Trial Outcomes & Findings for A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia (NCT NCT01187017)

Results Overview

The primary objective is to assess hematologic response of Refractory Severe aplastic anemia (SAA) subjects to who have received fludarabine and cyclophosphamide. Subjects blood counts will be evaluated at 6 months to assess a hematologic response. The hematologic response will be defined as complete, partial or no response.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

1 participants

Primary outcome timeframe

6 months

Results posted on

2021-07-02

Participant Flow

1 participant was enrolled in this study

Participant milestones

Participant milestones
Measure	Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia Participants with Severe Aplastic Anemia will receive Fludarabine at 125 mg/m squared plus Cyclophosphamide at 60 mg/kg (Flu/Cy).
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia n=1 Participants Participants with Severe Aplastic Anemia will receive Fludarabine at 125 mg/m squared plus Cyclophosphamide at 60 mg/kg (Flu/Cy).
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: There was 1 participant enrolled in this study.

The primary objective is to assess hematologic response of Refractory Severe aplastic anemia (SAA) subjects to who have received fludarabine and cyclophosphamide. Subjects blood counts will be evaluated at 6 months to assess a hematologic response. The hematologic response will be defined as complete, partial or no response.

Outcome measures

Outcome measures
Measure	Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia n=1 Participants Participants with Severe Aplastic Anemia will receive Fludarabine at 125 mg/m squared plus Cyclophosphamide at 60 mg/kg (Flu/Cy).
Response Rate at 6 Months No Response	1 participants
Response Rate at 6 Months Partial Response	0 participants
Response Rate at 6 Months Complete Response	0 participants

SECONDARY outcome

Timeframe: 3 months

Population: 1 participant was enrolled in this study. Patient was taken off study for lack of efficacy at month 6. No data is available to report past month 6.

Number of participants with hematologic response at 3 and 12 months and yearly. The hematologic response will be defined as complete, partial or no response.

Outcome measures

Outcome measures
Measure	Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia n=1 Participants Participants with Severe Aplastic Anemia will receive Fludarabine at 125 mg/m squared plus Cyclophosphamide at 60 mg/kg (Flu/Cy).
Number of Participants With Hematologic Response Complete Response	0 Participants
Number of Participants With Hematologic Response Partial Response	0 Participants
Number of Participants With Hematologic Response No Response	1 Participants

SECONDARY outcome

Timeframe: 6 months

Population: 1 participant was enrolled in this study. Patient was taken off study for lack of efficacy at month 6. No data is available to report past month 6.

Number of patients who relapsed who experienced disease relapse following Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia

Outcome measures

Outcome measures
Measure	Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia n=1 Participants Participants with Severe Aplastic Anemia will receive Fludarabine at 125 mg/m squared plus Cyclophosphamide at 60 mg/kg (Flu/Cy).
Number of Patients Who Experienced Disease Relapse	0 Participants

SECONDARY outcome

Timeframe: 6 months

Population: 1 participant was enrolled in this study. Patient was taken off study for lack of efficacy at month 6. No data is available to report past month 6.

Number of participants with Severe Aplastic Anemia who received Fludarabine Plus Cyclophosphamide that experienced Clonal Evolution to Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplasia or Acute Leukemia.

Outcome measures

Outcome measures
Measure	Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia n=1 Participants Participants with Severe Aplastic Anemia will receive Fludarabine at 125 mg/m squared plus Cyclophosphamide at 60 mg/kg (Flu/Cy).
Number of Participants With Clonal Evolution	0 Participants

SECONDARY outcome

Timeframe: 6 months

Population: 1 participant was enrolled in this study. Patient was taken off study for lack of efficacy at month 6. No data is available to report past month 6.

Participant survival at 6 months following Fludarabine/ Cyclophosphamide in participants with Severe Aplastic Anemia

Outcome measures

Outcome measures
Measure	Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia n=1 Participants Participants with Severe Aplastic Anemia will receive Fludarabine at 125 mg/m squared plus Cyclophosphamide at 60 mg/kg (Flu/Cy).
Participant Survival Following Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia	1 Participants

Adverse Events

Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia n=1 participants at risk Participants with Severe Aplastic Anemia will receive Fludarabine at 125 mg/m squared plus Cyclophosphamide at 60 mg/kg (Flu/Cy).
Infections and infestations infection	100.0% 1/1 • Number of events 1 • 6 months
Infections and infestations neutropenic fever	100.0% 1/1 • Number of events 2 • 6 months

Other adverse events

Adverse event data not reported

Additional Information

Bhavisha Patel, MD

NIHNHLBI

Phone: 301.402.3477

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place