Status:
NOT_YET_RECRUITING
A Study of Allogeneic Hematopoietic Cell Transplantation for Primary Progressive Multiple Sclerosis
Lead Sponsor:
Stanford University
Collaborating Sponsors:
Orca Biosystems, Inc.
Conditions:
Primary Progressive Multiple Sclerosis
Multiple Sclerosis
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
Brief Summary
A study of alloHCT with Orca-Q for the treatment of primary progressive multiple sclerosis (MS).
Detailed Description
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.
Eligibility Criteria
Inclusion
- Inclusion Criteria (Recipient):
- Participants aged ≥18 and ≤65 years with primary progressive multiple sclerosis
- A diagnosis of PPMS by 2017 McDonald criteria and 2013 clinical course revision102
- CSF with elevated IgG index or 2 or more oligoclonal bands
- EDSS (see Appendix 9) between 2.0 and 5.5 inclusive
- Based on review of the clinical records, there must be a deterioration in the EDSS of at least 1 or more points over the previous 4 years (or less).
- Eligibility criteria confirmed by the Eligibility Review Group (Appendix 10)
- Recipients who have been treated with ocrelizumab, rituximab, ofatumumab, ublituximab, or alemtuzumab must undergo a washout period as described in Appendix 14 prior to their planned day 0.
- Recipients must be willing to undergo mobilized autologous peripheral blood stem cell collection to create a cryopreserved rescue product prior to alloHCT.
- Ability to undergo MRI without general anesthesia
- Ability to undergo all tests and procedures in the study
- Patients who are not vaccinated for COVID-19 must have no symptoms of COVID-19 and have negative testing for COVID-19. For other vaccine-preventable illnesses, it is recommended that patients are current on their vaccination schedule.
- Exclusion Criteria:
- 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/mm3.
- 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 14.
- 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.
Exclusion
Key Trial Info
Start Date :
March 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2029
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT06900192
Start Date
March 1 2025
End Date
August 1 2029
Last Update
March 28 2025
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