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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Bioblast Pharma Ltd.
ClinicalTrials.gov Identifier:
NCT02015481
First received: December 8, 2013
Last updated: April 5, 2016
Last verified: March 2015
  Purpose
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Condition Intervention Phase
Oculopharyngeal Muscular Dystrophy Drug: Cabaletta Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients

Resource links provided by NLM:


Further study details as provided by Bioblast Pharma Ltd.:

Primary Outcome Measures:
  • vital signs [ Time Frame: 28 weeks ]
  • safety lab tests [ Time Frame: 28 weeks ]
  • Adverse Events [ Time Frame: 28 weeks ]

Secondary Outcome Measures:
  • Changes in disease markers [ Time Frame: 28 weeks ]

Enrollment: 25
Study Start Date: February 2014
Study Completion Date: April 2016
Primary Completion Date: April 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cabaletta 30gr.
weekly IV of Cabaletta 30gr.
Drug: Cabaletta

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02015481

Locations
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
Sponsors and Collaborators
Bioblast Pharma Ltd.
Investigators
Principal Investigator: Yoseph Caraco, M.D Hadassah Medical Center
Principal Investigator: Bernard Brais, MD Montreal Neurological Institute, McGill University
  More Information

Responsible Party: Bioblast Pharma Ltd.
ClinicalTrials.gov Identifier: NCT02015481     History of Changes
Other Study ID Numbers: BBCO-001
Study First Received: December 8, 2013
Last Updated: April 5, 2016

Keywords provided by Bioblast Pharma Ltd.:
Case control, safety, tolerability, efficacy.

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Oculopharyngeal
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 25, 2017