Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Part… (NCT02836418) | Clinical Trial Compass
CompletedPhase 1/2
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
United States, Denmark, Italy8 participantsStarted 2016-07-13
Plain-language summary
ATYR1940-C-006 is a multi-national, multicenter study being conducted at centers in the United States (US) and Europe who participated in Study ATYR1940-C-003 (Stage 1 only) or Study ATYR1940-C-004 (that is, the parent studies).
Who can participate
Age range
16 Years – 25 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Enrolled in and completed the treatment period in the parent study.
. Demonstrated, in the Sponsor's and Investigator's opinions, acceptable tolerability of ATYR1940.
. In the Investigator's opinion, participant has shown acceptable compliance with ATYR1940 and the study procedures in the parent study and is willing and able to comply with all procedures in the current study.
. Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued ATYR1940 treatment.
. Provide written informed consent or assent after the nature of the study has been explained and prior to the performance of any research-related procedures.
Exclusion criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timeframe: Up to End of Study (up to approximately Week 39)
2
Number of Participants With Positive Anti-Drug Antibodies (ADA)
Timeframe: Up to End of Study (up to approximately Week 39)
3
Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL)
Timeframe: Up to End of Study (up to approximately Week 39)
4
Number of Participants With a Clinical Laboratory Abnormality Leading to an AE
Timeframe: Up to End of Study (up to approximately Week 39)
5
Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE
Timeframe: Up to End of Study (up to approximately Week 39)
6
Number of Participants With a Clinically Significant Electrocardiogram (ECG) Abnormality Leading to a TEAE
Timeframe: Up to End of Study (up to approximately Week 39)
. Is expected to require treatment with curcumin or systemic albuterol (intermittent inhaled albuterol is permissible) during study participation; or use of a product that putatively enhances muscle growth (for example, insulin-like growth factor, growth hormone) or activity (for example, Coenzyme Q, Coenzyme A, creatine, L-carnitine) on a chronic basis; or statin treatment initiation or significant adjustment to statin regimen (stable, chronic statin use is permissible).
. Planned to receive any vaccination during study participation.
. Abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator's opinion, might jeopardize the participant's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
. Evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention, other treatment, or may not allow safe participation.
. If female and of childbearing potential (premenopausal and not surgically sterile), has a positive pregnancy test at entry or is unwilling to use contraception from the time of entry through the 3-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device.
. If male, is unwilling to use a condom plus spermicide during sexual intercourse from the time of entry through the 1 month Follow-up visit.
Number of Participants With Vital Sign Abnormality Resulting in a TEAE
Timeframe: Up to End of Study (up to approximately Week 39)
8
Change From Baseline in Manual Muscle Testing (MMT) Score at Week 12