Oculopharyngeal Muscular DystrophyPhase 2NCT04226924
Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
A Phase 2 study testing Trehalose in people with Oculopharyngeal Muscular Dystrophy, run by Bioblast Pharma Ltd. at 3 sites in Canada.
Trial summary
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period… Read more about this study
- Who is it for?
- Ages 50 to 70 years
- Conditions the study is for
- Oculopharyngeal Muscular Dystrophy
- What is being tested?
- Trehalose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 3 sitesCanada
- Study dates
- 15 Jun 2017to 15 Aug 2018
- 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 50 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Genetically confirmed OPMD with a (GCN)13 size PABPN1 mutation
- A score greater than 235 on the Sydney Swallow Questionnaire at screening
- Confirmation of oropharyngeal dysfunction by abnormal ice-cold water drinking test result, defined as drinking 80 cc of ice-cold water in ≥ 8 seconds at both drinking tests (at least 1 week apart) during the screening period
Who cannot join
- History of pharyngeal myotomy.
- Esophageal dilatation within the last 12 months.
- Treatment with botulinum toxin (any location) within 1 year prior to screening.
- Diagnosis of any other muscle disorder.
- Prior head and neck surgery or radiation.
- Oropharyngeal injury or oropharyngeal cancer.
Show all 17Show fewer
- Other esophageal disease that may be the cause of the dysphagia.
- Previously diagnosed with diabetes or a hemoglobin A1c (HgbA1c) result > 6.0% at screening.
- Prior treatment with IV trehalose.
- Known hypersensitivity to trehalose.
- Non-ambulatory (Use of a cane or short leg braces are permitted).
- Prior history of stroke (ischemic or hemorrhagic).
- Pregnancy or breast feeding.
- History of alcohol or drug abuse within the last 5 years.
- Evidence of hepatitis B, hepatitis C, or HIV infection at screening.
- Currently receiving anti-coagulant treatment (e.g., warfarin, enoxaparin) other than anti-platelet treatments, which are not a reason for exclusion.
- Currently participating in another clinical trial or has completed an interventional trial less than 90 days prior to planned first dosing.
Trial locations and contacts
3 sites in 1 country.
Canada3 sites
Ecogene-21
Chicoutimi, Quebec, G7H 7K9
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
CHU de Québec-Université Laval- Hôpital Enfant-Jésus
Québec, Quebec, G1J 1Z4
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment of
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.
Read the detailed description
After signing informed consent, patients will undergo two rounds of ice-cold water and nectar drinking tests at least 1 week apart to confirm oropharyngeal dysfunction. Patients who have confirmed oropharyngeal dysfunction, i.e., an ice-cold water drinking test time of 8 seconds or greater at both rounds, in addition to an SSQ score of >235, will be enrolled. Baseline values for all safety and efficacy parameters will be established during the screening period. Patients will be randomized in a 1:1 ratio, to trehalose or placebo, at the time of enrollment. Randomization will be stratified according to the patient's score on the SSQ at screening (≤ 799 or ≥ 800).
Patients randomized to trehalose will receive a 1-hour IV infusion of trehalose at a dose of 0.75 g/kg weekly for 24 weeks. Patients randomized to placebo (normal saline) will receive a weight-based equal volume of placebo weekly for 24 weeks.
After Week 24, patients may transition to an open-label extension of the study (extension period). During the extension period, patients will be treated with weekly infusion of trehalose at a dose of 0.75 g/kg for 24 weeks, followed by a 4-week safety follow-up (total duration of study = 56 weeks).
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Being tested
- Trehalose
- Keywords
- OPMD, DMOP, Dysphagia
- Medical subject headings
- Muscular Dystrophy, Oculopharyngeal, Deglutition Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrehaloseDrug
90 mg/ml trehalose solution for IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Trehalose | Experimental | Trehalose 9% solution: The dose is 0.75 g/kg administered IV over 60 ± 5 minutes once weekly. Receives: Drug: Trehalose |
| 0.9% Normal Saline | Placebo comparator | Normal saline: weight-based volume administered IV over 60 ± 5 minutes once weekly. Receives: Drug: Trehalose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Drinking Test Time
Change from baseline in timed drinking tests with 80 cc of ice-cold water and nectar.
Measured: 24 weeks
Secondary outcomes (7)
Muscle Strength Testing
Change from baseline in strength tests in selected muscle groups as measured by a handheld dynamometer
Measured: 24 weeks
Stair Climb Test
Change from baseline in functional muscle testing as measured by the Stair Climb test
Measured: 24 weeks
Timed Up and Go Test
Change from baseline in functional muscle testing as measured by the Timed Up and Go (TUG) test
Measured: 24 weeks
30-Second Lift Test
Change from baseline in functional muscle testing as measured by 30-Second Lift test
Measured: 24 weeks
EuroQol-5D-5L
Change from baseline in health status using the EuroQol-5D-5L Questionnaire
Measured: 24 weeks
Swallowing Quality of Life
Change from baseline in quality of life using modified Swallowing Quality of Life Questionnaire
Measured: 24 weeks
Sydney Swallow Questionnaire
Change from baseline in quality of life using Sydney Swallow Questionnaire
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2020
- Study start15 Jun 2017
- Primary completion15 Feb 2018 (estimated)
- Study completion15 Aug 2018 (estimated)
Why it stopped: Lack of funding for company
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oculopharyngeal Muscular DystrophyStudies 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
- Crossover
- Model description
- The study includes a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioblast Pharma Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bernard Brais, MD, Principal investigator, McGill University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04226924
- Sponsor's protocol ID
- BB-OPMD-202
- Status last verified
- Jan 2020
- First submitted
- 15 Jul 2019
- Last updated
- 13 Jan 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment of
- ClinicalTrials.gov ID
- NCT04226924
- Acronym / sponsor ID
- BB-OPMD-202
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Keywords
- OPMD, DMOP, Dysphagia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of funding for company
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Bioblast Pharma Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Jun 2017 (actual)
- Primary completion
- 15 Feb 2018 (estimated)
- Study completion
- 15 Aug 2018 (estimated)
- First posted
- 13 Jan 2020 (actual)
- 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.
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.
Detailed description
After signing informed consent, patients will undergo two rounds of ice-cold water and nectar drinking tests at least 1 week apart to confirm oropharyngeal dysfunction. Patients who have confirmed oropharyngeal dysfunction, i.e., an ice-cold water drinking test time of 8 seconds or greater at both rounds, in addition to an SSQ score of >235, will be enrolled. Baseline values for all safety and efficacy parameters will be established during the screening period. Patients will be randomized in a 1:1 ratio, to trehalose or placebo, at the time of enrollment. Randomization will be stratified according to the patient's score on the SSQ at screening (≤ 799 or ≥ 800).
Patients randomized to trehalose will receive a 1-hour IV infusion of trehalose at a dose of 0.75 g/kg weekly for 24 weeks. Patients randomized to placebo (normal saline) will receive a weight-based equal volume of placebo weekly for 24 weeks.
After Week 24, patients may transition to an open-label extension of the study (extension period). During the extension period, patients will be treated with weekly infusion of trehalose at a dose of 0.75 g/kg for 24 weeks, followed by a 4-week safety follow-up (total duration of study = 56 weeks).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- The study includes a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose.
| Arm | Type | Receives |
|---|---|---|
| Trehalose | Experimental | Drug: TrehaloseTrehalose 9% solution: The dose is 0.75 g/kg administered IV over 60 ± 5 minutes once weekly. |
| 0.9% Normal Saline | Placebo comparator | Drug: TrehaloseNormal saline: weight-based volume administered IV over 60 ± 5 minutes once weekly. |
Who may qualify
- Ages
- 50 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Genetically confirmed OPMD with a (GCN)13 size PABPN1 mutation
- A score greater than 235 on the Sydney Swallow Questionnaire at screening
- Confirmation of oropharyngeal dysfunction by abnormal ice-cold water drinking test result, defined as drinking 80 cc of ice-cold water in ≥ 8 seconds at both drinking tests (at least 1 week apart) during the screening period
Key exclusion criteria (17 listed)
- History of pharyngeal myotomy.
- Esophageal dilatation within the last 12 months.
- Treatment with botulinum toxin (any location) within 1 year prior to screening.
- Diagnosis of any other muscle disorder.
- Prior head and neck surgery or radiation.
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 |
|---|---|---|
| Trehalose90 mg/ml trehalose solution for IV infusion | Drug | 0.9% Normal Saline, Trehalose |
Primary outcome
Drinking Test Time
Time frame: 24 weeks
Change from baseline in timed drinking tests with 80 cc of ice-cold water and nectar.
7 secondary outcomes
Muscle Strength Testing
Time frame: 24 weeks
Stair Climb Test
Time frame: 24 weeks
Timed Up and Go Test
Time frame: 24 weeks
30-Second Lift Test
Time frame: 24 weeks
EuroQol-5D-5L
Time frame: 24 weeks
Swallowing Quality of Life
Time frame: 24 weeks
Sydney Swallow Questionnaire
Time frame: 24 weeks
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 13 Jan 2020.
- Individual participant dataSharing planned: No
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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, MD principal investigator
McGill University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Trehalose in people with Oculopharyngeal Muscular Dystrophy, run by Bioblast Pharma Ltd. at 3 sites 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 3 inclusion and 17 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 13 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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. 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?
Drinking Test Time (time frame: 24 weeks). 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 13 Jan 2020.