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NCT00145587TERMINATEDanonymous

Allogeneic Hematopoietic Stem Cell Transplantation for Children Affected With Malignant Osteopetrosis: A Pilot Study

Sponsor

Source record

St. Jude Children's Research Hospital

Phase

Source record

NA

Modality

AI-normalized

cell therapy

Target

AI-normalized

Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies

Indication / condition

AI-normalized

Osteopetrosis

Intervention

Source record

Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies

Source & freshness

Source record

NCT ID

NCT00145587

Original source

ClinicalTrials.gov

Source last updated

May 30, 2017

Ingested at

Jun 19, 2026

Internal sync

Jun 19, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

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NCT ID

NCT00145587

Title

Allogeneic Hematopoietic Stem Cell Transplantation for Children Affected With Malignant Osteopetrosis: A Pilot Study

Sponsor

St. Jude Children's Research Hospital

Status

TERMINATED

Phase

NA

Condition raw

Osteopetrosis

Condition normalized

Osteopetrosis

Modality raw

cell therapy

Modality normalized

cell therapy

Target raw

Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies

Target normalized

Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies

Interventions

Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies

Public preview

Source record

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.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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