Clinical trial · Interventional
OTI-010 for Graft-Versus-Host Disease Prophylaxis in Treating Patients Who Are Undergoing Donor Peripheral Stem Cell Transplantation for Hematologic Malignancies
A Phase II, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of OTI-010 in Subjects Who Receive HLA-Identical Sibling Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies
NCT00081055CI-TRIAL-00016239withdrawnPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
RATIONALE: OTI-010 may be effective for graft-versus-host disease prophylaxis (prevention) in patients who are undergoing donor peripheral stem cell transplantation for hematologic malignancies (cancer of the blood or bone marrow). PURPOSE: This randomized phase II trial is studying how well OTI-010 works in preventing graft-versus-host disease in patients who are undergoing donor peripheral stem cell transplantation for hematologic cancer.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Graft Versus Host Disease | — | UNRESOLVED | — |
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
Interventions
Interventions (7)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| autologous expanded mesenchymal stem cells OTI-010 | Biological | — | UNRESOLVED |
| busulfan | Drug | Busulfan | ALIAS |
| cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| cyclosporine | Drug | — | UNRESOLVED |
| methotrexate | Drug | Methotrexate | ALIAS |
| peripheral blood stem cell transplantation | Procedure | — | UNRESOLVED |
| radiation therapy | Radiation | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (1)
- measure
- Incidence of acute GVHD grade II-IV of skin, liver and gut (stomach to rectum) through Day 84 post-PBSC transplantation
- timeFrame
- Day 84
Secondary outcomes (1)
- measure
- Safety as measured by infusional toxicity, relapse nd survival, formation of potential ectopic tissue foci
- timeFrame
- 84 days
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 55 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Histologically confirmed diagnosis of 1 of the following hematologic malignancies:
* Acute lymphoblastic leukemia, meeting 1 of the following criteria:
* In first or second remission
* In early first or second relapse\*
* Acute myeloid leukemia, meeting 1 of the following criteria:
* In first or second remission
* In early first or second relapse\*
* Chronic myelogenous leukemia
* Chronic or accelerated phase
* Any of the following myelodysplastic syndromes:
* Refractory anemia (RA)
* RA with ringed sideroblasts
* RA with excess blasts NOTE: \*\< 24% marrow blasts and \< 5% peripheral blood blasts (within 10 days of beginning conditioning regimen)
* No secondary acute leukemia
* Prior CNS tumor involvement allowed provided patient is asymptomatic and there is no evidence of CNS disease on lumbar puncture and CT scan of the brain
* Must have a 6/6 HLA-identical sibling donor available
PATIENT CHARACTERISTICS:
Age
* 18 to 55
Performance status
* Karnofsky 70-100%
Life expectancy
* Not specified
Hematopoietic
* Not specified
Hepatic
* Bilirubin \< 2 times upper limit of normal (ULN)
* SGOT \< 10 times ULN
* Hepatitis B core antigen, surface antigen, and e-antigen negative
* Hepatitis B DNA negative
* Hepatitis C RNA negative
Renal
* Creatinine clearance ≥ 60 mL/min
Cardiovascular
* LVEF ≥ 50% by MUGA or echocardiogram
* No right sided heart failure
Pulmonary
* FEV\_1 \> 50% of predicted
* DLCO ≥ 50% of predicted (corrected for anemia)
* Oxygen saturation ≥ 97% on room air
* No pulmonary hypertension
Immunologic
* HIV-1 and 2 antibody negative
* HIV-1 antigen negative
* HTLV-I and II antibody negative
* No active infection
Other
* CNS function normal
* No uncontrolled alcohol or substance abuse within the past 6 months
* No other concurrent underlying medical condition that would preclude study participation
* Not pregnant
* Negative pregnancy test
* Fertile patients must use 2 effective methods of contraception
PRIOR CONCURRENT THERAPY:
Biologic therapy
* No prior allogeneic or autologous hematopoietic stem cell transplantation
* No concurrent medication to accelerate neutrophil or platelet engraftment except filgrastim (G-CSF)
Chemotherapy
* Not specified
Endocrine therapy
* Not specified
Radiotherapy
* Not specified
Surgery
* No prior solid organ transplantation
Other
* More than 30 days since prior investigational agents or devices
* No other concurrent investigational agents or devices
* No concurrent anti-infective therapy except prophylactic therapy
* No other concurrent conditioning regimen agents
* No concurrent herbal remedies except multivitamins
* No other concurrent graft-versus-host disease prophylaxis medications (e.g., ursodeoxycholic acid)References
Publications (0)
Data not yet available
No reference posted for this study.