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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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EUCTR |
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Last refreshed on:
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25 November 2019 |
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Main ID: |
EUCTR2004-000622-67-GB |
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Date of registration:
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10/02/2005 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Double-Blind, Placebo-Controlled, Randomized, Multiple Ascending Dose, Safety Study of MYO-029 Administered to Adult Patients with Becker, Facioscapulohumeral and Limb-Girdle Muscular Dystrophy.
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Scientific title:
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A Double-Blind, Placebo-Controlled, Randomized, Multiple Ascending Dose, Safety Study of MYO-029 Administered to Adult Patients with Becker, Facioscapulohumeral and Limb-Girdle Muscular Dystrophy. |
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Date of first enrolment:
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03/06/2005 |
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Target sample size:
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136 |
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Recruitment status: |
Not Recruiting |
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URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-000622-67 |
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Study type:
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Interventional clinical trial of medicinal product |
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Study design:
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Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: yes
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: no
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
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Countries of recruitment
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United Kingdom
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Contacts
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Key inclusion & exclusion criteria
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Inclusion criteria:
1. Age >=18 years.
2. Confirmed clinical and molecular diagnosis of BMD, FSHD, or LGMD 2A, 2B, 2C, 2D, 2E, or 2I using the specific diagnostic criteria listed in Section 19, Muscular Dystrophy History
3. Independent ambulation as assessed by completion of a 9-meter walking test. The use of an ankle foot orthosis is permitted if needed.
4. Screening period- first strength evaluation: at least 8 of the following 16 muscle groups must have a MMT muscle grade >= 3- and <= 4+ on the modified Medical Research Council Scale:
Shoulder flexors, Shoulder abductors, Shoulder external rotators, Shoulder internal rotators, Biceps, Elbow extensors, Brachioradialis (all bilateral), Neck Flexors, Neck Extensors.
5. Screening period- second strength evaluation: at least 10 of the following 16 muscle groups must have an MMT grade that is within 2 steps of the grade assigned at the first screening evaluation:
Shoulder flexors, Shoulder abductors, Shoulder external rotators, Shoulder internal rotators, Biceps, Elbow extensors, Brachioradialis (all bilateral), Neck flexors, Neck Extensors.
6. Forced vital capacity (FVC) >= 60% of predicted (performed while seated) at Visit 1
7. Ejection fraction > 40% as measured by echocardiogram at Visit 1.
8. Male subjects who are not surgically sterile agree and commit to the use of 2 reliable methods of birth control for the duration of the study and for 12 weeks after the last dose of test article.
9. Female subjects who are surgically sterile or postmenopausal.
10. Able to comply with all study evaluations and return for all study visits.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Exclusion criteria:
1. Known history of congestive heart failure.
2. Diagnosis of atherosclerotic heart disease.
3. Use of anti-arrhythmic treatment during the 12 weeks before randomization.
4. Use of chronic anti-coagulant treatment during the 12 weeks before randomization.
5. Use of steroids such as, but not limited to, prednisone, prednisolone, deflazacort, and oxandrolone, during the 6 months before randomization and for the duration of the study with the exception of topical, inhaled and/or intranasal steroids used for a condition other than muscular dystrophy.
6. Use of any other pharmacologic treatment with the potential to affect muscle function such as, but not limited to, albuterol and creatine during the 4 weeks before randomization and for the duration of the study.
7. Initiation of, or significant change in, endurance and/or strengthening exercises with the potential to affect muscle function during the 8 weeks before randomization and for the duration of the study.
8. Surgery performed during the 12 weeks before randomization or planned for the duration of the study.
9. Concomitant co-morbidity that functionally limits the ability to perform required study procedures (eg, MMT, MRI, etc.).
10. Any contraindication to MRI per site standard of care, including the presence of any metal implants.
11. Significant concomitant intercurrent illness.
12. History of sensitivity to monoclonal antibodies or protein pharmaceuticals.
13. Lactating women.
14. Women of childbearing potential
15. Use of any experimental treatments or participation in a clinical trial during the 4 weeks before randomization and for the duration of the study.
16. Any other major illness or reason that, in the investigator's judgment, will substantially increase the risk associated with the subject's participation in this study.
17. Diagnosis of LGMD other than LGMD 2A, 2B, 2C, 2D, 2E, and 2I.
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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Health Condition(s) or Problem(s) studied
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Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy.
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Intervention(s)
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Product Name: MYO-029
Pharmaceutical Form: Powder for injection*
INN or Proposed INN: Not Available
Current Sponsor code: MYO-029
Other descriptive name: Recombinant human anti-GDF-8 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 70-
Pharmaceutical form of the placebo: Powder for injection*
Route of administration of the placebo: Intravenous use
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Primary Outcome(s)
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Main Objective: Primary objective is to evaluate safety of MYO-029 in adult subjects with muscular dystrophy.
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Secondary Objective: Secondary objectives are to:
1) evaluate the biological activity of MYO-029 by using the following measurements: muscle mass, volume, strength; histopathology findings; patient-reported outcomes; and functional measurements.
2) explore associations between gene expression patterns, test article administration, and clinical parameters.
3) evaluate the pharmacokinetics of MYO-029 in adult subjects with muscular dystrophy.
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Primary end point(s): NA
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Secondary ID(s)
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3147K2-101WW
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Source(s) of Monetary Support
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Ethics review
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Status: Approved
Approval date:
Contact:
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