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. |
Register:
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EUCTR |
Last refreshed on:
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20 August 2018 |
Main ID: |
EUCTR2017-004139-35-BE |
Date of registration:
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03/04/2018 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Clinical Trial in Male Subjects with Muscular Disease
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Scientific title:
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A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy |
Date of first enrolment:
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18/06/2018 |
Target sample size:
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132 |
Recruitment status: |
Authorised-recruitment may be ongoing or finished |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-004139-35 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 4
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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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Argentina
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Belgium
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Canada
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Chile
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Germany
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Israel
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Italy
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Netherlands
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Serbia
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Spain
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Turkey
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trial Lead
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Address:
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1425 Route 206
NJ 07921
Bedminster
United States |
Telephone:
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+19082385657 |
Email:
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Bryan.Due@mallinckrodt.com |
Affiliation:
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Mallinckrodt ARD Inc. |
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Name:
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Clinical Trial Lead
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Address:
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1425 Route 206
NJ 07921
Bedminster
United States |
Telephone:
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+19082385657 |
Email:
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Bryan.Due@mallinckrodt.com |
Affiliation:
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Mallinckrodt ARD Inc. |
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Key inclusion & exclusion criteria
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Inclusion criteria: Subjects must meet all of the following criteria for inclusion in the study at the Screening Visit and at the Baseline Visit (except as noted):
1. Each subject’s parent or legal guardian must be adequately informed and understand the nature and risks of the study and must be able to provide a signature and date on the ICF. In addition, each subject who is capable of understanding it must sign an assent form. The
consent and assent (where applicable) for this study must be signed before any protocol required procedures are performed.
2. Subjects must be male and between 4 and 8 years of age (inclusive) at the Screening Visit.
3. Subjects must have a documented diagnosis of DMD confirmed by complete dystrophin deficiency (by immunofluorescence and/or immunoblot), or identifiable mutation in the DMD gene where reading frame can be predicated as “out of frame”, or complete
dystrophin gene sequencing consistent with DMD; AND in the opinion of the Investigator, a typical clinical profile consistent with DMD.
4. Subjects taking approved treatments for DMD (by a Health Authority) that target dystrophin gene mutations (eg, eteplirsen or ataluren) may be enrolled in the study if they have been on a stable dose for 30 days prior to the first dose of study drug, and plan to
remain on that dose throughout the study
5. Subjects should have serum potassium within the reference range at screening. Subjects who have potassium levels below the lower limit of normal may be given potassium supplements and then retested within the 28 day screening period.
6. Subjects and/or subjects’ parent or legal guardian must be able to communicate effectively with study personnel.
7. Subjects and subjects’ parent or legal guardian must be able and willing to follow all protocol requirements and study restrictions.
8. Subjects and subjects’ parent or legal guardian must be able and willing to return for all study visits. Are the trial subjects under 18? yes Number of subjects for this age range: 132 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria: Subjects are ineligible for study participation if they meet any of the following criteria at the Screening Visit and at the Baseline Visit (except as note):
1. Subject is from a vulnerable population (excluding pediatric populations), as defined by the US CFR Title 45, Part 46, Section 46.111(b) and other local and national regulations, including but not limited to, employees (temporary, part-time, full-time, etc) or family members of the research staff conducting the study, or of the sponsor, or of the clinical research organization, or of the IRB/IEC.
2. Subject has had previous systemic treatment with corticosteroids. Transient previous use of corticosteroids will be evaluated on a case-by-case basis by the sponsor or designee. Inhaled corticosteroids will be permitted if given at a stable dose for the 3 months prior to the first dose of study drug and the subject will remain on that dose throughout the study. The use of topical or intra-articular corticosteroids is permitted during the study.
3. Subject has been treated with immunosuppressants, or mineralocorticoids (including, but not limited to, spironolactone, eplerone, and carrenone) in the 3 months prior to the Screening Visit.
4. Subject has taken any investigation medication within 30 days or 5 half lifes (whichever is longer) prior to the first dose of study drug.
5. Subject is unwilling to receive, or is intolerant of, SC injections.
6. Subject has symptomatic cardiomyopathy in the opinion of the investigator.
7. Subject is unable to complete the 10 meter Walk/Run test at the Screening and/or Baseline Visit.
8. Subject has a history of any drug allergy, hypersensitivity, or intolerance to cosyntropin, H.P. Acthar® Gel or other ACTH products.
9. Subject has a history or evidence of active infection or febrile illness within 7 days prior to the Screening Visit or subject has a clinically significant infection requiring administration of intravenous antibiotics or hospitalization in the 4 weeks prior to the Screening Visit or between the Screening Visit and the first dose of study drug.
10. Subject has any known contraindication(s) to the class of ACTH products including, but not limited to:
Asthma.
Acute psychoses.
Any primary adrenocortical insufficiency or adrenal cortical hyperfunction.
Cushing’s Syndrome.
Any current congestive heart failure (defined as New York Heart Association Functional Class III to IV).
Peptic ulcer (within 24 weeks prior to the Screening Visit).
Recent major surgery (within 24 weeks prior to the Screening Visit).
11. Subject has Type 1 or Type 2 diabetes mellitus.
12. Subject has a history of chronic active hepatitis including acute or chronic hepatitis B, or acute or chronic hepatitis C.
13. Subject has a history of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
14. Subject has known immune compromised status (not related to disease/condition under study), including but not limited to, individuals who have undergone organ transplantation or who are known to be positive for the human immunodeficiency virus.
15. Subject has any solid tumor or hematologic malignancy currently diagnosed or undergoing therapy, or has received therapy for any solid tumor or hematological malignancy in the 5 years prior to the Screening Visit; with the exception of treated and cured basal cell
carcinoma or treated and cured squamous cell carcinoma of the skin.
16. Subjec
Age minimum:
Age maximum:
Gender:
Female: no Male: yes
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Distrophy MedDRA version: 20.0
Level: PT
Classification code 10013801
Term: Duchenne muscular dystrophy
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
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Intervention(s)
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Product Name: Cosyntropin acetate Product Code: MNK-1411 Pharmaceutical Form: Suspension for injection INN or Proposed INN: TETRACOSACTIDE HEXAACETATE CAS Number: 22633-88-1 Other descriptive name: Cosyntropin acetate Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 1- Pharmaceutical form of the placebo: Suspension for injection Route of administration of the placebo: Subcutaneous use
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Primary Outcome(s)
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Secondary Objective: 1-To assess the effect of MNK-1411 on motor skills and strength. 2-To determine the safety and tolerability of MNK-1411 in subjects with DMD.
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Timepoint(s) of evaluation of this end point: Week 24
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Main Objective: To determine the effect of MNK-1411 on motor function in subjects with DMD.
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Primary end point(s): Change from baseline in 10 meter walk/run time
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Secondary Outcome(s)
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Secondary end point(s): 1-Change from baseline in quantitative muscle testing, the 4 Stair Climb, Time to Stand, and the North Star Ambulatory Assessment (NSAA)
2-Summary of general safety profile, including AEs, vital signs, immunogenicity, and laboratory assessments over the entire study.
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Timepoint(s) of evaluation of this end point: 1-Week 24
2-Over the entire study
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Secondary ID(s)
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2017-004139-35-ES
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MNK14112096
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Source(s) of Monetary Support
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Mallinckrodt ARD Inc.
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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