NL-OMON56088招募中3 期
Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 1014 vector genomes) to participants 2 < 18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®) - COAV101B12302 (STRENGTH)
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- ovartis
- 入组人数
- 7
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
- 2 至 17（—）
入选标准
- •* Written informed consent
- •* SMA diagnosis based on gene mutation analysis with bi-allelic SMN1 mutations
- •and any copy of SMN2 gene
- •* Aged 2 < 18 years (screening visit must occur before the patient's 18th
- •birthday) at time of Screening Visit 1
- •* Have had at least four loading doses of nusinersen (Spinraza®) or at least 3
- •months of treatment with risdiplam (Evrysdi®) at Screening
- •* Must be able to sit independently but must never have taken steps
- •independently
- •* Diagnosed through newborn or neonatal screening or patients clinically
- •diagnosed must have age of clinical symptom onset < 18 months
- •* Meets age-appropriate institutional criteria for use of anesthesia/sedation
- •* Female participants who are sexually active or have reached menarche must
- •have a negative pregnancy test at Screening. Those females who are sexually
- •active must also agree to use highly effective methods of contraception.
排除标准
- •* Excluding SMA, any medical condition considered clinically significant
- •* Positive for human immunodeficiency virus (HIV), hepatitis B or hepatitis
- •* Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an
- •immunoassay is reported as elevated at Screening (reference to >1:50 or a
- •validated result consistent with being elevated)
- •* Clinically significant abnormalities in test results during screening period
- •and/or at Baseline
- •* Platelet count less than the lower limit of normal (LLN), or platelet
- •transfusion within 1 month at Screening Visit 1
- •* Clinically significant abnormal coagulation panel results at Screening
- •* Hepatic dysfunction (i.e. alanine aminotransferase (ALT), total bilirubin
- •(TBL), gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH) >
- •upper limit of normal (ULN) at Screening (with the exception of isolated AST
- •elevation: in the absence of other liver laboratory abnormalities, isolated
- •elevated AST is not considered exclusionary)
- •* Contraindications for lumbar puncture procedure
- •* At Baseline (Day-1), participants are excluded if they received:
- •* nusinersen (Spinraza®) within 4 months at Baseline
- •* risdiplam (Evrysdi®) within 15 days at Baseline
- •* Vaccinations 2 weeks prior to administration of OAV101
- •* Hospitalization for a pulmonary event, or for nutritional support within 2
- •months prior to Screening or inpatient major surgery planned.
- •* Presence of the following:
- •* An active infectious process requiring systemic antiviral or antimicrobial
- •therapy up to 30 days prior to OAV101 administration, or
- •* An active but untreated viral or bacterial infectious process up to 30 days
- •prior to administration of OAV101, or
- •* Any febrile illness up to 30 days prior to administration of OAV101
- •* Requiring invasive ventilation, awake noninvasive ventilation for > 6 hours
- •during a 24-hour period, noninvasive ventilation for >12 hours during a 24-hour
- •period or requiring tracheostomy, at Screening and up to OAV101 administration
- •* Concomitant use of any of the following medication categories within 90 days
- •prior to administration of OAV101
- •* Ongoing systemic immunosuppressive therapy (e.g., corticosteroids,
- •cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous
- •immunoglobulin, rituximab), plasmapheresis, immunomodulators (e.g., adalimumab)
- •* History of hypersensitivity to any of the study treatments or its excipients
- •or drugs of similar chemical classes
研究者
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