Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
|ClinicalTrials.gov Identifier: NCT01096095|
Recruitment Status : Withdrawn (Regulatory authorities did not allow the entrance of the study drug in the country)
First Posted : March 30, 2010
Last Update Posted : August 17, 2012
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.
|Condition or disease||Intervention/treatment||Phase|
|Spinocerebellar Ataxia Type 3||Drug: Placebo Drug: Sodium Phenylbutyrate||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3|
|Study Start Date :||June 2010|
|Estimated Primary Completion Date :||December 2010|
|Estimated Study Completion Date :||July 2011|
Placebo Comparator: Placebo
Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).
Experimental: Sodium phenylbutyrate
Drug: Sodium Phenylbutyrate
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 [ Time Frame: 6 months - 12 months ]The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).
- Efficacy of phenylbutyrate in SCA3 on neurological dysfunction and quality of life [ Time Frame: 6 months - 12 months ]Efficacy outcomes are the following scores in both groups: NESSCA, SARA, 9-hole peg board test, BDI, and WHOQol.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01096095
|Hospital de Clínicas de Porto Alegre|
|Porto Alegre, Rio Grande do Sul, Brazil, 90.035-903|
|Principal Investigator:||Laura B Jardim, PhD||Medical Genetics Service of Hospital de Clínicas de Porto Alegre|