2025-523811-12-00招募中3 期
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 versus Vosoritide in Children with Achondroplasia
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 25
- 试验地点
- 7
- 主要终点
- Phase 2: Predicted AGV at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data])
研究概览
简要总结
Phase 2: To evaluate the effect of 3 doses of BMN 333 versus vosoritide XXX on AGV (interim analysis) Phase 3: To evaluate the effect of BMN 333 at a selected dose versus vosoritide on AGV after 52 weeks of treatment.
研究设计
- 分配方式
- Randomized
- 主要目的
- Phase III
- 盲法
- Single (Analyst)
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •1.Participants must be aged ≥ 2 to < 11 years (Phase 2) or ≥ 2 to < 18 years (Phase 3), at the time of signing the informed consent.
- •2.Participants must have ACH (confirmed by documented genetic testing) and open epiphyses assessed using left hand postero-anterior (PA) X-rays, by the Greulich and Pyle method (Greulich 1971) as per standard of care.
- •3.Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening visit and be willing to have additional pregnancy tests during the study.
- •4.Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)
- •5.Have a body weight ≥ 8.0 kg at the Screening visit
- •6.Are ambulatory and able to stand without assistance
- •7.Caregivers are willing to administer injections to the participants and willing to complete the required training.
排除标准
- •1.Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency).
- •2.Have any of the following disorders:Hypothyroidism or hyperthyroidism (unless treated) ,Diabetes mellitus (unless considered well-controlled), Autoimmune inflammatory disease, Autoimmune inflammatory disease ,Autonomic neuropathy.
- •3.Have history of: Renal insufficiency, Cardiac or vascular disease, unstable clinical condition , significant hip pathology.
研究组 & 干预措施
BMN 333, BMN 333
Test
干预措施: BMN 333 (Drug)
Voxzogo 0.56 mg powder and solvent for solution for injection, Voxzogo 1.2 mg powder and solvent for solution for injection
Comparator
干预措施: Voxzogo 0.56 mg powder and solvent for solution for injection (Drug)
Voxzogo 0.56 mg powder and solvent for solution for injection, Voxzogo 1.2 mg powder and solvent for solution for injection
Comparator
干预措施: Voxzogo 1.2 mg powder and solvent for solution for injection (Drug)
结局指标
主要结局
Phase 2: Predicted AGV at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data])
Phase 2: Predicted AGV at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data])
Phase 3: AGV at Week 52
Phase 3: AGV at Week 52
次要结局
- Phase 2: 1.AGV at Weeks 26 and 52 2.Change from Baseline in standing height, height Z-score and in upper to lower body segment ratio at Weeks 26 and 52 3.Incidence of AEs, SAEs, and EOIsa; physical examinations; vital signs; ECG findings; imaging results (X-ray and DXA); clinical laboratory tests.
- Phase 3: • Change from Baseline in standing height, height Z-score, upper to lower body segment ratio at Week 52
研究者
BioMarin Pharmaceutical Inc.
Scientific
Biomarin Pharmaceutical Inc.
研究点 (7)
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