Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Spinocerebellar Ataxia Type 3 |
Drug: Placebo Drug: Sodium Phenylbutyrate |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3 |
- Safety and tolerability of phenylbutyrate in patients with SCA3 [ Time Frame: 6 months - 12 months ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: No ]Efficacy outcomes are the following scores in both groups: NESSCA, SARA, 9-hole peg board test, BDI, and WHOQol.
| Enrollment: | 0 |
| Study Start Date: | June 2010 |
| Estimated Study Completion Date: | July 2011 |
| Estimated Primary Completion Date: | December 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Placebo Comparator: Placebo
Placebo
|
Drug: 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
Active drug
|
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.
|
Eligibility| Ages Eligible for Study: | 16 Years to 80 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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.
Contacts and Locations| Brazil | |
| 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 |
More Information
No publications provided
| Responsible Party: | Hospital de Clinicas de Porto Alegre |
| ClinicalTrials.gov Identifier: | NCT01096095 History of Changes |
| Other Study ID Numbers: | CONEP CAAE 0527.0.001.000-07 |
| Study First Received: | March 29, 2010 |
| Last Updated: | August 15, 2012 |
| Health Authority: | Brazil: National Committee of Ethics in Research |
Keywords provided by Hospital de Clinicas de Porto Alegre:
|
SCA Spinocerebellar Ataxia Sodium Phenylbutyrate Machado-Joseph Disease Treatment |
Additional relevant MeSH terms:
|
4-phenylbutyric acid Ataxia Machado-Joseph Disease Spinocerebellar Ataxias Spinocerebellar Degenerations Dyskinesias Neurologic Manifestations Nervous System Diseases Signs and Symptoms Cerebellar Ataxia |
Cerebellar Diseases Brain Diseases Central Nervous System Diseases Spinal Cord Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Antineoplastic Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on June 17, 2013