Trial Outcomes & Findings for Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia (NCT NCT02162420)

NCT ID: NCT02162420

Last Updated: 2025-05-08

Results Overview

Incidence of neutrophil engraftment by day 42.

Recruitment status

COMPLETED

Study phase

Target enrollment

61 participants

Primary outcome timeframe

Day 42

Results posted on

2025-05-08

Participant Flow

Participant milestones

Participant milestones
Measure	Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)	Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)	Arm C: Severe Aplastic Anemia, Matched Related Donor Arm C: Severe Aplastic Anemia (matched related donor)	Arm E: Severe Aplastic Anemia, PTCy Platform Arm E: Severe Aplastic Anemia (SAA), PTCy platform
Overall Study STARTED	12	18	23	8
Overall Study COMPLETED	11	15	22	8
Overall Study NOT COMPLETED	1	3	1	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor n=12 Participants Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)	Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor n=18 Participants Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)	Arm C: Severe Aplastic Anemia, Matched Related Donor n=23 Participants Arm C: Severe Aplastic Anemia (matched related donor)	Arm D: Dyskeratosis Congenita, PTCy Platform Arm D: Dyskeratosis Congenita (DKC), PTCy platform	Arm E: Severe Aplastic Anemia, PTCy Platform n=8 Participants Arm E: Severe Aplastic Anemia (SAA), PTCy platform	Total n=61 Participants Total of all reporting groups
Age, Categorical <=18 years	9 Participants n=5 Participants	15 Participants n=7 Participants	13 Participants n=5 Participants	0 Participants n=4 Participants	4 Participants n=21 Participants	41 Participants n=8 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants	2 Participants n=7 Participants	9 Participants n=5 Participants	0 Participants n=4 Participants	4 Participants n=21 Participants	17 Participants n=8 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	3 Participants n=8 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	11 Participants n=7 Participants	10 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants	29 Participants n=8 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	7 Participants n=7 Participants	13 Participants n=5 Participants	0 Participants n=4 Participants	6 Participants n=21 Participants	32 Participants n=8 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	1 Participants n=8 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	11 Participants n=5 Participants	17 Participants n=7 Participants	21 Participants n=5 Participants	0 Participants n=4 Participants	7 Participants n=21 Participants	56 Participants n=8 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants	4 Participants n=8 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	3 Participants n=8 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	6 Participants n=8 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=8 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	3 Participants n=21 Participants	3 Participants n=8 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants	13 Participants n=7 Participants	14 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants	36 Participants n=8 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants	5 Participants n=8 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants	8 Participants n=8 Participants
Region of Enrollment United States	12 participants n=5 Participants	18 participants n=7 Participants	23 participants n=5 Participants	—	8 participants n=21 Participants	61 participants n=8 Participants

PRIMARY outcome

Timeframe: Day 42

Incidence of neutrophil engraftment by day 42.

Outcome measures

Outcome measures
Measure	Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor n=12 Participants Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)	Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor n=18 Participants Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)	Arm C: Severe Aplastic Anemia, Matched Related Donor n=23 Participants Arm C: Severe Aplastic Anemia (matched related donor)	Arm E: Severe Aplastic Anemia, PTCy Platform n=8 Participants Arm E: Severe Aplastic Anemia (SAA), PTCy platform
Incidence of Neutrophil Engraftment	100 Percentage of participants Interval 0.0 to 100.0	100 Percentage of participants Interval 0.0 to 100.0	96 Percentage of participants Interval 82.0 to 100.0	100 Percentage of participants Interval 0.0 to 100.0

PRIMARY outcome

Timeframe: 1 year

Incidence of platelet engraftment at 1 year

Outcome measures

Outcome measures
Measure	Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor n=12 Participants Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)	Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor n=18 Participants Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)	Arm C: Severe Aplastic Anemia, Matched Related Donor n=23 Participants Arm C: Severe Aplastic Anemia (matched related donor)	Arm E: Severe Aplastic Anemia, PTCy Platform n=8 Participants Arm E: Severe Aplastic Anemia (SAA), PTCy platform
Incidence of Platelet Engraftment	100 Percentage of participants Interval 0.0 to 100.0	100 Percentage of participants Interval 0.0 to 100.0	96 Percentage of participants Interval 82.0 to 100.0	100 Percentage of participants Interval 0.0 to 100.0

SECONDARY outcome

Timeframe: Day 100

Incidence of regimen related mortality by day 100.

Outcome measures

Outcome measures
Measure	Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor n=12 Participants Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)	Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor n=18 Participants Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)	Arm C: Severe Aplastic Anemia, Matched Related Donor n=23 Participants Arm C: Severe Aplastic Anemia (matched related donor)	Arm E: Severe Aplastic Anemia, PTCy Platform n=8 Participants Arm E: Severe Aplastic Anemia (SAA), PTCy platform
Incidence of Regimen Related Mortality	0 Percentage of participants Interval 0.0 to 0.0	6 Percentage of participants Interval 1.0 to 33.0	6 Percentage of participants Interval 1.0 to 33.0	0 Percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Day 100

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

Outcome measures

Outcome measures
Measure	Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor n=12 Participants Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)	Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor n=18 Participants Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)	Arm C: Severe Aplastic Anemia, Matched Related Donor n=23 Participants Arm C: Severe Aplastic Anemia (matched related donor)	Arm E: Severe Aplastic Anemia, PTCy Platform n=8 Participants Arm E: Severe Aplastic Anemia (SAA), PTCy platform
Incidence of Acute Graft-versus-host Disease	0 Percentage of participants Interval 0.0 to 0.0	11 Percentage of participants Interval 0.0 to 25.0	9 Percentage of participants Interval 0.0 to 21.0	0 Percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 6 Months

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

Outcome measures

Outcome measures
Measure	Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor n=12 Participants Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)	Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor n=18 Participants Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)	Arm C: Severe Aplastic Anemia, Matched Related Donor n=23 Participants Arm C: Severe Aplastic Anemia (matched related donor)	Arm E: Severe Aplastic Anemia, PTCy Platform n=8 Participants Arm E: Severe Aplastic Anemia (SAA), PTCy platform
Incidence of Chronic Graft-versus-host Disease	0 Percentage of participants Interval 0.0 to 0.0	6 Percentage of participants Interval 0.0 to 16.0	0 Percentage of participants Interval 0.0 to 0.0	0 Percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 1 Year

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

Outcome measures

Outcome measures
Measure	Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor n=12 Participants Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)	Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor n=18 Participants Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)	Arm C: Severe Aplastic Anemia, Matched Related Donor n=23 Participants Arm C: Severe Aplastic Anemia (matched related donor)	Arm E: Severe Aplastic Anemia, PTCy Platform n=8 Participants Arm E: Severe Aplastic Anemia (SAA), PTCy platform
Incidence of Chronic Graft-versus-host Disease	0 Percentage of participants Interval 0.0 to 0.0	6 Percentage of participants Interval 0.0 to 16.0	0 Percentage of participants Interval 0.0 to 0.0	0 Percentage of participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 1 Year

Incidence of secondary malingancies