Pilot Study of Safety and Efficacy of Sodium Phenylbutyra... | NCT01096095

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

NCT01096095

Sponsor

Hospital de Clinicas de Porto Alegre

Status

Withdrawn

Last Update Posted

Aug 17, 2012Estimated

Enrollment

0Actual

Phase

Phase 2

Conditions

Spinocerebellar Ataxia Type 3

Interventions

Placebo
Sodium Phenylbutyrate

Countries

Brazil

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2010

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Regulatory authorities did not allow the entrance of the study drug in the country

Expanded Access Info

Start Date

Jun 2010

Primary Completion Date

Dec 2010Estimated

Completion Date

Jul 2011Estimated

First Submitted Date

Mar 29, 2010

First Submission Date that Met QC Criteria

Mar 29, 2010

First Posted Date

Mar 30, 2010Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 15, 2012

Last Update Posted Date

Aug 17, 2012Estimated

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

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Placebo	Placebo Comparator	Placebo	Drug: Placebo
Sodium phenylbutyrate	Experimental	Active drug	Drug: Sodium Phenylbutyrate

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Placebo	Drug	Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).	Placebo

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Safety and tolerability of phenylbutyrate in patients with SCA3	The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).	6 months - 12 months

Secondary Outcomes

Measure	Description	Time Frame
Efficacy of phenylbutyrate in SCA3 on neurological dysfunction and quality of life	Efficacy outcomes are the following scores in both groups: NESSCA, SARA, 9-hole peg board test, BDI, and WHOQol.	6 months - 12 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

16 Years

Maximum Age

80 Years

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Laura B Jardim, PhD	Medical Genetics Service of Hospital de Clínicas de Porto Alegre	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital de Clínicas de Porto Alegre		Porto Alegre	Rio Grande do Sul	90.035-903	Brazil

Condition Browse Module

MeSH Terms

ID	Term
D017827	Machado-Joseph Disease
D020754	Spinocerebellar Ataxias

Ancestor Terms

ID	Term
D002524	Cerebellar Ataxia
D002526	Cerebellar Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C075773	4-phenylbutyric acid

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided