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Completed

NCT Number: NCT02015481

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

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

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Key information

Conditions

Oculopharyngeal Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Oculopharyngeal Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Montreal Neurological Institute, McGill University, Montreal, Quebec, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Cabaletta

Drug

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Bioblast Pharma Ltd.

Industry

Registry information

Official study title

Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients

Acronym: HOPEMD

Important dates

Study start
2014
Primary completion
2016
Study completion
2016
First posted
Dec 19, 2013
Registry last updated
Oct 17, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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