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

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ClinicalTrials.gov Identifier: NCT01096095
Recruitment Status : Withdrawn (Regulatory authorities did not allow the entrance of the study drug in the country)
First Posted : March 30, 2010
Last Update Posted : August 17, 2012
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

Tracking Information
First Submitted Date  ICMJE March 29, 2010
First Posted Date  ICMJE March 30, 2010
Last Update Posted Date August 17, 2012
Study Start Date  ICMJE June 2010
Estimated Primary Completion Date December 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 29, 2010)
Safety and tolerability of phenylbutyrate in patients with SCA3 [ Time Frame: 6 months - 12 months ]
The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 29, 2010)
Efficacy of phenylbutyrate in SCA3 on neurological dysfunction and quality of life [ Time Frame: 6 months - 12 months ]
Efficacy outcomes are the following scores in both groups: NESSCA, SARA, 9-hole peg board test, BDI, and WHOQol.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
Official Title  ICMJE Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Spinocerebellar Ataxia Type 3
Intervention  ICMJE
  • Drug: Placebo
    Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).
  • 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.
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Placebo
    Intervention: Drug: Placebo
  • Experimental: Sodium phenylbutyrate
    Active drug
    Intervention: Drug: Sodium Phenylbutyrate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: August 15, 2012)
0
Original Estimated Enrollment  ICMJE
 (submitted: March 29, 2010)
20
Estimated Study Completion Date  ICMJE July 2011
Estimated Primary Completion Date December 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 Years to 80 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Brazil
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01096095
Other Study ID Numbers  ICMJE CONEP CAAE 0527.0.001.000-07
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Hospital de Clinicas de Porto Alegre
Study Sponsor  ICMJE Hospital de Clinicas de Porto Alegre
Collaborators  ICMJE Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil
Investigators  ICMJE
Principal Investigator: Laura B Jardim, PhD Medical Genetics Service of Hospital de Clínicas de Porto Alegre
PRS Account Hospital de Clinicas de Porto Alegre
Verification Date May 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP