EUCTR2020-002372-13-SE招募中1 期
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION) - ENVISIO
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 148
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- Male
入选标准
- •A subject must meet the following criteria to be eligible to participate in this study:
- •1. Is male at birth and has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. Genetic report must describe a frameshift deletion, frameshift duplication, premature stop (nonsense”), canonical splice mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 79 (inclusive) that is expected to lead to complete absence of dystrophin protein.
- •Mutations fully or partially contained within exons 1-17 (inclusive) are not eligible.
- •2. Cohort 1 only (non-ambulatory):
- •a. Has been non-ambulatory for a minimum of 6 months with onset of non-ambulatory status defined as participant- or caregiver-reported age at continuous wheelchair use, approximated to the nearest month, with an NSAA walk score of 0 and inability to perform the 10MWR at the Screening visit.
- •b. Has a PUL entry item score = 3 at the Screening visit.
- •c. Has a total PUL score of = 20 and = 40 at the Screening visit.
- •3. Cohort 2 only (ambulatory = 8 years to < 18 years of age):
- •a. = 8 to < 18 years of age at the time of Screening.
- •b. Has a PUL entry item score > 3 and < 6 at the Screening visit.
- •c. Has a total PUL score of = 20 and = 40 at the Screening visit.
- •d. Has an NSAA score = 12 and = 26 at the Screening visit.
- •4. Able to cooperate with motor assessment testing.
- •5. Has been on a stable dose of oral corticosteroids 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 study. Note that no use of corticosteroids is considered a stable dose (ie, 0 mg).
- •6. Has rAAVrh74 antibody titers < 1:400 (ie, not elevated) as determined by an ELISA.
- •7. Subjects 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). Refer to Appendix 1 for guidance on highly effective contraceptive methods.
- •8. Has (a) parent(s) or legal caregiver(s) or is a subject who is = 18 years of age who is (are) able to understand and comply with the study visit schedule and all other protocol requirements.
- •9. Is willing to provide informed assent or consent (if applicable) and has (a) parent(s) or legal guardian(s) or is a subject =18 years of age who is (are) willing to provide informed consent for the subject to participate in the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 111
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 37
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •A subject who meets any of the following criteria will be excluded from this study:
- •1. Has left ventricular ejection fraction < 40% on the Screening ECHO or clinical signs and/or symptoms of cardiomyopathy.
- •2. Forced vital capacity < 40% of predicted value at Screening.
- •3. Major surgery within 3 months prior to Day 1 or planned surgery or procedure that would interfere with the conduct of the study for any time during this study.
- •4. Presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer therapy or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the subject’s ability to comply with the protocol required testing or procedures or compromise the subject’s wellbeing, safety, or clinical interpretability.
- •5. Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
- •6. Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
- •7. Demonstrates cognitive delay or impairment that could confound motor development in the opinion of the Investigator.
- •8. Treatment with any of the following therapies according to the time frames specified:
- •Any time:
- •- Gene therapy
- •- Cell based therapy (eg, stem cell transplantation)
- •- CRISPR/Cas9, or any other form of gene editing
- •Within 12 weeks of Day 1 and anytime during the study:
- •- Use of human growth factor or vamorolone
- •Within 6 months of Day 1 and anytime during the study:
- •- Any investigational medication
- •- Any treatment designed to increase dystrophin expression (eg, Translarna™, EXONDYS 51™, VILTEPSO™)
- •9. 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.
- •10. Has abnormal laboratory values considered clinically significant including but not limited
- •Gamma-glutamyl transferase (GGT) > 2 × ULN
- •Glutamate dehydrogenase (GLDH) >15 U/L
- •Total bilirubin > ULN. Note that elevations in total bilirubin confirmed to be due toGilbert's syndrome are not exclusionary.
- •White blood cell count > 18,500 per µL
- •Platelets = 150,000 per µL
- •11. Has a known hypersensitivity to SRP-9001 or its excipients
- •12. Subject or family does not want to disclose subject’s study participation with general practitioner/primary care physician and other medical providers.
- •13. In the opinion of the Investigator, the subject is not likely to be compliant with the study protocol.
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