Clinical trial NCT02603562: Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
Official title: An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Facioscapulohumeral Muscular Dystrophy (FSHD)
Keywords: FSHD
Lead sponsor: aTyr Pharma, Inc.
Sponsor class: INDUSTRY
First posted: 2015-11-13
Enrollment: 8
Sex: ALL
Minimum age (years): 16
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with early onset FSHD.

Detailed description: A Phase 1b/2 open-label, intraparticipant dose-escalation study aiming to evaluate the safety, tolerability, immunogenicity, biological and pharmacodynamic activity of intravenous ATYR1940, administered once weekly for 12 weeks, in early onset FSHD participants with signs or symptoms prior to 10 years of age.

Eligibility criteria: Inclusion Criteria:

* Established, genetically confirmed diagnosis of FSHD.
* Onset of FSHD signs or symptoms prior to 10 years of age, as documented in the participant's medical record or based on participant or family report.
* Provide written informed consent or assent
* In the Investigator's opinion, participant is willing and able to complete all study procedures and comply with the weekly study visit schedule.

Exclusion Criteria:

* Currently receiving treatment with an immunomodulatory agent including targeted biological therapies within the 3 months before baseline; corticosteroids within 3 months before baseline; or high-dose non-steroidal anti-inflammatory agents within 2 weeks before baseline.
* Currently receiving curcumin or albuterol; use of a product that putatively enhances muscle growth or activity on a chronic basis within 4 weeks before baseline; statin treatment initiation or significant adjustment to statin regimen within 3 months before baseline (stable, chronic statin use is permissible).
* Use of an investigational product or device within 30 days before baseline.
* Evidence of an alternative diagnosis other than FSHD or a coexisting myopathy or dystrophy, based on prior muscle biopsy or other available investigations.
* History of severe restrictive or obstructive lung disease, or evidence for interstitial lung disease on screening chest radiograph.
* History of anti-synthetase syndrome, prior Jo-1 Ab-positivity, or a positive or equivocally positive Jo-1 Ab test result during screening.
* Chronic infection, such as hepatitis B, hepatitis C, or human immunodeficiency virus or a history of tuberculosis.
* Vaccination within 8 weeks before baseline or vaccination is planned during study participation.
* Symptomatic cardiomyopathy or severe cardiac arrhythmia, that may, in the Investigator's opinion, limit the participant's ability to complete the study protocol.
* Muscle biopsy within 30 days before baseline.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
timeFrame: Up to End of Study (up to Week 25)
measure: Number of Participants With a Clinical Laboratory Abnormality Leading to an AE
timeFrame: Up to End of Study (Up to Week 25)
measure: Number of Participants With an Ocular Abnormality Leading to a TEAE
timeFrame: Up to End of Study (Up to Week 25)
measure: Number of Participants With an Impact on Hearing From ATYR1940 Treatment
timeFrame: Up to End of Study (Up to Week 25)
measure: Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE
timeFrame: Up to End of Study (up to Week 25)
Secondary outcomes:
measure: Number of Participants With Positive Anti-Drug Antibodies (ADA)
timeFrame: Baseline up to Week 12
measure: Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL)
timeFrame: Baseline up to Week 12
measure: Number of Participants With Infusion-Related Reactions
timeFrame: Baseline up to Week 12
measure: Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 14
timeFrame: Baseline, Week 14
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University
state: California
city: Iowa City
country: United States
facility: University of Iowa Children's Hospital
state: Iowa
city: Columbus
country: United States
facility: OSU Wexner Medical Center
state: Ohio
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Marseille
country: France
facility: Centre d'nvestigation Clinique - Centre de Pharmacologie Clinique et d'Evaluations Thérapeutiques (CICCPCET)
city: Paris
country: France
facility: Institut de Myologie
city: Milano
country: Italy
facility: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Study officials:
References: