NCT07704099尚未招募2 期
A Multicenter, Phase 2, Open-Label Study Evaluating the Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 36
- 主要终点
- Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with Duchenne Muscular Dystrophy (DMD) on stable background therapy.
详细描述
This is a Phase 2, multicenter, open-label study of KER-065.
The study will consist of 3 periods:
- Screening Period (up to 6 weeks)
- Treatment Period (48 weeks)
- Safety Follow-up Period (4 weeks)
Participants will be enrolled in parallel into 1 of 3 treatment cohorts:
- Cohort A1 (Late Ambulatory)
- Cohort A2 (Late Ambulatory)
- Cohort N1 (Early Nonambulatory)
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 9 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of DMD, defined as the presence of phenotypic features at screening consistent with DMD AND documented mutation in the dystrophin gene consistent with the diagnosis of DMD using a clinically validated genetic test.
- •Receiving a stable regimen of systemic CS (including, but not limited to, prednisone, prednisolone, deflazacort, or vamorolone) for at least 90 days before screening.
- •Body weight of ≥ 25.0 kg.
- •Ambulatory Participants Only (Cohort A1 and A2):
- •Ambulatory, defined as able to walk independently without assistive devices.
- •Able to TTR in < 10 seconds.
- •Has a NSAA score ≥ 15 points.
- •Cohort 2 only: Documentation of a stable dose of an approved exon-skipping therapy.
- •Nonambulatory Participants Only (Cohort N1):
- •Nonambulatory, characterized as being unable to ambulate for a minimum of 3 months before first dose with onset of nonambulatory status AND a NSAA walk score of 0 and inability to perform the 10MWR.
- •PUL v2.0 entry item score of 3 to 5, inclusive.
排除标准
- •Clinical symptoms or signs of cardiomyopathy or heart failure.
- •Exposure to any approved or investigational dystrophin restoration gene therapy product.
- •Exposure to any approved or investigational dystrophin restoration product other than gene therapy (Except for exon-skipping therapy for Cohort A2).
- •Exposure to any approved or investigational histone deacetylase inhibitor, antimyostatin therapy, therapy targeting transforming growth factor-beta ligands, or cell-based therapy.
- •Use of any other pharmacological treatment, except for CS
- •Treatment with immunosuppressant therapy (other than CS)
- •History of fracture of the upper limb
- •Nonambulatory Participants Only (Cohort N1):
- •Elbow-flexion contractures > 30° in both upper extremities.
- •Forced vital capacity (FVC) of < 50% or requirement for daytime or nocturnal ventilation, except for nocturnal non-invasive ventilation AND inability to perform consistent FVC measurements within ± 15% during paired testing.
研究组 & 干预措施
Cohort A1 (Late Ambulatory)
Experimental
Participants will receive stable corticosteroid (CS) along with KER-065.
干预措施: KER-065 (Drug)
Cohort A2 (Late Ambulatory)
Experimental
Participants will receive stable CS, exon skipper along with KER-065.
干预措施: KER-065 (Drug)
Cohort N1 (Early Nonambulatory)
Experimental
Participants will receive stable CS along with KER-065.
干预措施: KER-065 (Drug)
结局指标
主要结局
Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)
时间窗: Up to approximately 3 years
To evaluate the safety and tolerability of KER-065 in ambulatory and nonambulatory participants with DMD
Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)
时间窗: Up to approximately 14 months
To evaluate the safety and tolerability of KER-065 in ambulatory and nonambulatory participants with DMD
次要结局
- KER-065 serum concentration by visit, as appropriate(Up to Week 100)
- Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit(Up to Week 100)
- Change from baseline by visit in bone mineral density (BMD), fat mass, and lean body mass, as measured by dual- energy X-ray absorptiometry (DXA)(Up to Week 96)
- Change from baseline by visit in muscle volume and intramuscular fat by skeletal muscle MRI(Up to Week 96)
- Ambulatory: Change from baseline by visit in North Star Ambulatory Assessment (NSAA) total score(Up to Week 96)
- Ambulatory: Change from baseline by visit in 4-stair climb (4SC)(Up to Week 96)
- Ambulatory: Change from baseline by visit in 10-meter walk/run (10MWR) test(Up to Week 96)
- Ambulatory: Change from baseline by visit in TTR (time to rise)(Up to Week 96)
- Nonambulatory: Change from baseline by visit in PUL (Performance of Upper Limb) v2.0 score(Up to Week 96)
- KER-065 serum concentration by visit, as appropriate(Up to Week 52)
- Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit(Up to Week 52)
- Change from baseline by visit in bone mineral density (BMD), fat mass, and lean body mass, as measured by dual- energy X-ray absorptiometry (DXA)(Week 12, Week 24 and Week 48)
- Change from baseline by visit in muscle volume and intramuscular fat by skeletal muscle Magnetic Resonance Imaging (MRI)(Week 24 and Week 48)
- Ambulatory: Change from baseline by visit in North Star Ambulatory Assessment (NSAA) total score(Week 24 and Week 48)
- Ambulatory: Change from baseline by visit in 4-stair climb (4SC)(Week 24 and Week 48)
- Ambulatory: Change from baseline by visit in 10-meter walk/run (10MWR) test(Week 24 and Week 48)
- Ambulatory: Change from baseline by visit in time to rise (TTR)(Week 24 and Week 48)
- Nonambulatory: Change from baseline by visit in Performance of Upper Limb (PUL) v2.0 score(Week 24 and Week 48)
研究者
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