Trial Outcomes & Findings for Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders (NCT NCT00489281)

NCT ID: NCT00489281

Last Updated: 2019-04-17

Results Overview

Number of participants who died for reasons related to bone marrow transplant.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

43 participants

Primary outcome timeframe

Up to one year

Results posted on

2019-04-17

Participant Flow

One participant was a screen failure.

Participant milestones

Participant milestones
Measure	Transplant - 200 cGy Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 200. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.	Transplant - 400 cGy Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 400. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.
Overall Study STARTED	29	13
Overall Study COMPLETED	29	13
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Transplant - 200 cGy n=29 Participants Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 200. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.	Transplant - 400 cGy n=13 Participants Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 400. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.	Total n=42 Participants Total of all reporting groups
Age, Categorical <=18 years	9 Participants n=99 Participants	6 Participants n=107 Participants	15 Participants n=206 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=99 Participants	7 Participants n=107 Participants	27 Participants n=206 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Continuous	19 years n=99 Participants	21 years n=107 Participants	21 years n=206 Participants
Sex: Female, Male Female	17 Participants n=99 Participants	8 Participants n=107 Participants	25 Participants n=206 Participants
Sex: Female, Male Male	12 Participants n=99 Participants	5 Participants n=107 Participants	17 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	29 Participants n=99 Participants	13 Participants n=107 Participants	42 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	25 Participants n=99 Participants	6 Participants n=107 Participants	31 Participants n=206 Participants
Race (NIH/OMB) White	1 Participants n=99 Participants	2 Participants n=107 Participants	3 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=99 Participants	5 Participants n=107 Participants	8 Participants n=206 Participants
Region of Enrollment United States	29 Participants n=99 Participants	13 Participants n=107 Participants	42 Participants n=206 Participants
Disease Sickle cell disease	25 Participants n=99 Participants	7 Participants n=107 Participants	32 Participants n=206 Participants
Disease Thalassemia	4 Participants n=99 Participants	6 Participants n=107 Participants	10 Participants n=206 Participants

PRIMARY outcome

Timeframe: Up to one year

Number of participants who died for reasons related to bone marrow transplant.

Outcome measures

Outcome measures
Measure	Transplant - 200 cGy n=29 Participants Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 200. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.	Transplant - 400 cGy n=13 Participants Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 400. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.
Transplant-related Mortality	1 Participants	0 Participants

PRIMARY outcome

Timeframe: 2 years

Population: Due to early study termination, data was not collected to assess this outcome measure

Percentage of participants who are alive without relapse.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 30 days

Number of participants with full (95-100%), mixed (5-94%), and no (0-4%) donor cells. Chimerism is reported for unsorted whole blood and T cells.

Outcome measures

Outcome measures
Measure	Transplant - 200 cGy n=29 Participants Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 200. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.	Transplant - 400 cGy n=13 Participants Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 400. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.
Donor Chimerism at 30 Days Whole blood · 95-100%	12 Participants	8 Participants
Donor Chimerism at 30 Days Whole blood · 5-94%	15 Participants	3 Participants
Donor Chimerism at 30 Days Whole blood · 0-4%	1 Participants	1 Participants
Donor Chimerism at 30 Days Whole blood · Unknown or not measured	1 Participants	1 Participants
Donor Chimerism at 30 Days T cells · 95-100%	4 Participants	4 Participants
Donor Chimerism at 30 Days T cells · 5-94%	18 Participants	8 Participants
Donor Chimerism at 30 Days T cells · 0-4%	5 Participants	0 Participants
Donor Chimerism at 30 Days T cells · Unknown or not measured	2 Participants	1 Participants

SECONDARY outcome

Timeframe: 1 year

Number of participants with full (95-100%), mixed (5-94%), and no (0-4%) donor cells. Chimerism is reported for unsorted whole blood and T cells.

Outcome measures

Outcome measures
Measure	Transplant - 200 cGy n=29 Participants Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 200. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.	Transplant - 400 cGy n=13 Participants Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 400. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.
Donor Chimerism at 1 Year Whole blood · 95-100%	6 Participants	9 Participants
Donor Chimerism at 1 Year Whole blood · 5-94%	9 Participants	3 Participants
Donor Chimerism at 1 Year Whole blood · 0-4%	1 Participants	0 Participants
Donor Chimerism at 1 Year Whole blood · Unknown or not measured	13 Participants	1 Participants
Donor Chimerism at 1 Year T cells · 95-100%	7 Participants	10 Participants
Donor Chimerism at 1 Year T cells · 5-94%	9 Participants	2 Participants
Donor Chimerism at 1 Year T cells · 0-4%	0 Participants	0 Participants
Donor Chimerism at 1 Year T cells · Unknown or not measured	13 Participants	1 Participants

Adverse Events

Transplant - 200 cGy

Serious events: 21 serious events

Other events: 0 other events

Deaths: 1 deaths

Transplant - 400 cGy

Serious events: 7 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Adverse event data not reported

Additional Information

F Javier Bolanos Meade, MD

Johns Hopkins University

Phone: 4106146398

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Transplant - 200 cGy n=29 participants at risk Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 200. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.	Transplant - 400 cGy n=13 participants at risk Conditioning regimen with anti-thymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation - 400. Seizure prophylaxis with levetiracetam. Allogeneic bone marrow transplant infusion on Day 0. Graft-vs-host-disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and sirolimus.
Renal and urinary disorders Acute kidney injury	17.2% 5/29 • Number of events 5 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Immune system disorders Anaphylaxis	0.00% 0/29 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	7.7% 1/13 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Arthralgia	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Bacteremia	13.8% 4/29 • Number of events 4 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Cardiac disorders Cardiomyopathy	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Vascular disorders Central line thrombosis	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations CLABSI	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Cytomegalovirus	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Respiratory, thoracic and mediastinal disorders Cough	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Respiratory, thoracic and mediastinal disorders Dyspnea	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Gastrointestinal disorders Emesis	13.8% 4/29 • Number of events 4 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	7.7% 1/13 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Vascular disorders Epistaxis	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Fever	13.8% 4/29 • Number of events 6 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Nervous system disorders Hallucination	3.4% 1/29 • Number of events 2 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
General disorders Headache	13.8% 4/29 • Number of events 4 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	7.7% 1/13 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
General disorders Hematoma	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Herpes simplex virus	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Investigations Hyperkalemia	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Influenza A	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Myalgia	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Febrile neutropenia	27.6% 8/29 • Number of events 8 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	38.5% 5/13 • Number of events 6 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Gastrointestinal disorders Pain - abdomen	6.9% 2/29 • Number of events 2 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	7.7% 1/13 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Pain - ankle	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Pain - arm	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Pain - back	6.9% 2/29 • Number of events 3 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Pain - bone	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Blood and lymphatic system disorders Pain - chest	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Pain - flank	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Pain - knee	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Pain - legs	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Musculoskeletal and connective tissue disorders Pain - neck	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
General disorders Pain - unspecified	10.3% 3/29 • Number of events 4 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Blood and lymphatic system disorders Vaso-occlusive crisis	34.5% 10/29 • Number of events 22 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Nervous system disorders Paresthesia	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Nervous system disorders Posterior reversible encephalopathy syndrome	13.8% 4/29 • Number of events 4 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Respiratory, thoracic and mediastinal disorders Respiratory distress	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Respiratory syncytial virus	6.9% 2/29 • Number of events 2 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Nervous system disorders Seizure	10.3% 3/29 • Number of events 3 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Sepsis	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Immune system disorders Serum sickness	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Tuberculosis	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Upper respiratory infection	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.
Infections and infestations Urinary tract infection	3.4% 1/29 • Number of events 1 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.	0.00% 0/13 • Up to 60 days Only serious adverse events and grade 3-5 non-serious adverse events were collected.