Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03226691 · ClinicalTrials.gov registry record · Phase 1
Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients
A Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
NCT03226691: Completed Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT03226691 is a Phase 1 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 15 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03226691, a Phase 1 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
Study Summary
The constitution of blood relies upon hematopoietic stem cells (HSCs), which stay in the bone marrow and differentiate to all lineages of peripheral blood cells. HSC transplantation is the only curative option currently available for sickle cell disease (SCD) patients either via allogeneic HSC transplantation or HSC-targeted gene therapy. Granulocyte-colony stimulating factor (G-CSF)- mobilized HSCs are frequently utilized in the adult setting of HSC transplantation because of the faster hematologic recovery as compared to bone marrow. As an autologous HSC source for gene therapy, bone marrow harvest has been generally employed since G-CSF has been prohibitive in SCD patients due to granulocyte stimulation and the associated reports of vaso-occlusive crises, multi-organ failure, and death. However, when bone marrow harvest is used, the amounts of collected cells are limited and anesthesia is required. In order to obtain HSCs in large numbers without anesthesia, patients will undergo mobilization followed by large volume apheresis. Plerixafor is an alternative treatment for mobilization without direct stimulation to granulocytes, and it is theoretically applicable for SCD patients. The primary endpoint of this study is to obtain sufficient amounts of HSCs collected from the peripheral blood in SCD patients after plerixafor mobilization with an acceptable safety profile. The harvested products will be stored as backup for patients undergoing gene therapy as well as allogeneic HSC transplantation.
Primary Outcome
Sufficient collection of HSCs (target 2.0x106 CD34+ cells/kg) from the PB after plerixafor mobilization without serious adverse events (SAEs)
Interventions
- DRUG Plerixafor
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2017-07-25 |
| Est. Completion | 2019-02-27 |
| Phase | Phase 1 |
What the finished NCT03226691 record still lists
NCT03226691 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower).
The record links to 0 conditions, and to 1 intervention - of which Plerixafor is the first listed.
NCT03226691 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03226691 about?
NCT03226691 is a clinical study titled "Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients". The constitution of blood relies upon hematopoietic stem cells (HSCs), which stay in the bone marrow and differentiate to all lineages of peripheral blood cells. HSC transplantation is the only curative option currently available for sickle cell disease (SCD) patients either via allogeneic HSC trans...
What is the current status of trial NCT03226691?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2017-07-25. Estimated completion is 2019-02-27.
What interventions are being tested in trial NCT03226691?
The interventions under investigation include: Plerixafor (DRUG).
Who is sponsoring clinical trial NCT03226691?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03226691's enrollment target sits among peer trials
15 1798th of 2000 higher than 157 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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
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
- NCT01889381 · 15 participants · Phase 2
Human Craniomaxillofacial Allotransplantation
- NCT02530073 · 15 participants · NA
Trial of Fetoscopic Endoluminal Tracheal Occlusion (FETO) for CDH
- NCT02535936 · 15 participants · NA
Cortical Plasticity in Spastic Diplegia After Selective Dorsal Rhizotomy
- NCT02918474 · 15 participants · NA
Decision Making Tool in Supporting Decision Making in Contralateral Prophylactic Mastectomy in Patients With Newly Diagnosed Breast Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI