Oculopharyngeal Muscular DystrophyPhase 2HOPEMDNCT02015481
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
A Phase 2 study testing Cabaletta in people with Oculopharyngeal Muscular Dystrophy, run by Bioblast Pharma Ltd. at 4 sites in 3 countries.
Trial summary
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Oculopharyngeal Muscular Dystrophy
- What is being tested?
- Cabaletta
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 4 sitesin 3 countries
- Study dates
- Feb 2014to Apr 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Males and females
- 18 - 80 years (inclusive) of age
- Clinically and genetically diagnosed as OPMD
- Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
- Patients who provide written informed consent to participate in the study
- Body Mass Index (BMI) <30 kg/m2
Who cannot join
- Diabetes mellitus type 1 or 2
- Other major diseases, e.g.: renal failure (creatinine clearance <60ml/min), liver failure and chronic liver diseases (e.g. hepatitis B or C) , HIV carriers, tuberculosis, SLE, rheumatoid polyarthritis, sarcoidosis, collagenosis
- Uncontrolled heart disease , CHF,
- Other neuromuscular diseases
- Other disorders associated with esophageal dysphagia: e.g. severe gastroesophageal reflux (GERD), esophageal stricture due to mechanical or chemical trauma, infection (e.g. esophageal moniliasis), drug-induced dysphagia (e.g. bisphosphonates), esophageal rings and webs, spastic motility disorders of the esophagus.
- History of malignancy (except non-invasive skin malignancy)
Show all 13Show fewer
- History of neck irradiation
- Pregnant or currently lactating women
- Obesity (BMI≥ 30) and associated morbidity
- Prior pharyngeal myotomy
- Weight loss of more than 10% in the last 12 months.
- Known hypersensitivity to any ingredients in the injection
- Patient receiving anticoagulant treatment (e.g. warfarin)
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
United States2 sites
UCLA
Los Angeles, California, 90095
Tahseen Mozaffar
Orange, California, 92868
Canada1 site
Montreal Neurological Institute, McGill University
Montreal, Quebec, H3A 2B4
About this study
Official title: Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Being tested
- Cabaletta
- Keywords
- Case control, safety, tolerability, efficacy.
- Medical subject headings
- Muscular Dystrophy, Oculopharyngeal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CabalettaDrug
| Study group | Role | What they receive |
|---|---|---|
| Cabaletta 30gr. | Experimental | weekly IV of Cabaletta 30gr. Receives: Drug: Cabaletta |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Lab Evaluations
Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
Measured: 24 weeks
Secondary outcomes (3)
Drinking Test Score
Change from baseline in ice water drinking time, in seconds, at week 24. Times greater than 8 seconds to complete the drinking test are considered abnormal.
Measured: 24 weeks
Videofluoroscopy (VFS) Score
Penetration aspiration score results assessed by VFS comparing baseline, prior to treatment, to week 24. This is an 8 point scale. The higher the number the greater the risk of aspiration. The result reported is the difference from the baseline scores.
Measured: 24 Weeks
SWAL-QOL, Swallowing Quality of Life Questionnaire
Summary of Quality of Life in Swallowing Disorders total symptom score results over time, change from baseline at weeks 12 and 24 This is a 100 point scale. The higher the number the better the quality of life.
Measured: 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2013 (estimated)
- Study startFeb 2014
- Primary completionApr 2016
- Study completionApr 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioblast Pharma Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yoseph Caraco, M.D, Principal investigator, Hadassah Medical OrganizationBernard Brais, MD, Principal investigator, McGill University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02015481
- Sponsor's protocol ID
- BBCO-001
- Acronym
- HOPEMD
- Status last verified
- Sep 2017
- First submitted
- 8 Dec 2013
- Last updated
- 17 Oct 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients
- ClinicalTrials.gov ID
- NCT02015481
- Acronym / sponsor ID
- HOPEMD / BBCO-001
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Keywords
- Case control, safety, tolerability, efficacy.
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Bioblast Pharma Ltd.Industry (pharma, biotech, devices)
- Study start
- Feb 2014
- Primary completion
- Apr 2016 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 19 Dec 2013 (estimated)
- Results
- Posted 23 Aug 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cabaletta 30gr. | Experimental | Drug: Cabalettaweekly IV of Cabaletta 30gr. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Males and females
- 18 - 80 years (inclusive) of age
- Clinically and genetically diagnosed as OPMD
- Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
- Patients who provide written informed consent to participate in the study
Key exclusion criteria (13 listed)
- Diabetes mellitus type 1 or 2
- Other major diseases, e.g.: renal failure (creatinine clearance <60ml/min), liver failure and chronic liver diseases (e.g. hepatitis B or C) , HIV carriers, tuberculosis, SLE, rheumatoid polyarthritis, sarcoidosis, collagenosis
- Uncontrolled heart disease , CHF,
- Other neuromuscular diseases
- Other disorders associated with esophageal dysphagia: e.g. severe gastroesophageal reflux (GERD), esophageal stricture due to mechanical or chemical trauma, infection (e.g. esophageal moniliasis), drug-induced dysphagia (e.g. bisphosphonates), esophageal rings and webs, spastic motility disorders of the esophagus.
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 | Drug | Cabaletta 30gr. |
Primary outcome
Safety Lab Evaluations
Time frame: 24 weeks
Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
3 secondary outcomes
Drinking Test Score
Time frame: 24 weeks
Videofluoroscopy (VFS) Score
Time frame: 24 Weeks
SWAL-QOL, Swallowing Quality of Life Questionnaire
Time frame: 28 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Oct 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Yoseph Caraco, M.D principal investigator
Hadassah Medical Organization
Bernard Brais, MD principal investigator
McGill University
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cabaletta in people with Oculopharyngeal Muscular Dystrophy, run by Bioblast Pharma Ltd. at 4 sites in 3 countries.
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 13 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 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Canada, 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?
Yes. Results are posted in the registry (first posted 23 Aug 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Lab Evaluations (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 17 Oct 2017.