Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
Primary Purpose
Spinocerebellar Ataxia Type 3
Status
Withdrawn
Phase
Phase 2
Locations
Brazil
Study Type
Interventional
Intervention
Placebo
Sodium Phenylbutyrate
Sponsored by
About this trial
This is an interventional treatment trial for Spinocerebellar Ataxia Type 3 focused on measuring SCA, Spinocerebellar Ataxia, Sodium Phenylbutyrate, Machado-Joseph Disease, Treatment
Eligibility Criteria
Inclusion Criteria:
- All patients shall have their molecular diagnosis confirmed and expanded polyglutamine tract (CAG) measures already determined.
- Patients still able to walk with until 8 years of disease duration, and
- Patients aged 16 years or over will be invited to participate in the study.
Exclusion Criteria:
- they show electrocardiogram ou echocardiographic alterations suggestive of heart insufficiency at baseline;
- their serum creatinine levels are higher than 1.2 mg/dL, with the confirmation of renal insufficiency due to the rate of glomerular filtration;
- they show a history of hypersensibility to sodium phenylbutyrate, and if
- 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.
Sites / Locations
- Hospital de Clínicas de Porto Alegre
Arms of the Study
Arm 1
Arm 2
Arm Type
Placebo Comparator
Experimental
Arm Label
Placebo
Sodium phenylbutyrate
Arm Description
Placebo
Active drug
Outcomes
Primary Outcome Measures
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).
Secondary Outcome Measures
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.
Full Information
NCT ID
NCT01096095
First Posted
March 29, 2010
Last Updated
August 15, 2012
Sponsor
Hospital de Clinicas de Porto Alegre
Collaborators
Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil
1. Study Identification
Unique Protocol Identification Number
NCT01096095
Brief Title
Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
Official Title
Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3
Study Type
Interventional
2. Study Status
Record Verification Date
May 2010
Overall Recruitment Status
Withdrawn
Why Stopped
Regulatory authorities did not allow the entrance of the study drug in the country
Study Start Date
June 2010 (undefined)
Primary Completion Date
December 2010 (Anticipated)
Study Completion Date
July 2011 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hospital de Clinicas de Porto Alegre
Collaborators
Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinocerebellar Ataxia Type 3
Keywords
SCA, Spinocerebellar Ataxia, Sodium Phenylbutyrate, Machado-Joseph Disease, Treatment
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo
Arm Title
Sodium phenylbutyrate
Arm Type
Experimental
Arm Description
Active drug
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).
Intervention Type
Drug
Intervention Name(s)
Sodium Phenylbutyrate
Intervention Description
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.
Primary Outcome Measure Information:
Title
Safety and tolerability of phenylbutyrate in patients with SCA3
Description
The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).
Time Frame
6 months - 12 months
Secondary Outcome Measure Information:
Title
Efficacy of phenylbutyrate in SCA3 on neurological dysfunction and quality of life
Description
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
All patients shall have their molecular diagnosis confirmed and expanded polyglutamine tract (CAG) measures already determined.
Patients still able to walk with until 8 years of disease duration, and
Patients aged 16 years or over will be invited to participate in the study.
Exclusion Criteria:
they show electrocardiogram ou echocardiographic alterations suggestive of heart insufficiency at baseline;
their serum creatinine levels are higher than 1.2 mg/dL, with the confirmation of renal insufficiency due to the rate of glomerular filtration;
they show a history of hypersensibility to sodium phenylbutyrate, and if
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Laura B Jardim, PhD
Organizational Affiliation
Medical Genetics Service of Hospital de Clínicas de Porto Alegre
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital de Clínicas de Porto Alegre
City
Porto Alegre
State/Province
Rio Grande do Sul
ZIP/Postal Code
90.035-903
Country
Brazil
12. IPD Sharing Statement
Learn more about this trial
Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
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