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Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose

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.
 
ClinicalTrials.gov Identifier: NCT04226924
Recruitment Status : Withdrawn (Lack of funding for company)
First Posted : January 13, 2020
Last Update Posted : January 13, 2020
Sponsor:
Information provided by (Responsible Party):
Bioblast Pharma Ltd.

Brief Summary:
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.

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

Detailed Description:

After signing informed consent, patients will undergo two rounds of ice-cold water and nectar drinking tests at least 1 week apart to confirm oropharyngeal dysfunction. Patients who have confirmed oropharyngeal dysfunction, i.e., an ice-cold water drinking test time of 8 seconds or greater at both rounds, in addition to an SSQ score of >235, will be enrolled. Baseline values for all safety and efficacy parameters will be established during the screening period. Patients will be randomized in a 1:1 ratio, to trehalose or placebo, at the time of enrollment. Randomization will be stratified according to the patient's score on the SSQ at screening (≤ 799 or ≥ 800).

Patients randomized to trehalose will receive a 1-hour IV infusion of trehalose at a dose of 0.75 g/kg weekly for 24 weeks. Patients randomized to placebo (normal saline) will receive a weight-based equal volume of placebo weekly for 24 weeks.

After Week 24, patients may transition to an open-label extension of the study (extension period). During the extension period, patients will be treated with weekly infusion of trehalose at a dose of 0.75 g/kg for 24 weeks, followed by a 4-week safety follow-up (total duration of study = 56 weeks).

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: The study includes a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: The Treatment Period of the study is double-blind. The Extension Period is open label.
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment of
Actual Study Start Date : June 15, 2017
Estimated Primary Completion Date : February 15, 2018
Estimated Study Completion Date : August 15, 2018


Arm Intervention/treatment
Experimental: Trehalose
Trehalose 9% solution: The dose is 0.75 g/kg administered IV over 60 ± 5 minutes once weekly.
Drug: Trehalose
90 mg/ml trehalose solution for IV infusion

Placebo Comparator: 0.9% Normal Saline
Normal saline: weight-based volume administered IV over 60 ± 5 minutes once weekly.
Drug: Trehalose
90 mg/ml trehalose solution for IV infusion




Primary Outcome Measures :
  1. Drinking Test Time [ Time Frame: 24 weeks ]
    Change from baseline in timed drinking tests with 80 cc of ice-cold water and nectar.


Secondary Outcome Measures :
  1. Muscle Strength Testing [ Time Frame: 24 weeks ]
    Change from baseline in strength tests in selected muscle groups as measured by a handheld dynamometer

  2. Stair Climb Test [ Time Frame: 24 weeks ]
    Change from baseline in functional muscle testing as measured by the Stair Climb test

  3. Timed Up and Go Test [ Time Frame: 24 weeks ]
    Change from baseline in functional muscle testing as measured by the Timed Up and Go (TUG) test

  4. 30-Second Lift Test [ Time Frame: 24 weeks ]
    Change from baseline in functional muscle testing as measured by 30-Second Lift test

  5. EuroQol-5D-5L [ Time Frame: 24 weeks ]
    Change from baseline in health status using the EuroQol-5D-5L Questionnaire

  6. Swallowing Quality of Life [ Time Frame: 24 weeks ]
    Change from baseline in quality of life using modified Swallowing Quality of Life Questionnaire

  7. Sydney Swallow Questionnaire [ Time Frame: 24 weeks ]
    Change from baseline in quality of life using Sydney Swallow Questionnaire



Information from the National Library of Medicine

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Ages Eligible for Study:   50 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Genetically confirmed OPMD with a (GCN)13 size PABPN1 mutation
  • A score greater than 235 on the Sydney Swallow Questionnaire at screening
  • Confirmation of oropharyngeal dysfunction by abnormal ice-cold water drinking test result, defined as drinking 80 cc of ice-cold water in ≥ 8 seconds at both drinking tests (at least 1 week apart) during the screening period

Exclusion Criteria:

  • History of pharyngeal myotomy.
  • Esophageal dilatation within the last 12 months.
  • Treatment with botulinum toxin (any location) within 1 year prior to screening.
  • Diagnosis of any other muscle disorder.
  • Prior head and neck surgery or radiation.
  • Oropharyngeal injury or oropharyngeal cancer.
  • Other esophageal disease that may be the cause of the dysphagia.
  • Previously diagnosed with diabetes or a hemoglobin A1c (HgbA1c) result > 6.0% at screening.
  • Prior treatment with IV trehalose.
  • Known hypersensitivity to trehalose.
  • Non-ambulatory (Use of a cane or short leg braces are permitted).
  • Prior history of stroke (ischemic or hemorrhagic).
  • Pregnancy or breast feeding.
  • History of alcohol or drug abuse within the last 5 years.
  • Evidence of hepatitis B, hepatitis C, or HIV infection at screening.
  • Currently receiving anti-coagulant treatment (e.g., warfarin, enoxaparin) other than anti-platelet treatments, which are not a reason for exclusion.
  • Currently participating in another clinical trial or has completed an interventional trial less than 90 days prior to planned first dosing.

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 ClinicalTrials.gov identifier (NCT number): NCT04226924


Locations
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Canada, Quebec
Ecogene-21
Chicoutimi, Quebec, Canada, G7H 7K9
Montreal Neurological Institute and Hospital
Montréal, Quebec, Canada, H3A 2B4
CHU de Québec-Université Laval- Hôpital Enfant-Jésus
Québec, Quebec, Canada, G1J 1Z4
Sponsors and Collaborators
Bioblast Pharma Ltd.
Investigators
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Principal Investigator: Bernard Brais, MD McGill University
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Responsible Party: Bioblast Pharma Ltd.
ClinicalTrials.gov Identifier: NCT04226924    
Other Study ID Numbers: BB-OPMD-202
First Posted: January 13, 2020    Key Record Dates
Last Update Posted: January 13, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Bioblast Pharma Ltd.:
OPMD
DMOP
Dysphagia
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