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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02015481
Recruitment Status : Completed
First Posted : December 19, 2013
Results First Posted : August 23, 2017
Last Update Posted : October 17, 2017
Information provided by (Responsible Party):
Bioblast Pharma Ltd.

Brief Summary:
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Condition or disease Intervention/treatment Phase
Oculopharyngeal Muscular Dystrophy Drug: Cabaletta Phase 2

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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

Primary Outcome Measures :
  1. 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 .

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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)

Information from the National Library of Medicine

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 identifier (NCT number): NCT02015481

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United States, California
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
Hadassah medical center
Jerusalem, Israel, 91120
Sponsors and Collaborators
Bioblast Pharma Ltd.
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Principal Investigator: Yoseph Caraco, M.D Hadassah Medical Center
Principal Investigator: Bernard Brais, MD McGill University
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Responsible Party: Bioblast Pharma Ltd. 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
Keywords provided by Bioblast Pharma Ltd.:
Case control, safety, tolerability, efficacy.
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Oculopharyngeal
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn