PHASE II, OPEN LABEL, SINGLE DOSE STUDY OF THE SAFETY AND EFFICACY OF MNV-201 FOR THE TREATMENT OF PEARSON SYNDROME
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Occurrence of treatment-related adverse events
研究概览
简要总结
Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders caused by mutations in genes encoded by nuclear Deoxyribonucleic Acid (DNA) or by mutations and/or deletions in the mitochondrial DNA (mtDNA). While some mitochondrial disorders only affect a single organ (e.g., the eye in Leber hereditary optic neuropathy [LHON]), many involve multiple organs. Mitochondrial disorders may present at any age and a frequent feature is the increasing number of organs involved in the course of the disease.
Minovia Therapeutics Ltd. ("Minovia") is a biotech company developing novel therapeutics based on its mitochondrial augmentation technology (MAT). MNV-201 is a cell therapy produced by MAT that consists of the participant's autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) enriched with allogeneic placental-derived mitochondria, manufactured in Minovia's GMP facility.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants aged from 1 to 18 years old.
- •Diagnosis of Pearson Syndrome (current or history) as verified by molecular identification of deletion in mtDNA of peripheral blood. Participants are diagnosed with PS Participant can be in either the PS manifestations of the disease or may have transitioned to Kearns Sayre Syndrome (KSS) manifestations but has a history of PS.
- •Participants have failure to thrive (height SDS smaller than -1)
- •Participants should have at least 12 months' history of body weight and height and calculated GFR (from creatinine) before treatment.
- •Body weight ≥ 10 kg.
- •Participants' living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent.
- •Participants' parents or legal guardian have a good understanding of the study and nature of the procedure and are expected to be able to comply with study visit schedules and caregiver assessments without difficulty.
- •Participants' parents or legal guardian provides written informed consent prior to study participation.
- •Participants are medically able to undergo the study interventions as determined by the Investigator.
- •Exclusion criteria:
- •History of infection with HIV-1, HIV-2, or HTLV I/II.
- •Participants have any active infection.
- •Participants have been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype.
- •Participants are unable to undergo apheresis.
- •Participants have known hypersensitivity to murine proteins or iron-dextran.
- •Participants have severe chronic infection.
- •Participants have disease or conditions that may risk the participant or interfere with the ability to interpret the study results.
- •History of malignancy.
- •History of treatment with gene therapy, allogeneic bone marrow or cord blood transplantation.
- •Participants have had a change in growth hormone regimen in less than 2 years prior to treatment.
- •Participants have participated in another clinical trial or received other experimental medications outside a clinical trial within 1 month prior to start of this study.
- •Participants who are pregnant or intend to become pregnant in the next 12 months.
- •In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.
排除标准
- 未提供
研究组 & 干预措施
Autologous CD34+ cells enriched with allogenic placenta-derived mitochondria
干预措施: MNV-201 (Biological)
结局指标
主要结局
Occurrence of treatment-related adverse events
时间窗: 12 months post treatment.
Occurrence of treatment-related adverse events as assessed by CTCAE v5.0 following MNV-201 infusion
Height SDS
时间窗: 24 months
Improvement from baseline to 12 months post treatment in height SDS compared to the calculated change in height SDS in the 12 months prior to treatment.
次要结局
- Height SDS(12 months)
- Calculated GFR Slope(24 months)