). Patients with this disease
invariably become dependent.
OBJECTIVES I. To determine safety and tolerability of phenylbutyrate in patients with SCA3.
II. To provide early subsidies on the efficacy of phenylbutyrate in SCA3.
DURATION: 12 months of a double-blind study.
PLACE OF REALIZATION: Hospital de Clínicas de Porto Alegre, Brazil.
NUMBER OF PATIENTS: 20 patients.
CONCOMITANT MEDICATIONS: There are no concomitant medications that are prohibited unless
they affect safety parameters of this study (hemogram and platelets; fasting serum glucose,
AST, ALT, Gamma-GT, Bilirubins, Prothrombin time, Creatinine, Urea, Na, K, chlorides and
arterial gasometry; electrocardiogram and echocardiogram).
MEDICATIONS UNDER INVESTIGATION: Powdered sodium phenylbutyrate in sachets containing each
3g. At the start of the study, the dose will be 15g/day (five sachets) and may be reduced in
case of mild adverse events.
OUTCOMES Primary safety outcome: The number of adverse events, interruptions and dose
reductions in the two groups (cases and controls).
Efficacy outcomes: Efficacy outcomes are the following scores in both groups: NESSCA, SARA,
Barthel, BDI, and WHOQol.
n/a
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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