Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00730314 · ClinicalTrials.gov registry record · Phase 1
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
A Phase 1 study, sponsored by Children's Hospital Los Angeles.
- Completed
- Registry status
- Phase 1
- Development phase
- 25
- Enrollment target
NCT00730314: Completed Phase 1 study, sponsored by Children's Hospital Los Angeles.
NCT00730314 is a Phase 1 study that has completed, run by Children's Hospital Los Angeles. The registered enrollment target is 25 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (58% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00730314, a Phase 1 study, has completed, sponsored by Children's Hospital Los Angeles.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 25 participants
- Enrollment target
Study Summary
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic diseases of blood cell include: Red blood cell defects e.g. hemoglobinopathies (sickle cell disease and thalassemia), Blackfan-Diamond anemia and congenital or chronic hemolytic anemias; White blood cells defects/immune deficiencies e.g. chronic granulomatous disease, Wiskott-Aldrich syndrome,Osteopetrosis, Kostmann's syndrome (congenital neutropenia), Hereditary Lymphohistiocytosis (HLH); Platelets defects e.g.Congenital amegakaryocytic thrombocytopenia; Metabolic/storage disorders e.g. leukodystrophies,mucopolysaccharidoses as Hurler disease;Stem cell defects e.g.reticular agenesis, among many other rare similar conditions. The study treatment plan uses a new transplant treatment regimen that aims to try to decrease the acute toxicities and complications associated with the standard treatment plans and to improve outcome The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord blood.
Interventions
- PROCEDURE Hematopoietic stem cell transplantation
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2008-08 |
| Est. Completion | 2015-08 |
| Phase | Phase 1 |
What the finished NCT00730314 record still lists
NCT00730314 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (58% lower).
The record links to 0 conditions, and to 1 intervention - of which Hematopoietic stem cell transplantation is the first listed.
NCT00730314 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00730314 about?
NCT00730314 is a clinical study titled "Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells". This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic diseases of blood cell include: Red blood cell defects e.g. hemoglobinopathies (sickle cell disease and thalassemia), Bla...
What is the current status of trial NCT00730314?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 25 participants. The study started on 2008-08. Estimated completion is 2015-08.
What interventions are being tested in trial NCT00730314?
The interventions under investigation include: Hematopoietic stem cell transplantation (PROCEDURE).
Who is sponsoring clinical trial NCT00730314?
This trial is sponsored by Children's Hospital Los Angeles, which has 119 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00730314's enrollment target sits among peer trials
25 1495th of 2000 higher than 481 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
- NCT01166854 · 25 participants
Characterization of Familial Myopathy and Paget Disease of Bone
- NCT01913106 · 25 participants · Phase 1
HSV-tk + Valacyclovir Therapy in Combination With Brachytherapy for Recurrent Prostate Cancer
- NCT02138669 · 25 participants · NA
Intacs Prescription Inserts for Keratoconus Patients
- NCT02174549 · 25 participants · Phase 1
Dose-defining Study of Tirapazamine Combined With Embolization in Liver Cancer
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