Trial Outcomes & Findings for Stem Cell Transplant for Bone Marrow Failure Syndromes (NCT NCT00176878)

Results Overview

Calculated from day 1 of transplant to last contact.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

10 participants

Primary outcome timeframe

2 years

Results posted on

2017-12-28

Participant Flow

Participant milestones

Participant milestones
Measure	Bone Marrow Failure Patients All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplant for Bone Marrow Failure Syndromes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bone Marrow Failure Patients n=10 Participants All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Age, Categorical <=18 years	10 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	4.8 years STANDARD_DEVIATION 7.6 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Calculated from day 1 of transplant to last contact.

Outcome measures

Outcome measures
Measure	Bone Marrow Failure Patients n=10 Participants All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Number of Patients Alive (Survival) at 2 Years	6 Participants

SECONDARY outcome

Timeframe: 3 years

Number of subjects who survived 3 years post-transplant.

Outcome measures

Outcome measures
Measure	Bone Marrow Failure Patients n=10 Participants All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Number of Patients Alive at Three Years (Survival)	6 Participants

SECONDARY outcome

Timeframe: 42 Days

Number of patients who received non-genotypic identical marrow or cord blood cells using a "non-myeloablative" preparative regimen and exhibited engraftment at Day 42.

Outcome measures

Outcome measures
Measure	Bone Marrow Failure Patients n=10 Participants All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Number of Patients With Succcessful Engraftment After Transplantation	10 Participants

SECONDARY outcome

Timeframe: 100 Days

Number of patients with Grade 2, 3 and 4 Acute (normally observed within the first 100 days) Graft Versus Host Disease. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of 1 to a high of 4. Patients with grade IV GVHD usually have a poor prognosis. Grade 2 = moderate, Grade 3 = severe, Grade 4 = life threatening.

Outcome measures

Outcome measures
Measure	Bone Marrow Failure Patients n=10 Participants All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Number of Patients With Grade 2-4 Acute Graft Versus Host Disease	5 Participants

SECONDARY outcome

Timeframe: 2 years

Number of patients who exhibited chronic (normally occurs after 100 days) Graft Versus Host Disease at 2 years post transplant. Chronic graft-versus-host-disease, over its long-term course, can also cause damage to the connective tissue and exocrine glands.

Outcome measures

Outcome measures
Measure	Bone Marrow Failure Patients n=10 Participants All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Number of Patients With Chronic Graft Versus Host Disease	3 Participants

SECONDARY outcome

Timeframe: 2 years

Number of patients who exhibited disease recurrence at 2 years.

Outcome measures

Outcome measures
Measure	Bone Marrow Failure Patients n=10 Participants All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Number of Patients With Disease Recurrence	0 Participants

Adverse Events

Bone Marrow Failure Patients

Serious events: 5 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bone Marrow Failure Patients n=10 participants at risk All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Blood and lymphatic system disorders Bone marrow graft failure	30.0% 3/10 • Number of events 3 • Day 1 of study up through 1 year Only serious adverse events are reported. Other adverse events were not collected.
General disorders Death	30.0% 3/10 • Number of events 3 • Day 1 of study up through 1 year Only serious adverse events are reported. Other adverse events were not collected.
Blood and lymphatic system disorders Graft-versus-host disease (acute)	10.0% 1/10 • Number of events 1 • Day 1 of study up through 1 year Only serious adverse events are reported. Other adverse events were not collected.

Other adverse events

Adverse event data not reported

Additional Information

Paul Orchard, M.D.

Masonic Cancer Center, University of Minnesota

Phone: 612-626-2313

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place