BridgeMD
Phase 2 Randomized, Double-blind, Placebo-controlled Study of the Effect of EDG-5506 on Biomarkers in Adults with Neuromuscular Disease
EU CTIS ID: 2022-500215-39-00
What this study is testing
To assess the safety of sevasemten in adults with neuromuscular disease. To assess the effect of sevasemten treatment on biomarkers of muscle damage in adults with Becker Muscular Dystrophy (BMD), McArdle disease, or Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I).
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Participants with BMD or LGMD2I must be 18 to 65 years of age inclusive, at the time of signing the informed consent. Participants with McArdle disease may enroll up to 75 years of age inclusive, at the time of signing the informed consent form.
- 2. Participant has a molecular diagnosis of BMD (a documented dystrophin mutation and phenotype consistent with BMD, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids), McArdle disease, or LGMD2I with a consistent clinical phenotype.
- 3. Participant is able to comply with the cycle exercise protocol (in the opinion of the Principal Investigator).
- 4. Participant has no concurrent medical condition that would, in the opinion of the Investigator, impair the ability to give informed consent or undertake any of the protocol-mandated visits/assessments.
- 5. Participant has no active muscle injury at Screening.
- 6. Participant is willing and able to comply with all protocol requirements.
You likely can't join if
- 1. Medical history, clinically significant laboratory, or physical examination finding that, in the opinion of the Investigator, would render the participant unsuitable for the study. This includes anything that would prevent completion of the cycle ergometer test.
- 6. Moderate or severe renal impairment (estimated glomerular filtration rate [eGFR] <60 mL/min/1.73 m2 ). eGFR will be based on Cystatin C formula (eGFR = 133 x min(Scys/0.8, 1)^-0.499 x max (Scys/0.8, 1)^-1.328 X 0.996^Age x 0.932 [if female].
- 7. Positive test for hepatitis C antibody (unless negative hepatitis C virus polymerase chain reaction), hepatitis B surface antigen, or human immunodeficiency virus antibody.
- 8. Receipt of oral corticosteroids for >10 days in the previous 6 months. Inhaled/intranasal steroids are permitted.
- 09. Current use of any strong or moderate cytochrome P450 (CYP)3A4 inhibitors or inducers.
- 10. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior, recent (within the past year) history of substance abuse or dependency or laboratory results or abnormality that may increase the risk of study participation or, in the Investigator’s judgment, make the participant inappropriate for the study.
See the full eligibility criteria
Who can join
- 1. Participants with BMD or LGMD2I must be 18 to 65 years of age inclusive, at the time of signing the informed consent. Participants with McArdle disease may enroll up to 75 years of age inclusive, at the time of signing the informed consent form.
- 2. Participant has a molecular diagnosis of BMD (a documented dystrophin mutation and phenotype consistent with BMD, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids), McArdle disease, or LGMD2I with a consistent clinical phenotype.
- 3. Participant is able to comply with the cycle exercise protocol (in the opinion of the Principal Investigator).
- 4. Participant has no concurrent medical condition that would, in the opinion of the Investigator, impair the ability to give informed consent or undertake any of the protocol-mandated visits/assessments.
- 5. Participant has no active muscle injury at Screening.
- 6. Participant is willing and able to comply with all protocol requirements.
- 7. Participant is able to converse and read fluently in Danish or English.
- 8. Participant is willing to comply with contraception requirements described in protocol.
- 9. Capable of giving signed informed consent as described in the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
What rules you out
- 1. Medical history, clinically significant laboratory, or physical examination finding that, in the opinion of the Investigator, would render the participant unsuitable for the study. This includes anything that would prevent completion of the cycle ergometer test.
- 6. Moderate or severe renal impairment (estimated glomerular filtration rate [eGFR] <60 mL/min/1.73 m2 ). eGFR will be based on Cystatin C formula (eGFR = 133 x min(Scys/0.8, 1)^-0.499 x max (Scys/0.8, 1)^-1.328 X 0.996^Age x 0.932 [if female].
- 7. Positive test for hepatitis C antibody (unless negative hepatitis C virus polymerase chain reaction), hepatitis B surface antigen, or human immunodeficiency virus antibody.
- 8. Receipt of oral corticosteroids for >10 days in the previous 6 months. Inhaled/intranasal steroids are permitted.
- 09. Current use of any strong or moderate cytochrome P450 (CYP)3A4 inhibitors or inducers.
- 10. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior, recent (within the past year) history of substance abuse or dependency or laboratory results or abnormality that may increase the risk of study participation or, in the Investigator’s judgment, make the participant inappropriate for the study.
- 11. Participation in a previous exercise challenge study if the challenge itself has taken place less than 10 weeks prior to screening.
- 12. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
- 2. Left ventricular ejection fraction <45% or New York Heart Association (NYHA) Class III or Class IV, determined by local read.
- 3. 12-lead electrocardiogram (ECG) demonstrating clinically relevant abnormalities that may affect participant safety or interpretation of study results, determined by local read.
- 4. Forced vital capacity (FVC) predicted <60% or using daytime (mechanical or noninvasive) ventilatory support.
- 5. Moderate or severe hepatic impairment
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.