Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03301168 · ClinicalTrials.gov registry record · Phase 1
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
A Phase 1 study of Myelodysplastic Syndromes and Acute Lymphoblastic Leukemia, sponsored by Bellicum Pharmaceuticals.
- Active
- Registry status
- Phase 1
- Development phase
- 120
- Enrollment target
- 10
- Study locations
NCT03301168: Active Phase 1 study of Myelodysplastic Syndromes and Acute Lymphoblastic Leukemia, sponsored by Bellicum Pharmaceuticals.
NCT03301168 is a Phase 1 study of Myelodysplastic Syndromes and Acute Lymphoblastic Leukemia that is active but no longer recruiting, run by Bellicum Pharmaceuticals. The registered enrollment target is 120 participants, below the 284-participant average among 173 other Myelodysplastic Syndromes trials with a reported enrollment target (58% lower). The trial reports 10 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03301168, a Phase 1 study of Myelodysplastic Syndromes and Acute Lymphoblastic Leukemia, is active but no longer recruiting, sponsored by Bellicum Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 120 participants
- Enrollment target
- 10
- Study locations
Study Summary
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).
Primary Outcome
Demonstrate safety of BPX-501 MTD
Conditions Studied
Interventions
- DRUG Rimiducid
- BIOLOGICAL BPX-501 T cells
Study Locations (10)
California
- Children's Hospital Los Angeles - Los Angeles
- Stanford University - Division of Pediatric Stem Cell Transplant & Regenerative Medicine - Palo Alto
Texas
- University of Texas Southwestern-Children's Medical Center - Dallas
- Baylor College of Medicine/ Texas Children's Hospital - Houston
District of Columbia
- Children's National Medical Center - Washington D.C.
Georgia
- Children's Healthcare of Atlanta - Atlanta
Massachusetts
- Dana-Farber Boston Children's Cancer and Blood Disorders Center - Boston
New York
- Children's Hospital at Montefiore - The Bronx
Oregon
- Oregon Health Sciences University - Doernbecher Children's Hospital - Portland
Washington
- Fred Hutchinson Cancer Research Center - Seattle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 120 participants |
| Start Date | 2014-04 |
| Est. Completion | 2034-05 |
| Phase | Phase 1 |
What the registry record for NCT03301168 still lists
NCT03301168 is an interventional study that assigns participants to a tested intervention. The registered 120 participants enrollment target is mid-sized for trials with a published cap, below the 284-participant average among 173 other Myelodysplastic Syndromes trials with a reported enrollment target (58% lower).
The record links to 9 conditions, with Myelodysplastic Syndromes appearing as the primary indexed condition, and to 2 interventions - of which Rimiducid is the first listed.
NCT03301168 lists 10 locations in 8 states (California, Texas, District of Columbia).
Frequently Asked Questions
What is clinical trial NCT03301168 about?
NCT03301168 is a clinical study titled "Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant". This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T ce...
What is the current status of trial NCT03301168?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 120 participants. The study started on 2014-04. Estimated completion is 2034-05.
What conditions does trial NCT03301168 study?
This clinical trial studies the following conditions: Myelodysplastic Syndromes, Acute Lymphoblastic Leukemia, Lymphoma, Non-Hodgkin, Cytopenia, Primary Immune Deficiency Disorder.
What interventions are being tested in trial NCT03301168?
The interventions under investigation include: Rimiducid (DRUG), BPX-501 T cells (BIOLOGICAL).
Who is sponsoring clinical trial NCT03301168?
This trial is sponsored by Bellicum Pharmaceuticals, which has 8 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03301168 being conducted?
This trial has 10 study locations across California, District of Columbia, Georgia, Massachusetts, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Myelodysplastic Syndromes
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03301168's enrollment target sits among peer trials
120 45th of 173 higher than 128 of 173 other Myelodysplastic Syndromes trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Myelodysplastic Syndromes trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
RECRUITING · Phase 1
-
UAB 2419-CD34 Selection Using the Automated CliniMACS Prodigy
RECRUITING · Phase 1
-
A Study of TSC-100 and TSC-101 in AML, ALL and MDS in Patients Undergoing Allogeneic Peripheral Blood Stem Transplantation
RECRUITING · Phase 1
-
HM2023-05: GTB-3650 Trike for High Risk MDS and R/R AML
RECRUITING · Phase 1
-
A Study of GLB-001 in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher Risk Myelodysplastic Syndromes
RECRUITING · Phase 1
-
Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia
RECRUITING · Phase 1
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01174108 · 120 participants · Phase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
- NCT01627301 · 120 participants · NA
Sympathetic Overactivity in Post-traumatic Stress Disorder
- NCT02025023 · 120 participants · Phase 3
Local 5-Fluorouracil Injection for the Treatment of Chalazia: A Prospective, Comparative Study
- NCT02128906 · 120 participants · Phase 2
Radiotherapy With Cisplatin vs. Docetaxel-cetuximab in HNSCC: ERCC1 Biomarker Enrichment and Interaction Design
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia