MedDataX Logo

Trial Outcomes & Findings for Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia (NCT NCT00427336)

NCT ID: NCT00427336

Last Updated: 2011-10-27

Results Overview

Engraftment defined as (1) the first of three consecutive days of an Absolute neutrophil count (ANC) \>500/mL (b) the first of seven consecutive days of an unsupported platelet count 20,000. Patient needs to survive at least 28 days to be evaluable for engraftment. Chimerism studies need to demonstrate donor-derived hematopoiesis (\>90%)

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

9 participants

Primary outcome timeframe

First 100 days post transplant.

Results posted on

2011-10-27

Participant Flow

Recruitment Period: December 2000 to August 2009. All participants recruited at UT MD Anderson Cancer Center.

Participant milestones

Participant milestones
Measure
Fludarabine + Cyclophosphamide + ATG
Fludarabine 30 mg/m\^2/day by vein (IV), Cyclophosphamide IV 300 mg/m\^2/day, ATG (Antithymocyte Globulin) IV 3.75 mg/kg/day
Overall Study
STARTED
9
Overall Study
COMPLETED
9
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Fludarabine + Cyclophosphamide + ATG
n=9 Participants
Fludarabine 30 mg/m\^2/day by vein (IV), Cyclophosphamide IV 300 mg/m\^2/day, ATG (Antithymocyte Globulin) IV 3.75 mg/kg/day
Age Continuous
39 years
n=93 Participants
Sex: Female, Male
Female
6 Participants
n=93 Participants
Sex: Female, Male
Male
3 Participants
n=93 Participants
Region of Enrollment
United States
9 participants
n=93 Participants

PRIMARY outcome

Timeframe: First 100 days post transplant.

Engraftment defined as (1) the first of three consecutive days of an Absolute neutrophil count (ANC) \>500/mL (b) the first of seven consecutive days of an unsupported platelet count 20,000. Patient needs to survive at least 28 days to be evaluable for engraftment. Chimerism studies need to demonstrate donor-derived hematopoiesis (\>90%)

Outcome measures

Outcome measures
Measure
Fludarabine + Cyclophosphamide + ATG
n=9 Participants
Fludarabine 30 mg/m\^2/day by vein (IV), Cyclophosphamide IV 300 mg/m\^2/day, ATG (Antithymocyte Globulin) IV 3.75 mg/kg/day
Number of Patients With Engraftment Response
9 Participants

Adverse Events

Fludarabine + Cyclophosphamide + ATG

Serious events: 6 serious events
Other events: 9 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Fludarabine + Cyclophosphamide + ATG
n=9 participants at risk
Fludarabine 30 mg/m\^2/day by vein (IV), Cyclophosphamide IV 300 mg/m\^2/day, ATG (Antithymocyte Globulin) IV 3.75 mg/kg/day
Hepatobiliary disorders
Elevated Bilirubin
33.3%
3/9 • Number of events 3 • 7 years, 10 months
Respiratory, thoracic and mediastinal disorders
Shortness of Breath
11.1%
1/9 • Number of events 1 • 7 years, 10 months
Infections and infestations
Infection
22.2%
2/9 • Number of events 2 • 7 years, 10 months
Renal and urinary disorders
Elevated Creatinine
11.1%
1/9 • Number of events 1 • 7 years, 10 months
Skin and subcutaneous tissue disorders
Rash
11.1%
1/9 • Number of events 1 • 7 years, 10 months
Infections and infestations
Fever
11.1%
1/9 • Number of events 1 • 7 years, 10 months
Hepatobiliary disorders
Elevated Transaminases
11.1%
1/9 • Number of events 2 • 7 years, 10 months
Cardiac disorders
Hypotension
11.1%
1/9 • Number of events 1 • 7 years, 10 months
General disorders
Headache
11.1%
1/9 • Number of events 1 • 7 years, 10 months

Other adverse events

Other adverse events
Measure
Fludarabine + Cyclophosphamide + ATG
n=9 participants at risk
Fludarabine 30 mg/m\^2/day by vein (IV), Cyclophosphamide IV 300 mg/m\^2/day, ATG (Antithymocyte Globulin) IV 3.75 mg/kg/day
Gastrointestinal disorders
Nausea
88.9%
8/9 • Number of events 8 • 7 years, 10 months
Gastrointestinal disorders
Diarrhea
66.7%
6/9 • Number of events 6 • 7 years, 10 months
Infections and infestations
Fever
22.2%
2/9 • Number of events 2 • 7 years, 10 months
Gastrointestinal disorders
Mucositis
22.2%
2/9 • Number of events 2 • 7 years, 10 months
Skin and subcutaneous tissue disorders
Rash
22.2%
2/9 • Number of events 2 • 7 years, 10 months
Infections and infestations
Infection
11.1%
1/9 • Number of events 2 • 7 years, 10 months
Hepatobiliary disorders
Elevated Bilirubin
11.1%
1/9 • Number of events 1 • 7 years, 10 months
Musculoskeletal and connective tissue disorders
Bone Pain
11.1%
1/9 • Number of events 1 • 7 years, 10 months
Respiratory, thoracic and mediastinal disorders
Embolism
11.1%
1/9 • Number of events 1 • 7 years, 10 months
Cardiac disorders
Hypotension
11.1%
1/9 • Number of events 1 • 7 years, 10 months
Renal and urinary disorders
Elevated Creatinine
11.1%
1/9 • Number of events 1 • 7 years, 10 months

Additional Information

Paolo Anderlini, MD / Professor

UT MD Anderson Cancer Center

Results disclosure agreements

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