NCT02179359

Brief Summary

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
38

participants targeted

Target at P25-P50 for not_applicable

Timeline
Completed

Started Sep 2014

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
terminated

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 27, 2014

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 1, 2014

Completed
2 months until next milestone

Study Start

First participant enrolled

September 2, 2014

Completed
10.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 21, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 21, 2024

Completed
Last Updated

March 3, 2025

Status Verified

February 1, 2025

Enrollment Period

10.2 years

First QC Date

June 27, 2014

Last Update Submit

February 27, 2025

Conditions

Sickle Cell DiseaseTransfusion Dependent Alpha- or Beta- ThalassemiaDiamond Blackfan AnemiaParoxysmal Nocturnal HemoglobinuriaGlanzmann ThrombastheniaSevere Congenital NeutropeniaShwachman-Diamond SyndromeNon-Malignant Hematologic Disorders

Keywords

Stem Cell Transplant

Outcome Measures

Primary Outcomes (1)

  • incidence of graft failure

    42 days

Secondary Outcomes (2)

  • overall survival

    6 months, 1 and 2 years

  • disease free survival

    6 months, 1 and 2 years

Study Arms (3)

Reduced Toxicity Ablative Regimen

EXPERIMENTAL

For use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are \<12 years and/or have mild/moderate iron exposure.

Drug: Reduced Toxicity Ablative Regimen

Reduced Intensity Preparative Regimen

EXPERIMENTAL

For use in patients with unrelated donor bone marrow and for DBA patients who are \>12 years and/or have significant iron exposure.

Drug: Reduced Intensity Preparative Regimen

Myeloablative Preparative Regimen

EXPERIMENTAL

For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.

Drug: Myeloablative Preparative Regimen

Interventions

Reduced Toxicity Ablative RegimenDRUG

* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan * Stem Cell Infusion Day 0

Reduced Toxicity Ablative Regimen
Reduced Intensity Preparative RegimenDRUG

* Alemtuzumab * Cyclophosphamide * Fludarabine * Total Body Irradiation (TBI) * Stem Cell Infusion Day 0

Reduced Intensity Preparative Regimen
Myeloablative Preparative RegimenDRUG

* Alemtuzumab * Cyclophosphamide * Busulfan * Stem Cell Infusion Day 0

Myeloablative Preparative Regimen

Eligibility Criteria

AgeUp to 55 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
  • Acceptable stem cell source identified
  • Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
  • Creatinine \<2.0 mg/dl for adults or glomerular filtration rate \> 50 ml/min for children
  • Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase \<5 times the upper limit of institutional normal
  • Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \> 40%

You may not qualify if:

  • active, uncontrolled infection
  • pregnant or breastfeeding
  • HIV positive

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Minnesota Medical Center, Fairview

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cellbeta-ThalassemiaAnemia, Diamond-BlackfanHemoglobinuria, ParoxysmalThrombastheniaNeutropenia, Severe Congenital, Autosomal Recessive 3Shwachman-Diamond Syndrome

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesThalassemiaAnemia, Hypoplastic, CongenitalAnemia, AplasticRed-Cell Aplasia, PureCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesMyelodysplastic SyndromesBlood Coagulation Disorders, InheritedBlood Coagulation DisordersBlood Platelet DisordersHemorrhagic DisordersExocrine Pancreatic InsufficiencyPancreatic DiseasesDigestive System DiseasesLipid Metabolism, Inborn ErrorsLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesLipomatosis

Study Officials

  • Ashish Gupta, MBBS, MPH

    Masonic Cancer Center, University of Minnesota

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 27, 2014

First Posted

July 1, 2014

Study Start

September 2, 2014

Primary Completion

November 21, 2024

Study Completion

November 21, 2024

Last Updated

March 3, 2025

Record last verified: 2025-02

Locations

US(1)