Trial Outcomes & Findings for Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine (NCT NCT00595127)

NCT ID: NCT00595127

Last Updated: 2016-08-10

Results Overview

Number of patients who engrafted

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 21 participants
• Primary outcome timeframe: 8 years
• Results posted on: 2016-08-10

Participant Flow

Participant milestones

Measure	Cyclophosphamide and Fludarabine To evaluate a cytoreductive regimen using standard TBI \& cyclophosphamide w/ addition of fludarabine, to prepare pts w/ Fanconi anemia for Tcell depleted, allogeneic hematopoietic stem cell transplant
Overall Study STARTED	21
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Measure	Cyclophosphamide and Fludarabine To evaluate a cytoreductive regimen using standard TBI \& cyclophosphamide w/ addition of fludarabine, to prepare pts w/ Fanconi anemia for Tcell depleted, allogeneic hematopoietic stem cell transplant
Overall Study Death	6
Overall Study Relapsed	3
Overall Study joined another trial	1

Baseline Characteristics

Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

Baseline characteristics by cohort

Measure	Cyclophosphamide and Fludarabine n=21 Participants To evaluate a cytoreductive regimen using standard TBI \& cyclophosphamide w/ addition of fludarabine, to prepare pts w/ Fanconi anemia for Tcell depleted, allogeneic hematopoietic stem cell transplant
Age, Categorical <=18 years	16 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Region of Enrollment United States	21 participants n=5 Participants

PRIMARY outcome

Timeframe: 8 years

Number of patients who engrafted

Outcome measures

Measure	Cyclophosphamide and Fludarabine n=21 Participants To evaluate a cytoreductive regimen using standard TBI \& cyclophosphamide w/ addition of fludarabine, to prepare pts w/ Fanconi anemia for Tcell depleted, allogeneic hematopoietic stem cell transplant
Incidence & Quality of Engraftment & Hematopoietic Reconstitution	19 participants

Adverse Events

Cyclophosphamide and Fludarabine

Serious events: 5 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Measure	Cyclophosphamide and Fludarabine n=21 participants at risk To evaluate a cytoreductive regimen using standard TBI \& cyclophosphamide w/ addition of fludarabine, to prepare pts w/ Fanconi anemia for Tcell depleted, allogeneic hematopoietic stem cell transplant
Cardiac disorders Cardiovascular, other	4.8% 1/21
Gastrointestinal disorders Diarrhea (PEDS/BMT)	4.8% 1/21
Respiratory, thoracic and mediastinal disorders Dyspnea	4.8% 1/21
Blood and lymphatic system disorders Hemorrhage, other	4.8% 1/21
Respiratory, thoracic and mediastinal disorders Hypoxia	9.5% 2/21
Infections and infestations Infection w.out neutropenia	4.8% 1/21
Infections and infestations Infection/Feb Neut-Other	4.8% 1/21
Renal and urinary disorders Renal failure	4.8% 1/21

Other adverse events

Measure	Cyclophosphamide and Fludarabine n=21 participants at risk To evaluate a cytoreductive regimen using standard TBI \& cyclophosphamide w/ addition of fludarabine, to prepare pts w/ Fanconi anemia for Tcell depleted, allogeneic hematopoietic stem cell transplant
Metabolism and nutrition disorders Alkaline phosphatase	14.3% 3/21
Blood and lymphatic system disorders Bilirubin	47.6% 10/21
Metabolism and nutrition disorders Hemoglobin (Hgb)	100.0% 21/21
Metabolism and nutrition disorders Hyperglycemia	71.4% 15/21
Metabolism and nutrition disorders Hyperkalemia	19.0% 4/21
Metabolism and nutrition disorders Hypermagnesmia	9.5% 2/21
Metabolism and nutrition disorders Hypertriglyceridemia	9.5% 2/21
Metabolism and nutrition disorders Hypocalcemia	9.5% 2/21
Metabolism and nutrition disorders Hypokalemia	66.7% 14/21
Metabolism and nutrition disorders Hyponatremia	38.1% 8/21
Metabolism and nutrition disorders Hypophosphatemia	52.4% 11/21
Blood and lymphatic system disorders Leukocytes	100.0% 21/21
Blood and lymphatic system disorders Lymphopenia	100.0% 21/21
Infections and infestations Neutrophils/gran	100.0% 21/21
Blood and lymphatic system disorders Platelets	100.0% 21/21
Metabolism and nutrition disorders SGOT (AST)	42.9% 9/21
Metabolism and nutrition disorders SGPT (ALT)	61.9% 13/21

Additional Information

Farid Boulad, MD, Attending

Memorial Sloan Kettering Cancer Center

Phone: +1212-639-2429

Email: bouladf@mskcc.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place