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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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19 April 2022 |
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Main ID: |
EUCTR2014-001753-17-NL |
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Date of registration:
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03/06/2014 |
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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 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biological Activity of ATYR1940 in Adults with Genetic Myopathy
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Scientific title:
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A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies |
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Date of first enrolment:
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19/09/2014 |
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Target sample size:
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44 |
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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:2014-001753-17 |
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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
Number of treatment arms in the trial: 2
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Phase:
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Human pharmacology (Phase I): yes
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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France
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Italy
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Netherlands
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United States
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Contacts
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Name:
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Clinical Trial Operations
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Address:
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3 rue des Longs Prés
92100
Boulogne-Billancourt
France |
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Telephone:
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Email:
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clinicaltrialinformation@voisinconsulting.com |
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Affiliation:
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Voisin Consulting |
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Name:
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Clinical Trial Operations
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Address:
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3 rue des Longs Prés
92100
Boulogne-Billancourt
France |
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Telephone:
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Email:
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clinicaltrialinformation@voisinconsulting.com |
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Affiliation:
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Voisin Consulting |
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Key inclusion & exclusion criteria
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Inclusion criteria:
1. Patient is a male or female aged 18 to 65 years, inclusive.
2. Patient has an established, genetically-confirmed, diagnosis of FSHD with clinical findings meeting existing criteria.
3. Patient has provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.
4. Cohorts =2 only: Patient has imaging findings meeting defined criteria for muscle inflammation in at least 1 skeletal muscle (as per MRI Procedural Manual).
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 44
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion criteria:
1. Patient is currently receiving treatment with an immunomodulatory agent or has a history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4 weeks before Baseline; or high-dose non-steroidal anti-inflammatory agents (NSAIDs) (either chronic or intermittent) within 2 weeks before Baseline.
2. Patient has evidence of an alternative diagnosis other than FSHD, based on prior muscle biopsy or genetic test findings.
3. Patient has a presumptive diagnosis of FSHD, based on clinical assessment, but does not yet have genetic confirmation of the diagnosis.
4. Patient has a history of obstructive or restrictive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence for interstitial lung disease on Screening chest radiograph.
5. Patient has a history of anti-synthetase syndrome, prior Jo-1 antibody (Ab)-positivity, or has a positive or equivocally positive Jo-1 Ab test result during Screening.
6. Patient has symptomatic cardiomyopathy or severe cardiac arrhythmia that may, in the Investigator’s opinion, limit the patient’s ability to complete the study protocol.
7. Patient has evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention or other treatment or may not allow safe participation.
8. Patient has used any investigational product or device (other than a mobility assistance device) within 30 days before Baseline.
9. Patient underwent muscle biopsy within 30 days before Baseline.
10. If female and of childbearing potential (premenopausal and not surgically sterile), patient has a positive pregnancy test at Screening or is unwilling to use contraception.
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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Health Condition(s) or Problem(s) studied
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Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
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Facioscapulohumeral muscular dystrophy
MedDRA version: 17.1
Level: PT
Classification code 10064087
Term: Facioscapulohumeral muscular dystrophy
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Intervention(s)
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Product Name: ATYR1940
Product Code: ATYR1940
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: N/A
Current Sponsor code: ATYR1940
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use
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Primary Outcome(s)
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Timepoint(s) of evaluation of this end point: Cohorts 1 and 2
AEs: visits 1 to 9
Sampling for safety lab tests & vital signs: Screening, visits 1 to 9
ECG: Screening, visits 2, 4, 6, 8 and 9
Pulmonary function tests: Screening, visits 3, 5, 8 and 9
Pulse oximetry: visits 1, 2, 3, 4 and 6
ADA & Jo-1 Ab: Screening, visits 3 to 9
PK: Cohort 1 & Cohort =2 = visits 2 to 7
=Cohort 3
AEs: visits 1 to 16
Sampling for safety lab tests: Screening, visits 1 to 6, 8, 10, 12 and 14 to 16
Vital signs: Screening, visits 1 to 16
ECG: Screening, visits 2, 4, 6, 9, 14 and 15
Pulmonary function tests: Screening, visits 3, 8/9/10 and 15
Pulse oximetry: visits 1 to 13
Jo-1 Ab: Screening, visits 3 to 16
ADA: Screening, visits 3 to 6, 10 and 14 to 16
PK: visits 2 to 6, 10, 13, 14
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Main Objective: Evaluate the safety, tolerability, pharmacokinetics (PK), and immunogenicity of multiple doses of intravenous (IV) ATYR1940 in adults 18 to 65 years of age, inclusive, with FSHD
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Secondary Objective: Explore pharmacodynamic (PD) changes in the following biological parameters:
• FSHD-related inflammatory immune responses in skeletal muscle, as assessed by quantitative magnetic resonance imaging (MRI).
• FSHD-related inflammatory immune state in peripheral blood, as assessed by:
• Circulating immune proteins such as cytokines.
• Ex vivo inflammatory immune protein (including cytokines) release from peripheral blood mononuclear cells (PBMCs).
• Immunophenotyping (general and FSHD-related) of circulating PBMCs.
Explore PD changes in the following clinical parameters:
• Manual muscle testing (MMT), as determined by the Investigator.
• Individualized Neuromuscular Quality of Life (INQoL) instrument, as determined by the patient.
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Primary end point(s): Safety and Tolerability endpoints:
• Change from Baseline of physical examination.
• Incidence of AEs.
• Change from Baseline in safety laboratory test results.
• Change from Baseline in ECG findings.
• Change from Baseline in vital sign measurements and pulmonary evaluations.
• Antibody test results.
• Incidence of infusion reactions and infusion site examination findings.
PK Endpoints:
• Standard PK parameters (Cmax, tmax, t1/2, etc).
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Secondary Outcome(s)
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Timepoint(s) of evaluation of this end point: Cohorts 1 and 2
MRI: Screening/visit 1 and visit 7
INQoL: Screening and visit 7
MMT: Sceening and visits 3, 5 and 7
Serum/plasma for biomarkers: visits 1, 2, 5, 6 and 7
Immunophenotyping & protein release: visits 1, 5 and 7
=Cohort 3
MRI: Screening and visits 6, 14 and 15
INQoL: Screening and visits 6 and 14
MMT: Screening and visits 6, 10 and 14
Serum/plasma for biomarkers: visits 1, 2, 5, 9, 13 to 15
Immunophenotyping and protein release: Screening, visits 1, 5, 14 and 15
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Secondary end point(s): PD Endpoints:
• Changes in FSHD-related inflammatory immune state in peripheral blood and muscle.
• Changes in the clinical parameters.
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Secondary ID(s)
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ATYR1940-C-002
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Source(s) of Monetary Support
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aTyr Pharma, Inc.
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Ethics review
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Status: Approved
Approval date: 19/09/2014
Contact:
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