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NCT02162420

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Completed NA Results posted Last updated 8 May 2025
What this trial tests

NA trial testing Alemtuzumab in Dyskeratosis Congenita in 61 participants. Completed in 11 March 2025.

Timeline
10 January 2015
Primary endpoint
8 August 2024
11 March 2025

Quick facts

Lead sponsorMasonic Cancer Center, University of Minnesota
PhaseNA
StatusCompleted
Study typeINTERVENTIONAL
Allocationnon randomized
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment61
Start date10 January 2015
Primary completion8 August 2024
Estimated completion11 March 2025
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Masonic Cancer Center, University of Minnesota

Who can join

Adults 0 to 70, any sex, with Dyskeratosis Congenita or Aplastic Anemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Incidence of Neutrophil Engraftment Primary · Day 42

Incidence of neutrophil engraftment by day 42.

GroupValue95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor1000 – 100
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor1000 – 100
Arm C: Severe Aplastic Anemia, Matched Related Donor9682 – 100
Arm E: Severe Aplastic Anemia, PTCy Platform1000 – 100
Incidence of Platelet Engraftment Primary · 1 year

Incidence of platelet engraftment at 1 year

GroupValue95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor1000 – 100
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor1000 – 100
Arm C: Severe Aplastic Anemia, Matched Related Donor9682 – 100
Arm E: Severe Aplastic Anemia, Haploidentical Donor1000 – 100
Incidence of Regimen Related Mortality Secondary · Day 100

Incidence of regimen related mortality by day 100.

GroupValue95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor00 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor61 – 33
Arm C: Severe Aplastic Anemia, Matched Related Donor61 – 33
Arm E: Severe Aplastic Anemia, PTCy Platform00 – 0
Incidence of Acute Graft-versus-host Disease Secondary · Day 100

Incidence of acute graft-versus-host disease by day 100.

GroupValue95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor00 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor110 – 25
Arm C: Severe Aplastic Anemia, Matched Related Donor90 – 21
Arm E: Severe Aplastic Anemia, PTCy Platform00 – 0
Incidence of Chronic Graft-versus-host Disease Secondary · 6 Months

Incidence of chronic graft-versus-host disease by 6 months

GroupValue95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor00 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor60 – 16
Arm C: Severe Aplastic Anemia, Matched Related Donor00 – 0
Arm E: Severe Aplastic Anemia, PTCy Platform00 – 0
Incidence of Chronic Graft-versus-host Disease Secondary · 1 Year

Incidence of chronic graft-versus-host disease by 1 year

GroupValue95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor00 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor60 – 16
Arm C: Severe Aplastic Anemia, Matched Related Donor00 – 0
Arm E: Severe Aplastic Anemia, PTCy Platform00 – 0
Incidence of Secondary Malignancies Secondary · 1 Year

Incidence of secondary malingancies

GroupValue95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor00 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor00 – 0
Arm C: Severe Aplastic Anemia, Matched Related Donor00 – 0
Arm E: Severe Aplastic Anemia, PTCy Platform00 – 0

Sponsor's own description

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

Verify or expand the search:

Other trials of Alemtuzumab

Trials testing the same drug.

Other recruiting trials for Dyskeratosis Congenita

Currently open trials in the same condition.

Other Masonic Cancer Center, University of Minnesota trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02162420.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing