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.
Status | Withdrawn |
Enrollment | 0 |
Est. completion date | July 2011 |
Est. primary completion date | December 2010 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 16 Years to 80 Years |
Eligibility |
Inclusion Criteria: 1. All patients shall have their molecular diagnosis confirmed and expanded polyglutamine tract (CAG) measures already determined. 2. Patients still able to walk with until 8 years of disease duration, and 3. Patients aged 16 years or over will be invited to participate in the study. Exclusion Criteria: 1. they show electrocardiogram ou echocardiographic alterations suggestive of heart insufficiency at baseline; 2. their serum creatinine levels are higher than 1.2 mg/dL, with the confirmation of renal insufficiency due to the rate of glomerular filtration; 3. they show a history of hypersensibility to sodium phenylbutyrate, and if 4. they (men and women) do not agree to use a reliable contraceptive method during the entire study period and for three months after its end. |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Brazil | Hospital de Clínicas de Porto Alegre | Porto Alegre | Rio Grande do Sul |
Lead Sponsor | Collaborator |
---|---|
Hospital de Clinicas de Porto Alegre | Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil |
Brazil,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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). | 6 months - 12 months | Yes |
Secondary | 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. | 6 months - 12 months | No |
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