Trial record 1 of 46 for:    phenylbutyrate
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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This study has been withdrawn prior to enrollment.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

March 29, 2010
August 15, 2012
June 2010
December 2010   (final data collection date for primary outcome measure)
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).
Same as current
Complete list of historical versions of study NCT01096095 on ClinicalTrials.gov Archive Site
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.
Same as current
Not Provided
Not Provided
 
Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3

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.

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Spinocerebellar Ataxia Type 3
  • 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.
  • Placebo Comparator: Placebo
    Placebo
    Intervention: Drug: Placebo
  • Experimental: Sodium phenylbutyrate
    Active drug
    Intervention: Drug: Sodium Phenylbutyrate
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Withdrawn
0
July 2011
December 2010   (final data collection date for primary outcome measure)

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.
Both
16 Years to 80 Years
No
Contact information is only displayed when the study is recruiting subjects
Brazil
 
NCT01096095
CONEP CAAE 0527.0.001.000-07
No
Hospital de Clinicas de Porto Alegre
Hospital de Clinicas de Porto Alegre
Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil
Principal Investigator: Laura B Jardim, PhD Medical Genetics Service of Hospital de Clínicas de Porto Alegre
Hospital de Clinicas de Porto Alegre
May 2010

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