NCT06900192尚未招募1 期
A Multicenter Phase 1 Study of Allogeneic Hematopoietic Cell Transplantation for Primary Progressive Multiple Sclerosis Using Orca-Q, an Engineered Donor Graft Derived From Mobilized Peripheral Blood
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 10
- 主要终点
- Severe acute Graft-versus-Host-Disease-free survival
研究概览
简要总结
A study of alloHCT with Orca-Q for the treatment of primary progressive multiple sclerosis (MS).
详细描述
This study will evaluate alloHCT with Orca-Q, an allogeneic hematopoietic graft isolated from a donor's hematopoietic cells for the treatment of primary progressive MS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •History of Progressive Multifocal Leukoencephalopathy
- •Organ dysfunction or disease that would jeopardize survival after hematopoietic cell transplantation, including but not limited to the following:
- •Renal insufficiency as defined by an estimated GFR <60 mL/minute
- •Cardiac dysfunction as defined by symptomatic coronary artery disease, congestive heart failure, valvular heart disease, cardiomyopathy, uncontrolled arrhythmia(s), or left ventricular ejection fraction <50%. Participants with a history of these conditions may enroll if they are demonstrated to have optimal cardiac function (as defined by echocardiography or multi-gated acquisition scan)
- •Pulmonary dysfunction that poses a risk of mortality after transplant, defined as pre-transplant pulmonary function testing demonstrating a FEV1 <70% expected and/or a DLCOadj <70% expected.
- •Necroinflammatory or fibrotic liver disease with evidence of liver dysfunction, including but not limited to jaundice, hepatic encephalopathy, or portal hypertension
- •Marrow dysfunction that poses a risk of peri-transplant mortality, defined as an absolute neutrophil count (<1000/mm3) below the lower limit of normal, or a platelet count below 50,000/mm
- •Poorly controlled hypertension despite appropriate therapy, defined as a diastolic blood pressure greater than 90 mm Hg while on therapy.
- •Poorly controlled diabetes mellitus, defined as HgbA1c ≥ 6.5% despite therapy or recurrent hypoglycemia while on therapy.
- •Extreme protein-calorie malnutrition defined by Body Mass Index <18 and unintentional weight loss (3 kg in the last month or 6 kg in the last 6 months.)
- •History of smoking either tobacco or other herbal products in the last 3 months.
- •Planned pharmaceutical in vivo or ex vivo T cell depletion (TCD), eg, cladribine, or peritransplant antithymocyte globulin (ATG). For participants who have previously been exposed to a TCD agent, a 5-half-life washout of the agent must occur prior to planned day 0 (day 0 is defined as the day of infusion Orca-Q Prime). The washout period for alemtuzumab is listed in Appendix
- •HIV seropositive.
- •HBV serology results indicating chronic HBV infection per https://www.cdc.gov/hepatitis/hbv/interpretationOfHepBSerologicResults.htm, unless HBV PCR negative. HBV seropositive participants should be on antiviral therapy after transplant.
- •HCV seropositive, unless PCR negative and having undergone 'curative' antiviral therapy.
- •Participant has active uncontrolled infection
- •Participant has demonstrated lack of compliance with prior medical care
- •Participants with known active malignancy. It is recommended that patients are current on cancer screening tests for their age and family history as per the NCCN [The National Comprehensive Cancer Network®] Guidelines. Screening should be performed, if indicated, per NCCN guidelines prior to study treatment.
- •Participants whose life expectancy is severely limited by illness other than multiple sclerosis
- •Females who are pregnant or breast feeding.
- •Medical or psychiatric conditions that compromise ability to give informed consent or to comply with treatment protocol
- •Inability to undergo an MRI scan
- •Currently receiving treatment with investigational agents
- •Positive for JC virus DNA in the CSF or blood during screening.
研究组 & 干预措施
Orca-Q Graft with lymphodepleting conditioning regimen
Experimental
Donor Orca-Q stem cell graft
干预措施: Orca-Q (Biological)
Orca-Q Graft with lymphodepleting conditioning regimen
Experimental
Donor Orca-Q stem cell graft
干预措施: myeloablative regimen (Drug)
结局指标
主要结局
Severe acute Graft-versus-Host-Disease-free survival
时间窗: 365 days after infusion
Alive without a history of moderate to severe aGVHD
次要结局
- Evaluate treatment response(Measurements will be taken at 9 and 12 months after infusion)
- Evaluate safety of treatment(Measured from time of enrollment to end of study participation (from date of consent to month 12).)
研究者
Everett Meyer
Associate Professor of Medicine
Stanford University
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