Trial record 2 of 11 for:    Machado-Joseph Disease

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

This study has been withdrawn prior to enrollment.
(Regulatory authorities did not allow the entrance of the study drug in the country)
Sponsor:
Collaborator:
Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil
Information provided by (Responsible Party):
Hospital de Clinicas de Porto Alegre
ClinicalTrials.gov Identifier:
NCT01096095
First received: March 29, 2010
Last updated: August 15, 2012
Last verified: May 2010
  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

Resource links provided by NLM:


Further study details as provided by Hospital de Clinicas de Porto Alegre:

Primary Outcome Measures:
  • 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).


Secondary Outcome Measures:
  • 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
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01096095

Locations
Brazil
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil, 90.035-903
Sponsors and Collaborators
Hospital de Clinicas de Porto Alegre
Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil
Investigators
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:
Machado-Joseph Disease
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Dyskinesias
Neurologic Manifestations
Signs and Symptoms
Cerebellar Ataxia
4-phenylbutyric acid
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 17, 2014