Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
NCT ID: NCT01461837
Last Updated: 2025-08-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
21 participants
INTERVENTIONAL
2012-01-31
2026-12-31
Brief Summary
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Funding Source - FDA OOPD
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Detailed Description
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Patients 2-20.99 years of age with a diagnosis of high-risk SCD and with an unaffected HLA partially matched family donor and meeting eligibility criteria (inclusion and exclusion criteria) are eligible.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Haplo Stem Cell Transplantation
CD34 selected T-cell depleted allogeneic SCT
CD34 selected T-cell depleted allogeneic SCT
Hydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11; fludarabine (30 mg/m2) Days -17, -16, -15, -14, -13; busulfan (3.2 mg/kg/day) Days -12, -11, -10, -9; thiotepa (10 mg/kg IV) day -8; cyclophosphamide (50 mg/kg) Days -7, -6, -5, -4; TLI on day -3; rabbit ATG (2.0 mg/kg/day) day -5,-4,-3, and -2; Stem Cell infusion day 0
Interventions
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CD34 selected T-cell depleted allogeneic SCT
Hydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11; fludarabine (30 mg/m2) Days -17, -16, -15, -14, -13; busulfan (3.2 mg/kg/day) Days -12, -11, -10, -9; thiotepa (10 mg/kg IV) day -8; cyclophosphamide (50 mg/kg) Days -7, -6, -5, -4; TLI on day -3; rabbit ATG (2.0 mg/kg/day) day -5,-4,-3, and -2; Stem Cell infusion day 0
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patients must demonstrate one or more of the following Sickle Cell Disease Complications
1. Clinically significant neurologic event (stroke) or any neurologic deficit lasting \>24 hours that is accompanied by an infarct on cerebral MRI
2. Minimum of two episodes of acute chest syndrome.
3. Recurrent painful events (at least 3 in the 2 years prior to enrollment).
4. Abnormal TCD study requiring starting on chronic transfusion therapy.
5. At least one silent infarct lesion on a MRI scan of the head.
* A familial haploidentical donor without homozygous sickle cell disease
* Adequate organ function (renal, liver, cardiac and pulmonary function)
* Karnofsky or Lansky (age appropriate) Performance Score ≥50%
* Liver biopsy is optional to assess for iron overload in chronically transfused patients.
Exclusion Criteria
* SCD Patients with documented uncontrolled infection
* SCD patients who have an unaffected HLA matched family donor willing to proceed to donation
* Karnofsky/Lansky (age appropriate) Performance Score \<50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
* Demonstrated lack of compliance with medical care.
* Clinically significant fibrosis or cirrhosis of the liver
* Previously received a HSCT
2 Years
20 Years
ALL
No
Sponsors
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UCSF Benioff Children's Hospital Oakland
OTHER
Medical College of Wisconsin
OTHER
Washington University School of Medicine
OTHER
Tufts Medical Center
OTHER
University of California, San Francisco
OTHER
University of California, Los Angeles
OTHER
Miltenyi Biomedicine GmbH
INDUSTRY
Ann & Robert H Lurie Children's Hospital of Chicago
OTHER
New York Medical College
OTHER
Responsible Party
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Mitchell Cairo
Principal Investigator
Principal Investigators
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Mitchell S Cairo, MD
Role: PRINCIPAL_INVESTIGATOR
New York Medical College
Locations
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University of California Los Angeles (UCLA)
Los Angeles, California, United States
Children's Hospital and Research Center Oakland
Oakland, California, United States
Lurie Children's Hospital
Chicago, Illinois, United States
Washington University/St. Louis Children's Hospital
St Louis, Missouri, United States
New York Medical College
Valhalla, New York, United States
Medical College of Wisconsin/Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Countries
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References
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Braniecki S, Vichinsky E, Walters MC, Shenoy S, Shi Q, Moore TB, Talano JA, Parsons SK, Flower A, Panarella A, Fabricatore S, Morris E, Mahanti H, Milner J, McKinstry RC, Duncan CN, van de Ven C, Cairo MS. Neurocognitive outcome in children with sickle cell disease after myeloimmunoablative conditioning and haploidentical hematopoietic stem cell transplantation: a non-randomized clinical trial. Front Neurol. 2024 May 22;15:1263373. doi: 10.3389/fneur.2024.1263373. eCollection 2024.
Parsons SK, Rodday AM, Weidner RA, Morris E, Braniecki S, Shenoy S, Talano JA, Moore TB, Panarella A, Flower A, Milner J, Fabricatore S, Mahanti H, van de Ven C, Shi Q, Cairo MS. Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant. Bone Marrow Transplant. 2022 Apr;57(4):586-592. doi: 10.1038/s41409-022-01584-y. Epub 2022 Feb 2.
Related Links
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Consortium website for Parent-to-Child (haplo) Bone Marrow Transplant for Sickle Cell Disease (SCD)
Other Identifiers
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FD-R-0004090
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
NYMC526-4090
Identifier Type: -
Identifier Source: org_study_id
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