A Phase 2 interventional study of Placebo and Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3, sponsored by Hospital de Clinicas de Porto Alegre. Withdrawn at 1 site in Brazil. Open to participants aged 16 Years to 80 Years. Per ClinicalTrials.gov, last updated 2012-08-17.
Sponsored by Hospital de Clinicas de Porto Alegre · Phase 2, Interventional, and Treatment
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.
295 studies on the registry are indexed under Ataxia; 51 are open to participants now.
Browse Ataxia studies →Hospital de Clinicas de Porto Alegre is the lead sponsor of 450 studies on the registry; 61 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Placebo
Drug: Placebo
Active drug
Drug: Sodium Phenylbutyrate
Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).
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.
Safety and tolerability of phenylbutyrate in patients with SCA3
The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).
Time frame: 6 months - 12 months
Efficacy of phenylbutyrate in SCA3 on neurological dysfunction and quality of life
Efficacy outcomes are the following scores in both groups: NESSCA, SARA, 9-hole peg board test, BDI, and WHOQol.
Time frame: 6 months - 12 months
This study is withdrawn, as verified in May 2010. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Hospital de Clinicas de Porto Alegre