Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02015481
• Date of registration: 08/12/2013
• Prospective Registration: Yes
• Primary sponsor: Bioblast Pharma Ltd.
• Public title: Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients HOPEMD
• Scientific title: Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients
• Date of first enrolment: February 2014
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02015481
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Canada, Israel, United States

Contacts

• Name: Bernard Brais, MD
• Affiliation: Montreal Neurological Institute, McGill University

• Name: Yoseph Caraco, M.D
• Affiliation: Hadassah Medical Center

Key inclusion & exclusion criteria

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)



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Oculopharyngeal Muscular Dystrophy

Intervention(s)

Drug: Cabaletta

Primary Outcome(s)

Safety Lab Evaluations [Time Frame: 24 weeks]

Secondary Outcome(s)

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]

Secondary ID(s)

BBCO-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 23/08/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02015481