Spinocerebellar Ataxia Type 3Phase 2NCT01096095
Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
A Phase 2 study testing Sodium Phenylbutyrate in people with Spinocerebellar Ataxia Type 3, run by Hospital de Clinicas de Porto Alegre at 1 site in Brazil.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 80 years
- Conditions the study is for
- Spinocerebellar Ataxia Type 3
- What is being tested?
- Sodium Phenylbutyrate
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteBrazil
- Study dates
- Jun 2010to Jul 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
Brazil1 site
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90.035-903
About this study
Official title: 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.
Read more
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.
- Conditions
- Spinocerebellar Ataxia Type 3
- Being tested
- Placebo, Sodium Phenylbutyrate
- Keywords
- SCA, Spinocerebellar Ataxia, Sodium Phenylbutyrate, Machado-Joseph Disease, Treatment
- Medical subject headings
- Machado-Joseph Disease, Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).
Sodium PhenylbutyrateDrug
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 group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
| Sodium phenylbutyrate | Experimental | Active drug Receives: Drug: Sodium Phenylbutyrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 6 months - 12 months
Secondary outcomes (1)
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.
Measured: 6 months - 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2010 (estimated)
- Study startJun 2010
- Primary completionDec 2010 (estimated)
- Study completionJul 2011 (estimated)
Why it stopped: Regulatory authorities did not allow the entrance of the study drug in the country
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia Type 3Studies of the same condition, open and under way.
- More trials run by Hospital de Clinicas de Porto AlegreThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital de Clinicas de Porto Alegre
- Sponsor type
- University, hospital or other
- Collaborators
- Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil
- Responsible party
- Sponsor
- Investigators
- Laura B Jardim, PhD, Principal investigator, Medical Genetics Service of Hospital de Clínicas de Porto Alegre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01096095
- Sponsor's protocol ID
- CONEP CAAE 0527.0.001.000-07
- Status last verified
- May 2010
- First submitted
- 29 Mar 2010
- Last updated
- 17 Aug 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3
- ClinicalTrials.gov ID
- NCT01096095
- Acronym / sponsor ID
- CONEP CAAE 0527.0.001.000-07
- Conditions
- Spinocerebellar Ataxia Type 3
- Keywords
- SCA, Spinocerebellar Ataxia, Sodium Phenylbutyrate, Machado-Joseph Disease, Treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Regulatory authorities did not allow the entrance of the study drug in the country
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Hospital de Clinicas de Porto AlegreUniversity, hospital or other
- Collaborators
- Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil
- Study start
- Jun 2010
- Primary completion
- Dec 2010 (estimated)
- Study completion
- Jul 2011 (estimated)
- First posted
- 30 Mar 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
| Sodium phenylbutyrate | Experimental | Drug: Sodium PhenylbutyrateActive drug |
Who may qualify
- Ages
- 16 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlaceboPowdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets). | Drug | Placebo |
| Sodium PhenylbutyratePowdered 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. | Drug | Sodium phenylbutyrate |
Primary outcome
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).
1 secondary outcome
Efficacy of phenylbutyrate in SCA3 on neurological dysfunction and quality of life
Time frame: 6 months - 12 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Laura B Jardim, PhD principal investigator
Medical Genetics Service of Hospital de Clínicas de Porto Alegre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sodium Phenylbutyrate in people with Spinocerebellar Ataxia Type 3, run by Hospital de Clinicas de Porto Alegre at 1 site in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 17 Aug 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of phenylbutyrate in patients with SCA3 (time frame: 6 months - 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Aug 2012; AzTrials last refreshed its copy on 8 Nov 2026.