Machado-Joseph Disease / Spinocerebellar Ataxia 3Phase 2NCT02147886
Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease
A Phase 2 study testing Cabaletta for IV infusion once weekly during 24 weeks in people with Machado-Joseph Disease / Spinocerebellar Ataxia 3, run by Bioblast Pharma Ltd. at 1 site in Israel.
Trial summary
This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm. Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly for 22 weeks (total of 24 weeks of treatment). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Machado-Joseph Disease / Spinocerebellar Ataxia 3
- What is being tested?
- Cabaletta for IV infusion once weekly during 24 weeks
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteIsrael
- Study dates
- Jul 2014to Nov 2016
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and women, 18 - 75 years
- Clinically diagnosed as Machado-Joseph disease/Spinocerebellar ataxia 3 confirmed by genetic testing
- With disease stage 2 or less
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
- Body Mass Index (BMI) ≤32 kg/m2.
- Ability to ambulate with or without assistance
Who cannot join
- Diabetes mellitus type 1 or 2
- Other major diseases
- Uncontrolled heart disease, chronic heart failure (CHF).
- Other neurological diseases.
- Ataxia derived from any other cause than genetically-confirmed spinocerebellar ataxia
- Presence of psychosis, bipolar disorder, untreated depression
- History of malignancy (except non-invasive skin malignancy).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Patients With Machado-Joseph Disease
- This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm.
- Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly for 22 weeks (total of 24 weeks of treatment).
- Being tested
- Cabaletta for IV infusion once weekly during 24 weeks
- Keywords
- Bioblast, Randomized, parallel-group, and dose-controlled
- Medical subject headings
- Machado-Joseph Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cabaletta for IV infusion once weekly during 24 weeksDrug
Cabaletta for IV infusion once weekly
Cabaletta for IV infusion once weekly during 24 weeksDrug
| Study group | Role | What they receive |
|---|---|---|
| Cabaletta 15gr | Experimental | Cabaletta 15gr Receives: Drug: Cabaletta for IV infusion once weekly during 24 weeks |
| Cabaletta 30gr | Experimental | Cabaletta 30gr Receives: Drug: Cabaletta for IV infusion once weekly during 24 weeks |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events
Safety will be evaluated on the basis of the following assessments:
Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Measured: 28 weeks
Physical examination
Safety will be evaluated on the basis of the following assessments:
Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Measured: 28 weeks
Vital signs
Safety will be evaluated on the basis of the following assessments:
Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Measured: 28weeks
12-lead ECG
Safety will be evaluated on the basis of the following assessments:
Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Measured: 28weeks
Safety laboratory tests
Safety will be evaluated on the basis of the following assessments:
Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Measured: 28weeks
Secondary outcomes (1)
Disease markers
Changes in disease markers will be assessed based on the following assessments:
Scale for the Assessment and Rating of Ataxia (SARA); Neurological Examination Score for Spinocerebellar Ataxia (NESSCA); Change in BMI - screening, spinocerebellar Ataxia Functional Tests; quality of life
Measured: 27 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2014 (estimated)
- Study startJul 2014
- Primary completionOct 2016
- Study completionNov 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Machado-Joseph Disease / Spinocerebellar Ataxia 3Studies of the same condition, open and under way.
- 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
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioblast Pharma Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Carlos Gordon, Prof., Principal investigator, Meir Medical Center Kfar Saba Israel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02147886
- Sponsor's protocol ID
- BB-MJD-201
- Status last verified
- Nov 2016
- First submitted
- 21 May 2014
- Last updated
- 22 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Patients With Machado-Joseph Disease
- ClinicalTrials.gov ID
- NCT02147886
- Acronym / sponsor ID
- BB-MJD-201
- Keywords
- Bioblast, Randomized, parallel-group, and dose-controlled
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Bioblast Pharma Ltd.Industry (pharma, biotech, devices)
- Study start
- Jul 2014
- Primary completion
- Oct 2016 (actual)
- Study completion
- Nov 2016 (actual)
- First posted
- 28 May 2014 (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.
- This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm.
- Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly for 22 weeks (total of 24 weeks of treatment).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cabaletta 15gr | Experimental | Drug: Cabaletta for IV infusion once weekly during 24 weeksCabaletta 15gr |
| Cabaletta 30gr | Experimental | Drug: Cabaletta for IV infusion once weekly during 24 weeksCabaletta 30gr |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Men and women, 18 - 75 years
- Clinically diagnosed as Machado-Joseph disease/Spinocerebellar ataxia 3 confirmed by genetic testing
- With disease stage 2 or less
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
- Body Mass Index (BMI) ≤32 kg/m2.
Key exclusion criteria (7 listed)
- Diabetes mellitus type 1 or 2
- Other major diseases
- Uncontrolled heart disease, chronic heart failure (CHF).
- Other neurological diseases.
- Ataxia derived from any other cause than genetically-confirmed spinocerebellar ataxia
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 |
|---|---|---|
| Cabaletta for IV infusion once weekly during 24 weeksCabaletta for IV infusion once weekly | Drug | Cabaletta 15gr |
| Cabaletta for IV infusion once weekly during 24 weeks | Drug | Cabaletta 30gr |
Primary outcome
Adverse events
Time frame: 28 weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Physical examination
Time frame: 28 weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Vital signs
Time frame: 28weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
12-lead ECG
Time frame: 28weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Safety laboratory tests
Time frame: 28weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
1 secondary outcome
Disease markers
Time frame: 27 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2016.
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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.
Carlos Gordon, Prof. principal investigator
Meir Medical Center Kfar Saba Israel
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 Cabaletta for IV infusion once weekly during 24 weeks in people with Machado-Joseph Disease / Spinocerebellar Ataxia 3, run by Bioblast Pharma Ltd. at 1 site in Israel.
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 6 inclusion and 7 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: finished, as of the sponsor's last update on 22 Nov 2016. 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 Israel. 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. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Adverse events (time frame: 28 weeks). The study lists 5 primary outcomes in all. 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 22 Nov 2016.