EUCTR2018-001762-42-GR进行中（未招募）1 期
A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 154
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •A patient must meet all of the following criteria to be eligible to
- •participate in this study.
- •1. Be a male with an established clinical diagnosis of DMD and an out-of
- •frame deletion mutation of the DMD gene amenable to exon 51 skipping
- •(for example, deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, and
- •2. Be aged 4 to 13 years, inclusive
- •3. Ambulatory patient, able to perform TTRISE in 10 seconds or less at
- •the time of screening visit.
- •4. Able to walk independently without assistive devices.
- •5. Has intact right and left biceps muscles (the preferred biopsy site) or
- •an alternative upper arm muscle group that will allow for sufficiently
- •sized (1 cm3) muscle biopsies to be obtained prior to and on treatment
- •(for patients in the double-blind part of the study).
- •6. Has been on a stable dose or dose equivalent of oral corticosteroids
- •for at least 12 weeks prior to randomization, and the dose is expected to
- •remain constant (except for modifications to accommodate changes in
- •weight and stress-related needs as per recently published guidelines
- •throughout the study).
- •7. For ages 7 years and older, has stable pulmonary function (forced
- •vital capacity =50% of predicted and no requirement for nocturnal
- •ventilation) that, in the Investigator's opinion, is unlikely to
- •decompensate significantly over the duration of the study.
- •For ages 4 to 6 years , does not require support from ventilator or noninvasive
- •ventilation at time of screening.
- •8. If sexually active, agree to use a male condom during such activity for
- •the entire duration of the study and for 90 days after the last dose. The
- •sexual partner must also use a medically acceptable form of
- •contraceptive (ie, female oral contraceptives) during this timeframe.
- •Acceptable methods of contraception include combined (estrogen and
- •progesterone containing) hormonal contraception associated with
- •inhibition of ovulation (oral, intravaginal, or transdermal); progesteroneonly
- •hormonal contraception associated with inhibition of ovulation
- •(oral, injectable, or implantable); intrauterine device; intra-uterine
- •hormone-releasing system; bilateral tubal occlusion; vasectomized
- •partner; sexual abstinence; or condom in combination with either cap,
- •diaphragm, or sponge with spermicide (double-barrier contraception).
- •9. Has (a) parent(s) or legal guardian(s) who is (are) able to
- •understand and comply with all the study requirements.
- •10. Is willing to provide informed assent (if applicable) and has (a)
- •parent(s) or legal guardian(s) who is (are) willing to provide informed
- •consent for the patient to participate in the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 154
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •A patient who meets any of the following criteria will be excluded from
- •this study:
- •1. Use of any pharmacologic treatment (other than corticosteroids)
- •within 12 weeks prior to randomization that may have an effect on
- •muscle strength or function. Growth hormone for short stature and
- •testosterone for delayed puberty are permitted if physician has
- •documented the diagnosis and medical necessity of treatment and if the
- •patient has been on a stable dose for at least 24 weeks prior to
- •randomization.
- •2. Current or previous treatment with any other experimental
- •pharmacologic treatment for DMD or any prior exposure to antisense
- •oligonucleotide, gene therapy or gene editing; except the following:
- •Ezutromid in the last 12 weeks prior to first dose.
- •Drisapersen in the last 36 weeks prior to first dose
- •Suvodirsen in the last 12 weeks prior to first dose.
- •Vamorolone in the last 12 weeks prior to first dose.
- •Eteplirsen (previous or current use)
- •3. Major surgery within 3 months prior to randomization or planned surgery for any time during this study, except for allowed protocolspecified
- •surgery, as applicable.
- •4. Presence of any significant neuromuscular or genetic disease other
- •than DMD (eg, dwarfism).
- •5. Gamma-glutamyl transpeptidase (GGT) > 3 × the upper limit of
- •normal (ULN) or serum bilirubin > ULN unexplained by Gilbert's
- •6. Any known impairment of renal function (eg, estimated glomerular
- •filtration rate [eGFR] = 60 mL/min as assessed by the Chronic Kidney
- •Disease Epidemiology Collaboration [CKD-EPI] equation), or dipstick
- •protein result +2, or persistent and unexplained dipstick protein result
- •7. Platelet count < the lower limit of normal.
- •8. Presence of other clinically significant illness including significant
- •cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or
- •behavioral disease or malignancy.
- •9. Has evidence of cardiomyopathy, as defined by left ventricular
- •ejection fraction <50% on the screening ECHO or the Fridericia's
- •correction formula (QTcF) =450 milliseconds based on the screening
- •10. Prior or ongoing medical condition that could, in the Investigator's
- •opinion, adversely affect the safety of the patient, make it unlikely that
- •the course of treatment would be completed, or impair the assessment
- •of study results.
- •11. Known hypersensitivity to eteplirsen or any excipients of eteplirsen.
- •12. Is, in the Investigator's opinion, unable or unwilling to comply with
- •the study procedures.
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