Muscular Dystrophy, Oculopharyngeal (OPMD)Phase 3HOPEMDNCT02328482
Continuation Protocol to Protocol BBCO-001
A Phase 3 study testing Tehalose 30gr in people with Muscular Dystrophy, Oculopharyngeal (OPMD), run by Bioblast Pharma Ltd. at 1 site in Canada.
Trial summary
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Muscular Dystrophy, Oculopharyngeal (OPMD)
- What is being tested?
- Tehalose 30gr
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteCanada
- Study dates
- Jan 2015to Dec 2017
- Phase
- Phase 3
- 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult men and women who participated and completed study BBCO-001
- Clinical and genetic diagnosis of OPMD
- Able to provide written informed consent to participate in this study
- Able to understand the requirements of the study and willing to comply with the requirements of the study
Who cannot join
- Pregnant or lactating
- Currently receiving anticoagulant treatment (e.g., warfarin)
- Any life-threatening illness, medical condition or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety
- Known hypersensitivity to any ingredient in the Cabaletta IV infusion
- Currently participating in another clinical trial (other than BBCO-001) or have completed an interventional trial less than 30 days prior to the planned treatment start date
Trial locations and contacts
1 site in 1 country.
Canada1 site
Montreal Neurological Institute, McGill University
Montreal, Quebec, H3A 2B4
About this study
Official title: A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in OPMD Patients Who Participated in Study BBCO-001
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
Read the detailed description
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
Eligible patients will be randomized to one of the following treatment arms:
- Treatment Arm 1: active treatment; continuation of 30 g IV Cabaletta once a week over an additional 52 weeks.
- Treatment Arm 2: no-treatment concurrent control; discontinuation of IV Cabaletta and follow-up over 52 weeks.
IV Cabaletta will be administered once a week to patients in Treatment Arm 1. All patients, regardless of treatment arm allocation, will undergo the same safety and efficacy assessments during the monthly site visits.
- Being tested
- Tehalose 30gr
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Oculopharyngeal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tehalose 30grDrug
Also called Cabellta
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Trehalose 30 g for IV infusion administered every week over an additional 52 weeks Receives: Drug: Tehalose 30gr |
| Arm 2 | No intervention | no-treatment concurrent control; follow-up over 52 weeks |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in disease markers
long term effect of Cabaletta on disease progression as measured by the changes in the disease markers
Measured: 52 weeks
Change in swallowing quality of life
long term effect of Cabaletta on disease progression as measured by the changes in the patient's swallowing quality of life
Measured: 52 weeks
Secondary outcomes (1)
Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g
The safety and tolerability will be evaluated by measuring the adverse events, vital signs, safety labs and physical examination during the entire study period. The number of events of change in the safety evaluations will be compared between the treatment and non treatment groups.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2014 (estimated)
- Study startJan 2015
- Primary completionOct 2017
- Study completionDec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophy, Oculopharyngeal (OPMD)Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioblast Pharma Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bernard Brais, Principal investigator, McGill University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02328482
- Sponsor's protocol ID
- BB-OPMD-301
- Acronym
- HOPEMD
- Status last verified
- Feb 2018
- First submitted
- 25 Dec 2014
- Last updated
- 17 Jul 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in OPMD Patients Who Participated in Study BBCO-001
- ClinicalTrials.gov ID
- NCT02328482
- Acronym / sponsor ID
- HOPEMD / BB-OPMD-301
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Bioblast Pharma Ltd.Industry (pharma, biotech, devices)
- Study start
- Jan 2015 (actual)
- Primary completion
- Oct 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 31 Dec 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 will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
Detailed description
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
Eligible patients will be randomized to one of the following treatment arms:
- Treatment Arm 1: active treatment; continuation of 30 g IV Cabaletta once a week over an additional 52 weeks.
- Treatment Arm 2: no-treatment concurrent control; discontinuation of IV Cabaletta and follow-up over 52 weeks.
IV Cabaletta will be administered once a week to patients in Treatment Arm 1. All patients, regardless of treatment arm allocation, will undergo the same safety and efficacy assessments during the monthly site visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: Tehalose 30grTrehalose 30 g for IV infusion administered every week over an additional 52 weeks |
| Arm 2 | No intervention | –no-treatment concurrent control; follow-up over 52 weeks |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Adult men and women who participated and completed study BBCO-001
- Clinical and genetic diagnosis of OPMD
- Able to provide written informed consent to participate in this study
- Able to understand the requirements of the study and willing to comply with the requirements of the study
Key exclusion criteria (5 listed)
- Pregnant or lactating
- Currently receiving anticoagulant treatment (e.g., warfarin)
- Any life-threatening illness, medical condition or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety
- Known hypersensitivity to any ingredient in the Cabaletta IV infusion
- Currently participating in another clinical trial (other than BBCO-001) or have completed an interventional trial less than 30 days prior to the planned treatment start date
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 |
|---|---|---|
| Tehalose 30grAlso: CabelltaTrehalose 30 g for IV infusion administered every week over an additional 52 weeks | Drug | Arm 1 |
Primary outcome
Change in disease markers
Time frame: 52 weeks
long term effect of Cabaletta on disease progression as measured by the changes in the disease markers
Change in swallowing quality of life
Time frame: 52 weeks
long term effect of Cabaletta on disease progression as measured by the changes in the patient's swallowing quality of life
1 secondary outcome
Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g
Time frame: 52 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 17 Jul 2019.
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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.
Bernard Brais principal investigator
McGill University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Tehalose 30gr in people with Muscular Dystrophy, Oculopharyngeal (OPMD), run by Bioblast Pharma Ltd. at 1 site in Canada.
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 4 inclusion and 5 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 17 Jul 2019. 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 Canada. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Change in disease markers (time frame: 52 weeks). The study lists 2 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 17 Jul 2019.