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NCT00145587 · ClinicalTrials.gov registry record · NA

Stem Cell Transplantation for Children Affected With Osteopetrosis

A NA study, sponsored by St. Jude Children's Research Hospital.

Terminated
Registry status
NA
Development phase
15
Enrollment target

NCT00145587: Clinical Trial NA study, sponsored by St. Jude Children's Research Hospital.

NCT00145587 is a NA study that was terminated before completion, run by St. Jude Children's Research Hospital. The registered enrollment target is 15 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.

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The verdict

NCT00145587, a NA study, was terminated before completion, sponsored by St. Jude Children's Research Hospital.

TERMINATED
Registry status
NA
Development phase
15 participants
Enrollment target

Study Summary

Malignant infantile osteopetrosis (MIOP) is a rare fatal genetic disorder that is characterized by the bone's inability to regulate remodeling. The only curative therapy is hematopoietic stem cell transplantation. Stem cells provided from an HLA identical matched sibling donor is the standard of care, but not feasible for the majority of patients. In addition, due to the potentially rapid progression of this disease, the time to identify a suitable HLA matched unrelated donor is not optimal. Therefore this study is designed to test the hypothesis that children with osteopetrosis can properly engraft hematopoietic stem cells that are donated from a partially matched parental donor, or "haploidentical" stem cell donor that are processed on the investigational device, CliniMACS selection system.

Primary Outcome

To determine the need for blood or platelet transfusions and the presence of donor cells being present in the transplant recipient's bone marrow or peripheral blood by 100 day after transplantation for children with malignant infantile osteopetrosis who have received a haploidentical stem cell graft.

Interventions

  • PROCEDURE Stem Cell Transplantation
  • DEVICE Miltenyi Biotec CliniMACS
  • DRUG Systemic chemotherapy and antibodies

Trial Details

FieldValue
Enrollment Target 15 participants
Start Date 2004-07
Est. Completion 2009-02
Phase NA

Why NCT00145587 stopped before completion

NCT00145587 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 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 3 interventions - of which Stem Cell Transplantation is the first listed.

NCT00145587 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT00145587 about?

NCT00145587 is a clinical study titled "Stem Cell Transplantation for Children Affected With Osteopetrosis". Malignant infantile osteopetrosis (MIOP) is a rare fatal genetic disorder that is characterized by the bone's inability to regulate remodeling. The only curative therapy is hematopoietic stem cell transplantation. Stem cells provided from an HLA identical matched sibling donor is the standard of car...

What is the current status of trial NCT00145587?

This trial is currently terminated. It is a NA study. The enrollment target is 15 participants. The study started on 2004-07. Estimated completion is 2009-02.

What interventions are being tested in trial NCT00145587?

The interventions under investigation include: Stem Cell Transplantation (PROCEDURE), Miltenyi Biotec CliniMACS (DEVICE), Systemic chemotherapy and antibodies (DRUG).

Who is sponsoring clinical trial NCT00145587?

This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT00145587's enrollment target sits among peer trials

15 1881st of 2000 higher than 92 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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Source: ClinicalTrials.gov NCT00145587, the US trial registry maintained by the National Library of Medicine. NCT00145587 (small enrollment · none site footprint · terminated) retrieved and formatted by PlainTrial, see methodology.