EUCTR2021-006709-31-NL招募中1 期
Phase IIIb, open-label, single-arm, multi-center study to evaluate thesafety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x1014 vector genomes) to participants 2 to < 18 years of age with spinalmuscular atrophy (SMA) who have discontinued treatment with nusinersen(Spinraza®) or risdiplam (Evrysdi®) - STRENGTH
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 28
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
- 性别
- All
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 to < 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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 28
- •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
- •? 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
- •? 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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