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NCT00977691 · ClinicalTrials.gov registry record · Phase 1
Haploidentical PBMC Transplant for Severe Congenital Anemias
A Phase 1 study of Sickle Cell Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Active
- Registry status
- Phase 1
- Development phase
- 23
- Enrollment target
- 1
- Study location
NCT00977691: Active Phase 1 study of Sickle Cell Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT00977691 is a Phase 1 study of Sickle Cell Anemia that is active but no longer recruiting, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 23 participants, below the 100-participant average among 22 other Sickle Cell Anemia trials with a reported enrollment target (77% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00977691, a Phase 1 study of Sickle Cell Anemia, is active but no longer recruiting, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 23 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias. BMT usually involves high-intensity treatment with chemotherapy and radiation to kill abnormal cells, which affects all systems of the body. People with anemias often have damage to other organs such as the kidneys, which can be further damaged by the chemotherapy. Only approximately 20 percent of patients have a full-matched donor, making treatment for many people with anemias unavailable. However, 90 percent of patients may have a half-matched donor, but using a half-matched donor increases the toxicity of BMT. Objectives: To determine if a research BMT with half-matched donor cells, low-intensity radiation, immunosuppressant drugs, and no chemotherapy will be effective in patients with sickle cell disease and Beta-thalassemia. To determine the effectiveness of cyclophosphamide, an immunosuppressant drug, in preventing rejection of the donor cells. Eligibility: Recipients are individuals at least 18 years of age who have been diagnosed with sickle cell disease and Beta-thalassemia, and who have a family member who is a haploidentical (i.e., half match) tissue match. Donors are healthy individuals between the ages of 2 and 80 who are found to be suitable donors. Design: Donors will undergo apheresis, which involves withdrawing blood from one arm vein, passing it through a machine that removes bone marrow stem cells, and returning the remaining blood through the vein in the other arm. Donors will receive a drug that causes the stem cells to be released into the bloodstream prior to the apheresis procedure. Recipients will undergo routine physical and laboratory examinations, including bone marrow sampling at the beginning of the study. After transplantation, physical and laboratory examinations will occur on a weekly or twice weekly basis at the outpatient clinic. Recipients will be examined every 6 months
Primary Outcome
Percentage of patients post transplant with sustained donor type hemoglobin on hemoglobin electrophoresis
Conditions Studied
Interventions
- DRUG Cyclophosphamide
- DRUG Sirolimus
- DRUG Alemtuzumab
- PROCEDURE PBSC Transplant
- PROCEDURE Low Dose Irradiation
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center, 9000 Rockville Pike - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 23 participants |
| Start Date | 2009-12-14 |
| Est. Completion | 2026-09-10 |
| Phase | Phase 1 |
What the registry record for NCT00977691 still lists
NCT00977691 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 23 participants, a relatively small participant target, below the 100-participant average among 22 other Sickle Cell Anemia trials with a reported enrollment target (77% lower).
The record links to 1 condition, with Sickle Cell Anemia appearing as the primary indexed condition, and to 5 interventions - of which Cyclophosphamide is the first listed.
NCT00977691 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT00977691 about?
NCT00977691 is a clinical study titled "Haploidentical PBMC Transplant for Severe Congenital Anemias". Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias. BMT usually involves high-intensity treatment with chemotherapy and radiation to kill abnormal cells, which affects all systems of the body. People...
What is the current status of trial NCT00977691?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 23 participants. The study started on 2009-12-14. Estimated completion is 2026-09-10.
What conditions does trial NCT00977691 study?
This clinical trial studies the following conditions: Sickle Cell Anemia.
What interventions are being tested in trial NCT00977691?
The interventions under investigation include: Cyclophosphamide (DRUG), Sirolimus (DRUG), Alemtuzumab (DRUG), PBSC Transplant (PROCEDURE), Low Dose Irradiation (PROCEDURE).
Who is sponsoring clinical trial NCT00977691?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00977691 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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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 NCT00977691's enrollment target sits among peer trials
23 19th of 22 higher than 3 of 22 other Sickle Cell Anemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Anemia 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.
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