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Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

NCT02162420 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 61

Last updated 2025-05-08

Study results available
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Summary

Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type.

Conditions

  • Dyskeratosis Congenita
  • Aplastic Anemia

Interventions

DRUG

Alemtuzumab

Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.

DRUG

Fludarabine

Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.

DRUG

Cyclophosphamide

Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.

RADIATION

Total Body Irradiation

TBI 200 cGy as a single fraction on day -1 from transplant.

BIOLOGICAL

Stem Cell Transplant

Stem cell transplant on day 0.

DRUG

Anti-thymocyte globulin

ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant.

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-01-10
Primary Completion
2024-08-08
Completion
2025-03-11

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02162420 on ClinicalTrials.gov