Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients (HOPEMD)
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ClinicalTrials.gov Identifier: NCT02015481 |
Recruitment Status :
Completed
First Posted : December 19, 2013
Results First Posted : August 23, 2017
Last Update Posted : October 17, 2017
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Oculopharyngeal Muscular Dystrophy | Drug: Cabaletta | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 25 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients |
Study Start Date : | February 2014 |
Actual Primary Completion Date : | April 2016 |
Actual Study Completion Date : | April 2016 |

Arm | Intervention/treatment |
---|---|
Experimental: Cabaletta 30gr.
weekly IV of Cabaletta 30gr.
|
Drug: Cabaletta |
- 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 .
- Drinking Test Score [ Time Frame: 24 weeks ]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.
- Videofluoroscopy (VFS) Score [ Time Frame: 24 Weeks ]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.
- SWAL-QOL, Swallowing Quality of Life Questionnaire [ Time Frame: 28 weeks ]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.

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Ages Eligible for Study: | 18 Years to 80 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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)
- 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)

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02015481
United States, California | |
UCLA | |
Los Angeles, California, United States, 90095 | |
Tahseen Mozaffar | |
Orange, California, United States, 92868 | |
Canada, Quebec | |
Montreal Neurological Institute, McGill University | |
Montreal, Quebec, Canada, H3A 2B4 | |
Israel | |
Hadassah medical center | |
Jerusalem, Israel, 91120 |
Principal Investigator: | Yoseph Caraco, M.D | Hadassah Medical Center | |
Principal Investigator: | Bernard Brais, MD | McGill University |
Responsible Party: | Bioblast Pharma Ltd. |
ClinicalTrials.gov Identifier: | NCT02015481 |
Other Study ID Numbers: |
BBCO-001 |
First Posted: | December 19, 2013 Key Record Dates |
Results First Posted: | August 23, 2017 |
Last Update Posted: | October 17, 2017 |
Last Verified: | September 2017 |
Case control, safety, tolerability, efficacy. |
Muscular Dystrophies Muscular Dystrophy, Oculopharyngeal Muscular Disorders, Atrophic Muscular Diseases |
Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn |