NL-OMON54626招募中2 期
A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment - SRP-5051-201
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 6
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
- 年龄范围
- 18 至 99（—）
入选标准
- •Inclusion Criteria for Patients Previously Treated with SRP-5051.
- •Patients previously treated with SRP-5051 must meet all of the following
- •criteria to participate in this study:
- •I1. Has received prior SRP-5051 treatment in Part A of this study or in Study
- •5051-102.
- •I2. If sexually active, agrees to use a male condom during such activity for
- •the entire duration of the study and for 90 days after the last dose of study
- •drug. The sexual partner must also use a highly effective form of contraceptive
- •(refer to Appendix 1 of the protocol for guidance on highly effective
- •contraceptive methods) during this timeframe.
- •I3. Is willing to provide informed consent or informed assent (if applicable)
- •and has (a) parent(s) or legal guardian(s) who is (are) willing to provide
- •written informed consent for the patient to participate in the study.
- •Inclusion Criteria for Patients Treatment-Naïve to SRP-5051 Patients who are
- •treatment-naïve to SRP-5051 must meet all of the following criteria to
- •participate in this study:
- •I1. Is male.
- •I2. Is 7 to 21 years of age, inclusive.
- •I3. Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the
- •DMD gene amenable to exon 51-skipping treatment.
- •I4. Has been on a stable dose of oral corticosteroids for at least 12 weeks
- •prior to study drug administration or has not received corticosteroids for at
- •least 12 weeks prior to study drug administration.
- •I 5.Has stable pulmonary function (FVC >= 40% of predicted and no requirement
- •for nocturnal ventilation as a result of the complications of DMD) that, in the
- •Investigator*s opinion, is unlikely to decompensate significantly over the
- •duration of the study. NOTE: patients on nocturnal ventilation because of sleep
- •apnea, obesity, or other conditions caused by corticosteroid use are allowed to
- •participate in the study if FVC % predicted is >= 40
- •I 6. If sexually active,.
- •I6. If sexually active, agrees to use a male condom during such activity for
- •the entire duration of the study and for 90 days after the last dose of study
- •drug. The sexual partner must also use a highly effective form of contraceptive
- •(refer to Appendix 1 of the protocol for guidance on highly effective
- •contraceptive methods) during this timeframe.
- •I7. Is willing to provide informed consent or informed assent (if applicable)
- •and has (a) parent(s) or legal guardian(s) who is (are) willing to provide
- •written informed consent for the patient to participate in the study.
排除标准
- •Exclusion Criteria for Patients Previously Treated with SRP-5051:
- •E 1. Has a current infection, or history of an infection within 12 weeks prior
- •to Day -1 requiring intravenous treatment with an antibiotic, or oral
- •antibiotics that may affect renal or cardiac function.
- •E 2. Has a known kidney disease (identified by eGFR [calculated using Schwartz
- •2012 cystatin C equation] of < 90 mL/min/1.73 m2) or had an acute kidney injury
- •within 24 weeks prior to Screening.
- •E 3. Major surgery within 12 weeks prior to Screening, or planned surgery or
- •procedures that would interfere with the conduct of the study.
- •E 4. Presence of other clinically significant illness, including cardiac,
- •pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or
- •infection or malignancy.
- •E 5. Any other condition that, in the Investigator's opinion, could interfere
- •with the patient's participation in the trial, including body weight loss to <
- •E 6. Inability to comply with the study protocol.
- •E 7. Is an employee of the Investigator or study center, with direct
- •involvement in the proposed study or other studies under the direction of that
- •Investigator or study center, as well as family members of the employees or the
- •Investigator.
- •E 8. Any patient who is taking medications that increase the risk of bleeding,
- •in the Investigator's opinion
- •E 9. Platelet count < 150 × 10^3/µL.
- •E 10. Known hypersensitivity to the study drug (ie, SRP-5051) or to any of its
- •components.
- •E 11. Has:
- •a. Hypomagnesemia (< lower limit of normal) at Screening
- •b. Other abnormal electrolyte values considered clinically significant by the
- •Investigator upon medical review and in consultation with the Medical Monitor
- •at Screening;
- •c. Serum creatinine > upper limit of normal (ULN) at Screening.
- •E 12. Has quantitative urinalysis or urine microscopy findings above the ULN
- •for RBCs or WBCs.
- •E 13. Urine Protein/Creatinine Ratio >= 200 mg/g and UACR >= 30 mg/g OR 24-hour
- •urine values for protein >= 200 mg/24 hr at Screening and albumin >= 30 mg/24 hr.
- •(Note that 24-hour urine protein does not need to be performed during screening
- •if the UPCR/UACR criteria are met).
- •E 14. GGT > 3 × ULN at Screening
- •E 15. Is being treated with a proton pump inhibitor, loop diuretic, or thiazide
- •diuretic at the time of study initiation.
- •E 16. Treatment with any exon 51-skipping therapy within 4 weeks prior to
- •Screening, or with any experimental gene therapy for the treatment of DMD at
- •For other exclusion criteria please refer to Protocol
- •Exclusion Criteria for Patients Treatment-Naïve to SRP-5051:
- •E 1. History of hypomagnesemia within 12 weeks prior to Screening.
- •For TN Cohort patients entering the study in Part B
- •E 2. Has body weight < 18 kg.
- •E 3. Has a diagnosis of diabetes (any type).
- •E 4. Initiation or change of dosing (except for modifications to accommodate
- •changes in weight or changes in standard of care) within 12 weeks prior to
- •Screening for any of the following: angiotensinconverting enzyme inhibitors,
- 另有 4 项未显示
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