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 EngraftmentPrimary· Day 42
Incidence of neutrophil engraftment by day 42.
Group
Value
95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor
100
0 – 100
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor
100
0 – 100
Arm C: Severe Aplastic Anemia, Matched Related Donor
96
82 – 100
Arm E: Severe Aplastic Anemia, PTCy Platform
100
0 – 100
Incidence of Platelet EngraftmentPrimary· 1 year
Incidence of platelet engraftment at 1 year
Group
Value
95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor
100
0 – 100
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor
100
0 – 100
Arm C: Severe Aplastic Anemia, Matched Related Donor
96
82 – 100
Arm E: Severe Aplastic Anemia, Haploidentical Donor
100
0 – 100
Incidence of Regimen Related MortalitySecondary· Day 100
Incidence of regimen related mortality by day 100.
Group
Value
95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor
0
0 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor
6
1 – 33
Arm C: Severe Aplastic Anemia, Matched Related Donor
6
1 – 33
Arm E: Severe Aplastic Anemia, PTCy Platform
0
0 – 0
Incidence of Acute Graft-versus-host DiseaseSecondary· Day 100
Incidence of acute graft-versus-host disease by day 100.
Group
Value
95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor
0
0 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor
11
0 – 25
Arm C: Severe Aplastic Anemia, Matched Related Donor
9
0 – 21
Arm E: Severe Aplastic Anemia, PTCy Platform
0
0 – 0
Incidence of Chronic Graft-versus-host DiseaseSecondary· 6 Months
Incidence of chronic graft-versus-host disease by 6 months
Group
Value
95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor
0
0 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor
6
0 – 16
Arm C: Severe Aplastic Anemia, Matched Related Donor
0
0 – 0
Arm E: Severe Aplastic Anemia, PTCy Platform
0
0 – 0
Incidence of Chronic Graft-versus-host DiseaseSecondary· 1 Year
Incidence of chronic graft-versus-host disease by 1 year
Group
Value
95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor
0
0 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor
6
0 – 16
Arm C: Severe Aplastic Anemia, Matched Related Donor
0
0 – 0
Arm E: Severe Aplastic Anemia, PTCy Platform
0
0 – 0
Incidence of Secondary MalignanciesSecondary· 1 Year
Incidence of secondary malingancies
Group
Value
95% CI
Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor
0
0 – 0
Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor
0
0 – 0
Arm C: Severe Aplastic Anemia, Matched Related Donor
0
0 – 0
Arm E: Severe Aplastic Anemia, PTCy Platform
0
0 – 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.
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Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Masonic Cancer Center, University of Minnesota
Last refreshed: 8 May 2025
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.