Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01096095
• Other study ID #: CONEP CAAE 0527.0.001.000-07
• Status: Withdrawn
• Phase: Phase 2
• First received: March 29, 2010
• Last updated: August 15, 2012
• Start date: June 2010
• Est. completion date: July 2011

Study information

• Verified date: May 2010
• Source: Hospital de Clinicas de Porto Alegre
• Contact: n/a
• Is FDA regulated: No
• Health authority: Brazil: National Committee of Ethics in Research
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Withdrawn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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Machado-Joseph Disease
• Spinocerebellar Ataxia Type 3
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

Drug:
• Placebo
Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).
• 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.

Locations

Country	Name	City	State
Brazil	Hospital de Clínicas de Porto Alegre	Porto Alegre	Rio Grande do Sul

Sponsors (2)

Lead Sponsor	Collaborator
Hospital de Clinicas de Porto Alegre	Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil

Country where clinical trial is conducted

Brazil, 

Outcome

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