New stem cell transplant approach aims to tame deadly bone marrow cancer
NCT ID NCT03118492
First seen Jun 24, 2026 · Last updated Aug 21, 2026 · Updated 2 times
Summary
This early-phase trial tests a reduced-intensity stem cell transplant using a half-matched donor and a drug called cyclophosphamide after the transplant to prevent rejection. The goal is to see if this approach is safe enough for patients with advanced myelofibrosis, a serious bone marrow disorder. Only 22 patients are enrolled, and the main focus is on side effects and tolerability.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- donor blood stem cells and cyclophosphamide
- What this could lead to
- If successful, this approach could offer a safer transplant option for patients with advanced myelofibrosis, potentially controlling the disease long-term.
- What could go wrong
- This is a very early, small Phase 1 trial focused on safety, not effectiveness. The transplant carries serious risks including infection, graft failure, and organ damage.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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22 people
The number who actually took part.
- Started
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Jun 2017
- Expected to finish
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Apr 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Diagnosis of primary of secondary myelofibrosis with transplant indication by Dynamic International Prognostic Scoring System (DIPSS)-plus (\> intermediate-1) * Patients \>= age 50 must have a comorbidity score (hematopoietic cell transplant-comorbidity index \[HCT-CI\]) \< 4 (Sorror) * Patients can be in chronic phase (CP) with bone marrow (BM) blast count =\< 15% as long as no evidence of disease acceleration per principal investigator (PI) and treating physician's opinion or after progression to acute myeloid leukemia (AML) and achieved =\< 5% BM blasts (morphologic complete remission \[CR\] prior to transplant) * Lack of an human leukocyte antigen (HLA) matched donor or need to proceed fast to transplantation when a patient does not have an immediately available matched unrelated donor (typed by high-resolution in the registry) * Performance status \>= 70% (Karnofsky); patients \> 50 years should have adequate cognitive function; any concerns regarding cognitive function should be addressed by a geriatrician/neurologist * Alanine aminotransferase (ALT)/aspartate aminotransferase (AST)/bilirubin =\< 5 X upper limit of normal (ULN) * Measured creatinine clearance \> 60 mls/min * Left ventricular ejection fraction (LVEF) \>= 50% * Corrected carbon monoxide diffusing capability (DLCOc) \>= 50% * No active infections * Prior treatment with JAK2 inhibitor therapy is not excluded; a JAK2 inhibitor will need to be stopped 1-2 days prior to starting conditioning regimen * DONOR: Documented informed consent per local, state and federal guidelines * DONOR: Genotypically haploidentical as determined by HLA typing * Preferably a non-maternal HLA haploidentical relative due to data of high incidence of graft failure with use of maternal HLA haploidentical cells * Eligible donors include biological parents, siblings or half-siblings, children, or cousins in rare instances * DONOR: Absence of pre-existing donor-specific anti-HLA antibodies (DSA) in the recipient; Patients with pre-existing DSA could undergo desensitization per City of Hope (COH) standard operating procedures \[SOP\] and should have DSA \< MFI of 2000 prior to conditioning at discretion of PI * DONOR: Infectious disease screening performed within 30 days prior to stem cell mobilization per federal guidelines and is: * Seronegative for HIV 1+2 antibody (Ab) and/or HIV polymerase chain reaction (PCR), human T-cell leukemia virus (HTLV) I/II Ab, hepatitis B virus surface antigen (HBsAg), hepatitis B virus surface antibody (HBcAb), hepatitis C virus (HCV) Ab * Negative rapid plasma reagin (RPR) for syphilis * DONOR: Women of childbearing potential (WOCBP): Urine pregnancy testing performed within 7 days prior to stem cell mobilization * DONOR: Is approved and completed evaluation prior to recipient initiation of the preparative regimen per institutional guidelines Exclusion Criteria: * Evidence of severe portal hypertension with evidence of decompensation either with bleeding varices, large volume ascites, or hepatic encephalopathy * In a bone marrow biopsy 4 weeks prior to start of conditioning on study: * \> 15% bone marrow blasts at transplant if no history of AML and per PI and treating physician's opinion of disease acceleration * \> 5% if had previous progression to AML * Human immunodeficiency virus (HIV) positive; active hepatitis B or C * Patients with active infections; the PI is the final arbiter of the eligibility * Patients with evidence of severe pulmonary hypertension by echocardiogram and confirmed by a subsequent right side cardiac catheterization pre-enrollment * Liver cirrhosis * Prior central nervous system (CNS) involvement by tumor cells * History of another primary malignancy that has not been in remission for at least 3 years (the following are exempt from the 3-year limit: non-melanoma skin cancer, fully excised melanoma in situ \[stage 0\], curatively treated localized prostate cancer, and cervical or breast carcinoma in situ on biopsy or a squamous intraepithelial lesion on papanicolaou \[PAP\] smear) * Positive beta human chorionic gonadotropin (HCG) test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization * Noncompliance - inability or unwillingness to comply with medical recommendations regarding therapy or follow-up, including smoking tobacco * DONOR: Has undergone solid organ, stem cell, bone marrow or blood transplantation * DONOR: Receiving any investigational agents, or concurrent biological, chemotherapy, immunosuppression or radiation therapy * DONOR: Active infection * DONOR: Thrombocytopenia \< 150,000 cells /mm\^3 at baseline evaluation * DONOR: Sero-positive for HIV-1 \& 2 antibody, HTLV-I \& II antibody, hepatitis B virus (HBV) and HCV * DONOR: Medical or physical reason which makes the donor unlikely to tolerate or cooperate with growth factor therapy and leukapheresis * DONOR: Factors which place the donor at increased risk for complications from leukapheresis or G-CSF therapy * DONOR: WOCBP: Pregnant or =\< 6 months breastfeeding
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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City of Hope Medical Center
Duarte, California, 91010, United States
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