MedDataX Logo

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

NCT ID: NCT01096095

Last Updated: 2012-08-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2010-06-30

Study Completion Date

2011-07-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Spinocerebellar Ataxia Type 3

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

SCA Spinocerebellar Ataxia Sodium Phenylbutyrate Machado-Joseph Disease Treatment

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Placebo

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).

Sodium phenylbutyrate

Active drug

Group Type EXPERIMENTAL

Sodium Phenylbutyrate

Intervention Type DRUG

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Placebo

Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).

Intervention Type 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.

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Minimum Eligible Age

16 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil

OTHER

Sponsor Role collaborator

Hospital de Clinicas de Porto Alegre

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Laura B Jardim, PhD

Role: PRINCIPAL_INVESTIGATOR

Medical Genetics Service of Hospital de Clínicas de Porto Alegre

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Hospital de Clínicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Brazil

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

CONEP CAAE 0527.0.001.000-07

Identifier Type: -

Identifier Source: org_study_id