NCT07180355招募中1 期
A Phase 1b First-in-Human, Open-Label, Dose-Finding Trial to Evaluate the Safety and Tolerability of SGT-212 Delivered Via Dual Intradentate Nucleus (IDN) and Intravenous (IV) Administration to Participants With Friedreich's Ataxia (FA)
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 4
- 主要终点
- Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA.
All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.
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
- 18 Years 至 40 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has history of FA symptom onset ≤25 years of age
- •Has a clinical and genetic diagnosis of FA
- •Has a staging score of ≥1 but <6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score
- •Is willing to agree to the following rules for use of omaveloxolone (Skyclarys):
- •For a candidate who is currently taking omaveloxolone, has been on a stable dose for 12 weeks, expects to continue taking omaveloxolone at that dose throughout the study, and is willing to stop taking omaveloxolone at the direction of the Investigator or Sponsor's Medical Monitor if evidence of transaminitis or synthetic liver dysfunction is detected during the study
- •For a candidate who is not actively taking omaveloxolone, at least 12 weeks have passed since the last dose and the candidate agrees not to resume omaveloxolone during the 18-month period after SGT-212 infusion NOTE: The use of any other approved or investigational medicinal product for the treatment of FA should be discussed with the study team.
排除标准
- •Antibodies against adeno-associated virus serotype 9 (AAV9)
- •Has a modified FARS (mFARS) score <20
- •Has a body weight ≤25 kilogram (kg) or has body mass index (BMI) ≥33 kg/m^2
- •Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization
- •Is unable to undergo cardiac and brain MRI with contrast, including hypersensitivity to gadolinium contrast agent, presence of a non-MRI-compatible cardiac pacemaker, presence of a non-MRI-compatible implantable cardiac defibrillator, or physical condition (e.g., contractures)
- •Has uncontrolled diabetes as defined by a hemoglobin (Hb) A1c >9%
- •Has participated in recent interventional clinical studies or received any investigational therapy administered within 3 months or 5 half-lives (whichever is longer) prior to Screening
- •Has received gene therapy at any time
- •Has contraindications to receiving corticosteroids
- •Has any contraindication to the surgical procedures involved with IDN infusion of SGT-212
- •Has any known cardiac disease not related to FA including known obstructive coronary artery disease (CAD)
- •Other Inclusion/Exclusion criteria to be applied as per protocol.
研究组 & 干预措施
Cohort 3 (Ambulatory and Non-Ambulatory)
Experimental
Participants will receive bilateral IDN followed by systemic IV infusion.
干预措施: SGT-212 (Drug)
Cohort 1 (Non-Ambulatory)
Experimental
Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion.
干预措施: SGT-212 (Drug)
Cohort 2 (Ambulatory)
Experimental
Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion.
干预措施: SGT-212 (Drug)
结局指标
主要结局
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
时间窗: Month 12
次要结局
- Number of Participants with Change from Baseline in Significant Abnormalities in Physical Examination Findings(Baseline, Months 18 and 60)
- Incidence and Severity of TEAEs(Months 18 and 60)
- Incidence and Severity of Treatment-emergent Serious adverse events (SAEs)(Months 18 and 60)
- Number of Treatment-emergent deaths(Months 18 and 60)
- Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)(Baseline, Months 18 and 60)
- Number of Participants with Change from Baseline in Significant Abnormalities in Laboratory Tests(Baseline, Months 18 and 60)
- Number of Participants with Change from Baseline in Significant Abnormalities in Vital Signs(Baseline, Months 18 and 60)
- Number of Participants with Change from Baseline in Innate and Adaptive Immune Responses(Baseline, Months 18 and 60)
- Change from Baseline in Blood Biomarkers Including Inflammatory Markers(Baseline, Month 12)
研究者
研究点 (4)
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