NCT01881334

Brief Summary

The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants who are at a higher risk of not engrafting in a safe amount of time. The purpose of the related stem cells is to give the bone marrow a "jump start" towards recovery. Ultimately, the cord blood cells will grow and permanently rescue the bone marrow.

Trial Health

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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 17, 2013

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 19, 2013

Completed
Last Updated

November 20, 2025

Status Verified

November 1, 2025

First QC Date

June 17, 2013

Last Update Submit

November 17, 2025

Conditions

Hematologic MalignanciesInborn Errors of Metabolism DisordersImmune Deficiencies

Keywords

Haploidentical DonorT-cell depleted Stem CellsAllogeneic TransplantUmbilical Cord Blood DonorHigh Risk MalignanciesMetabolic DisordersImmune DeficiencyAcute Lymphoblastic LeukemiaAcute Myelogenous LeukemiaMyelodysplastic SyndromeALLAMLMDSCGDSCIDAdrenoleukodystrophyMetachromaticleukodystrophyKrabbePMDHunter'sHurler'sSevere Aplastic AnemiaLymphomaSickle Cell DiseaseThalassemia

Interventions

CliniMACS CD34 Reagent SystemBIOLOGICAL

The CliniMACS CD34 Reagent System is a medical device that is used in vitro to select and enrich CD34+ cells from heterogeneous hematologic cell populations for transplantation in cases where this is clinically indicated.

Eligibility Criteria

AgeUp to 65 Years
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Have a consenting related haplo-identical (3/6, 4/6, or 5/6 if DRB1 mismatch) stem cell donor.
  • Have one or two available 4, 5, or 6/6 antigen matching unrelated UCB unit(s) that will deliver a total cell dose \>3.0 x 10e7 cells/kg. Patients who do not have a single UCB unit that will deliver the minimum required cell dose, two partially HLA-matched UCB units which together meet the minimum cell dose requirement, can be used for 1 transplant. These units must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient, and HLA-matched at 3 of 6 HLA- A, B, DRB1 loci with each other (using same resolution of HLA typing as indicated above). There is no limitation on maximum cell dose.
  • Have a high risk or refractory malignancy, or non-malignant disorder amenable to stem cell transplantation therapy.
  • Meet eligibility requirements for allogeneic transplant per institutional standard practices.
  • Have given written informed consent according to FDA guidelines (or consent of parent/legal guardian as applicable).
  • Be \<65 years of age at the time of study enrollment.

You may not qualify if:

  • Have a consenting 8/8 or 10/10 allele matched, consenting, related or unrelated hematopoietic stem cell transplant (HSCT) donor.
  • Have a life expectancy of less than 3 months.
  • Have uncontrolled infections at time of cytoreduction.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

AVAILABLE

MeSH Terms

Conditions

Hematologic NeoplasmsMetabolic DiseasesImmunologic Deficiency SyndromesPrecursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Myeloid, AcuteMyelodysplastic SyndromesAdrenoleukodystrophyAnemia, AplasticLymphomaAnemia, Sickle CellThalassemia

Condition Hierarchy (Ancestors)

Neoplasms by SiteNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesNutritional and Metabolic DiseasesImmune System DiseasesLeukemia, LymphoidLeukemiaNeoplasms by Histologic TypeLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersLeukemia, MyeloidBone Marrow DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesHereditary Central Nervous System Demyelinating DiseasesLeukoencephalopathiesDemyelinating DiseasesX-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHeredodegenerative Disorders, Nervous SystemMetabolism, Inborn ErrorsPeroxisomal DisordersAdrenal InsufficiencyAdrenal Gland DiseasesEndocrine System DiseasesAnemiaBone Marrow Failure DisordersAnemia, Hemolytic, CongenitalAnemia, HemolyticHemoglobinopathies

Study Officials

  • Joanne Kurtzberg, MD

    Duke University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Erin Arbuckle

CONTACT

919-684-3293erin.arbuckle@duke.edu

Study Design

Study Type
expanded access
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

June 17, 2013

First Posted

June 19, 2013

Last Updated

November 20, 2025

Record last verified: 2025-11

Locations

US(1)