DRKS00031683招募中3 期
A Randomized, Double-Blind, PlaceboControlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-AmbulatoryParticipants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT) - BHV2000-301
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 16
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized controlled study
- 盲法
- Blinded (masking used)
入排标准
- 年龄范围
- 4 Years 至 21 Years（—）
- 性别
- All
入选标准
- •1. Signed Written Informed Consent
- •2. Participants must agree to provide all requested demographic information (i.e. gender, race).
- •Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number (genetic diagnosis and SMN2 copy number confirmed with documentation either prior to enrollment from previous testing or confirmed during study)
排除标准
- •1. Participation in any other investigational clinical trial while participating in this clinical trial with the exception of registry and natural history studies that do not involve procedures that
- •could impact blinding (such as drawing blood for biomarkers). Also see exclusion 4.j regarding investigational drug trials.
- •2. Receiving or have received previous administration of anti-myostatin therapies
- •3. Weight <15 kg
- •4. Medical History and Concurrent Diseases
- •a. Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)
- •b. Hospitalization for a pulmonary event within the last 2 months or planned hospitalization at the time of screening
- •c. History of spinal fusion within 6 months prior to screening. Note: MAGEC rod nonsurgical adjustments are allowed during the study.
- •5. Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration.
- •6. All vaccines should not be given within 2 weeks of the first dose.
- •7. The participant has a history of intolerance to venipuncture and/or difficult venipuncture access.
- •8. ECG and laboratory test findings:
- •Clinically significant abnormality identified on the medical or laboratory evaluation. A participant with a clinical abnormality or laboratory parameters outside the reference range may be included only if the investigator considers the finding not clinically significant, that it will not introduce additional risk factors, nor interfere with the studyprocedures.
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