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NCT00427661 · ClinicalTrials.gov registry record · NA
A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen
A NA study, sponsored by University of Pittsburgh.
- Completed
- Registry status
- NA
- Development phase
- 8
- Enrollment target
NCT00427661: Completed NA study, sponsored by University of Pittsburgh.
NCT00427661 is a NA study that has completed, run by University of Pittsburgh. The registered enrollment target is 8 participants, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (99% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00427661, a NA study, has completed, sponsored by University of Pittsburgh.
- COMPLETED
- Registry status
- NA
- Development phase
- 8 participants
- Enrollment target
Study Summary
Hypothesis 1: A novel nonmyeloablative condition regimen will be safe and efficacious in producing stable donor chimerism and cure of severe hemoglobinopathy. Hypothesis 2: Stable donor chimerism will result in amelioration of cerebral vasculopathy, improved cerebral perfusion and neurocognitive function. Specific Aim 1: Study the safety and efficacy of a novel non-toxic conditioning regimen for HSCT for patients with severe hemoglobinopathies and the kinetics of lineage specific chimerism after HSCT We will test our hypothesis that a novel nonmyeloablative condition regimen will be safe and efficacious in producing stable donor chimerism and cure of severe hemoglobinopathy: Specific Aim 2: Optimize the immunosuppressive regimen for HSCT patients through a thorough understanding of the pharmacokinetics of Busulfan (BU) and mycophenolate mofetil (MMF) in the patient population. This will involve: 1. Determine the pharmacokinetics of intravenously and orally administered MMF and intravenous BU in patients receiving HSCT. 2. Determine the relationship of Area under the curve (AUC) of BU and mean trough concentrations of mycophenolic acid (MPA) to engraftment and graft versus host disease (GVHD). 3. Determine the relationship of Area under the curve (AUC) and steady state concentration of BU to engraftment at day 30 and 1 year post HSCT. Specific Aim 3: Study the effect of complete or partial donor chimerism on silent and overt cerebral vasculopathy, and neurocognitive functioning in patients with SCD undergoing HSCT. We will test our hypothesis that stable donor chimerism will result in improvement in cerebral vasculopathy and neurocognitive function. This will include. 1. Determine effect of transplantation silent and overt cerebral vasculopathy by comparison MRA and TCD 1 year after HSCT to pre-HSCT studies. 2. Determine effect on HSCT on neurocognitive function. Specific Aim 4: To determine the rate of T cell immune reconstitution in children with sickle cell
Primary Outcome
GVHD is assessed by physical exam, bloodwork and biopsy.
Interventions
- OTHER Busulfan; Fludarabine; cyclosporine A and MMF
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2002-06 |
| Est. Completion | 2014-05 |
| Phase | NA |
What the finished NCT00427661 record still lists
NCT00427661 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 participants, a relatively small participant target, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (99% lower).
The record links to 0 conditions, and to 1 intervention - of which Busulfan; Fludarabine; cyclosporine A and MMF is the first listed.
NCT00427661 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00427661 about?
NCT00427661 is a clinical study titled "A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen". Hypothesis 1: A novel nonmyeloablative condition regimen will be safe and efficacious in producing stable donor chimerism and cure of severe hemoglobinopathy. Hypothesis 2: Stable donor chimerism will result in amelioration of cerebral vasculopathy, improved cerebral perfusion and neurocognitive fu...
What is the current status of trial NCT00427661?
This trial is currently completed. It is a NA study. The enrollment target is 8 participants. The study started on 2002-06. Estimated completion is 2014-05.
What interventions are being tested in trial NCT00427661?
The interventions under investigation include: Busulfan; Fludarabine; cyclosporine A and MMF (OTHER).
Who is sponsoring clinical trial NCT00427661?
This trial is sponsored by University of Pittsburgh, which has 1,238 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00427661's enrollment target sits among peer trials
8 1976th of 2000 higher than 19 of 2,000 other NA trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other NA 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
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