[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Sarepta Therapeutics, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":160},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,45,71,104,132],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100526745","phase-1-study-of-aro-dm1-in-subjects-with-type-1-myotonic-dystrophy-100526745",false,"NCT06138743","Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy","A Phase 1\u002F2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 (SRP-1003) in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years","Key Inclusion Criteria:\n\n* Genetically confirmed diagnosis of DM1\n* Clinician-assessed signs of DM1 including clinically apparent myotonia\n* Onset of DM1 symptoms occurred after the age of 12 years\n* Walk for at least 10 meters independently at screening\n* Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later.\n\nKey Exclusion Criteria:\n\n* Inadequately controlled diabetes\n* Confirmed diagnosis of congenital DM1\n* Uncontrolled hypertension\n* History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period\n* Clinically significant cardiac, liver or renal disease\n* Human immunodeficiency virus infection (seropositive) at screening\n* Seropositive for hepatitis B or hepatitis C at screening\n* Untreated or poorly controlled epilepsy\n* Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to screening.\n* Abnormal coagulation parameters at screening including platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol","ALL","18 Years","65 Years",{"count":20,"type":21},78,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This is a phase 1\u002F2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.",[28],"Myotonic Dystrophy 1",[28,30,31],"SRP-1003","ARO-DM1","RECRUITING","2026-08-07",{"date":35,"type":36},"2026-08-11","ACTUAL",{"date":38,"type":36},"2024-03-04",{"date":40,"type":21},"2026-12-31",{"name":42,"class":43},"Sarepta Therapeutics, Inc.","INDUSTRY",35,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":16,"minAge":52,"maxAge":53,"enrollmentInfo":54,"targetDuration":4,"studyType":22,"phases":56,"briefSummary":57,"conditions":58,"keywords":60,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":64,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":70},"100526226","phase-1-study-of-aro-dux4-in-adult-and-adolescent-patients-with-facioscapulohumeral-muscular-dystrophy-type-1-100526226","NCT06131983","Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1","A Phase 1\u002F2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 (SRP-1001) in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1","Key Inclusion Criteria:\n\n* Genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record\n* Clinical severity score between 3 and 8 (scale, 0 to 10)\n* Must have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader, with muscle fat fraction ≥10% and less than approximately 40%\n* Males or nonpregnant, nonlactating females ≥18 years of age who do not plan to become pregnant during the study, with an upper age limit of ≤70 years\n* Able and willing to provide written informed consent prior to the performance of any study specific procedures\n* Participants with a body mass index (BMI) between 18.0 and 35.0 kilograms\u002Fsquare meter, inclusive. A participant with FSHD1 and a BMI outside this range may be allowed into the study at the discretion of the principal investigator.\n* Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader\n* A 12-lead electrocardiogram at screening with no abnormalities that may compromise participant's safety in the study\n* Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 9 months following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 9 months following the end of study or last dose of study medication, whichever is later.\n\nKey Exclusion Criteria:\n\n* Human immunodeficiency virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at screening\n* Seropositive for hepatitis B or hepatitis C at screening\n* Uncontrolled hypertension\n* Severe cardiovascular disease\n* History of thrombolic events\n* Platelet count less that the lower limit of normal at screening\n* History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.\n* Any contraindication to muscle biopsy or MRI\n\nNote: additional inclusion\u002Fexclusion criteria may apply per protocol","16 Years","70 Years",{"count":55,"type":21},60,[24,25],"The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose magnetic imaging resonance (MRI)-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.",[59],"Muscular Dystrophy, Facioscapulohumeral",[61,62,63],"Facio-Scapulo-Humeral Muscular Dystrophy","SRP-1001","ARO-DUX4",{"date":35,"type":36},{"date":66,"type":36},"2024-06-19",{"date":68,"type":21},"2028-12-31",{"name":42,"class":43},16,{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":76,"acronym":77,"eligibilityCriteria":78,"healthyVolunteers":11,"sex":79,"minAge":80,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":22,"phases":83,"briefSummary":84,"conditions":85,"keywords":87,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":95,"lastUpdatePostDateStruct":96,"startDateStruct":98,"completionDateStruct":100,"leadSponsor":102,"locationsCount":103},"100410604","phase-1-a-gene-transfer-therapy-study-to-evaluate-the-safety-of-and-expression-from-delandistrogene-moxeparvovec-srp-9001-in-participants-with-duchenne-muscular-dystrophy-dmd---non-ambulatory-cohort-100410604","NCT04626674","A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort","An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)","ENDEAVOR","Inclusion Criteria:\n\n* For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.\n* Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.\n* Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.\n* Cohort 1: Is ambulatory, and ≥4 to \\\u003C8 years of age at the time of Screening.\n* Cohort 2: Is ambulatory, and ≥8 to \\\u003C18 years of age at the time of Screening.\n* Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.\n* Cohort 4: Is ambulatory and ≥3 to \\\u003C4 years of age at the time of Screening.\n* Cohort 5a: Is ambulatory and ≥4 to \\\u003C9 years of age with time to rise from the floor ≤7 seconds at the screening visit.\n* Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.\n* Cohort 6: Is ambulatory, and ≥2 to \\\u003C3 years of age at the time of Screening.\n* Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.\n* Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.\n* Genetic mutation inclusion criteria vary by cohort.\n\nAll Cohorts:\n\n* Ability to cooperate with motor assessment testing.\n* rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.\n\nExclusion Criteria:\n\n* Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.\n* Has a concomitant illness, autoimmune disease, chronic drug treatment, and\u002For cognitive delay\u002Fimpairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.\n* Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.\n* Abnormality in protocol-specified diagnostic evaluations or laboratory tests.\n\nNote: Other inclusion\u002Fexclusion criteria apply.","MALE","2 Years",{"count":82,"type":21},83,[24],"Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.\n\nThis is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.",[86],"Duchenne Muscular Dystrophy",[86,88,89,90,91,92,93,94],"Gene-Delivery","Gene Therapy","DMD","Ambulatory Non-ambulatory","Pediatric","Adult","Dystrophin","2026-06-19",{"date":97,"type":36},"2026-06-24",{"date":99,"type":36},"2020-11-23",{"date":101,"type":21},"2028-02-29",{"name":42,"class":43},7,{"id":105,"slug":106,"hasResults":11,"nctId":107,"briefTitle":108,"officialTitle":109,"acronym":4,"eligibilityCriteria":110,"healthyVolunteers":11,"sex":16,"minAge":111,"maxAge":53,"enrollmentInfo":112,"targetDuration":4,"studyType":22,"phases":114,"briefSummary":115,"conditions":116,"keywords":118,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":123,"lastUpdatePostDateStruct":124,"startDateStruct":126,"completionDateStruct":128,"leadSponsor":130,"locationsCount":131},"100634159","phase-1-a-first-in-human-study-of-the-effects-of-srp-1005-in-participants-with-huntingtons-disease-100634159","NCT07536061","A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease","A Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study to Evaluate the Effect of SRP-1005 in Subjects With Huntington's Disease (INSIGHTT)","Key Inclusion Criteria:\n\n* Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine repeat length ≥40.\n* Participant has HD Integrated Staging System (HD-ISS) Stage 2 or Mild Stage 3. At screening, the participant must be classified with the Enroll-HD HD-ISS Modified Calculator as either Stage 2 or Mild Stage 3.\n* Participants who are of childbearing potential, or with partners of childbearing potential, who are sexually active must agree to use a highly effective method of contraception throughout study participation, and for at least 90 days following the end of study.\n\nKey Exclusion Criteria:\n\n* Any condition that would compromise the safety or feasibility of lumbar puncture or magnetic resonance imaging (MRI).\n* Presence of other significant neurological or systemic illnesses.\n* Current, chronic or active human immunodeficiency virus, hepatitis B\u002FC.\n* Recent use of investigational agents or HTT-lowering therapies.\n* Uncontrolled psychiatric illness or substance use disorders.\n* Pregnancy or lactation.\n\nNote: Other inclusion\u002Fexclusion criteria apply.","21 Years",{"count":113,"type":21},32,[24],"This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).",[117],"Huntington's Disease",[119,120,121,122],"Huntington's disease","SRP-1005","Dose Escalation","First in Human","2026-06-03",{"date":125,"type":36},"2026-06-04",{"date":127,"type":36},"2026-05-12",{"date":129,"type":21},"2027-11-30",{"name":42,"class":43},2,{"id":133,"slug":134,"hasResults":11,"nctId":135,"briefTitle":136,"officialTitle":137,"acronym":138,"eligibilityCriteria":139,"healthyVolunteers":11,"sex":79,"minAge":140,"maxAge":4,"enrollmentInfo":141,"targetDuration":4,"studyType":22,"phases":143,"briefSummary":145,"conditions":146,"keywords":147,"overallStatus":151,"whyStopped":4,"lastUpdateSubmitDate":152,"lastUpdatePostDateStruct":153,"startDateStruct":155,"completionDateStruct":157,"leadSponsor":159,"locationsCount":4},"100634640","phase-4-study-to-evaluate-the-safety-and-effectiveness-of-elevidys-in-participants-with-duchenne-muscular-dystrophy-treated-in-a-post-marketing-setting-100634640","NCT07542314","Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting","Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)","ENHANCE","Key Inclusion Criteria:\n\n1. Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of dosing.\n2. Cohort 1 only: Is eligible for commercial ELEVIDYS.\n3. Cohort 2 only: Is male at birth and has previously received ELEVIDYS in a commercial setting after pre-treatment with sirolimus and corticosteroids.\n4. Cohort 1 only: Participants who are sexually active must agree to use, for the entire duration of the study, a condom and the female sexual partner must also use a medically acceptable form of birth control (eg, oral contraceptive).\n5. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all other protocol requirements, or is ≥ 18 years of age and personally able to understand and comply with the protocol requirements.\n6. Either has a parent or legal guardian who is willing to provide informed consent, or is ≥ 18 years of age and able to provide informed consent independently.\n\nKey Exclusion Criteria:\n\n1. Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package Insert (USPI).\n2. Cohort 1 only: Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.\n3. Has a medical condition or confounding circumstances (eg, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:\n\n   1. The participant's ability to comply with the protocol-required procedures, and\u002For\n   2. The participant's well-being or safety, and\u002For\n   3. The clinical interpretability of the data collected from the participant\n4. Cohort 1 only: Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.\n5. Cohort 1 only: Has received a live virus vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit or expects to receive a vaccination during the first 3 months after Day 1.\n6. Cohort 1 only: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.\n7. Cohort 1 only: Any wounds or recent injuries that, in the opinion of the Investigator, would be at risk of dehiscence or impaired healing in the setting of sirolimus administration.\n\nOther inclusion\u002Fexclusion criteria may apply, per protocol.","4 Years",{"count":142,"type":21},20,[144],"PHASE4","The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.",[86],[86,90,148,149,150],"ELEVIDYS","Delandistrogene moxeparvovec-rokl","SRP-9001","NOT_YET_RECRUITING","2026-05-22",{"date":154,"type":36},"2026-05-26",{"date":156,"type":21},"2026-07-31",{"date":158,"type":21},"2027-03-31",{"name":42,"class":43},""]