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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01187017
• Date of registration: 20/08/2010
• Prospective Registration: No
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
• Scientific title: A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
• Date of first enrolment: August 2010
• Target sample size: 1
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01187017
• Study type: Interventional
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Danielle M Townsley, M.D.
• Affiliation: National Heart, Lung, and Blood Institute (NHLBI)

Key inclusion & exclusion criteria

-INCLUSION CRITERIA:

1. Severe aplastic anemia characterized by:

Bone marrow cellularity < 30 percent (excluding lymphocytes)

AND

At least two of the following:

- Absolute neutrophil count < 500/ microL

- Platelet count < 20,000/ microL

- Absolute reticulocyte count < 60,000/ microL

2. Failure to respond to an initial course of h-ATG/CsA at least 3 months post-treatment
or a suboptimal response to initial h-ATG/CsA defined by both platelet and
reticulocyte count < 50,000 /microL at 3 months post-treatment

OR

3. Refractory SAA unresponsive to both horse and rabbit ATG-based regimens

4. Age greater than or equal to 2 years old

5. Weight greater than or equal to 12 kg

EXCLUSION CRITERIA:

1. Diagnosis of Fanconi anemia

2. Cardiac ejection fraction < 30 percent (evaluated by ECHO)

3. Evidence of a clonal hematologic bone marrow disorder on cytogenetics. Patients with
the presence of trisomy 8, loss of Y or del(20q) will not be excluded in the absence
of dysplastic changes in the marrow. Patients with very severe neutropenia (ANC < 200
/microL) will not be excluded initially if cytogenetics are not available or pending.
If evidence of a clonal disorder is later identified, the patient will go off study)

4. Prior immunosuppressive therapy with high dose Cy

5. Infection not adequately controlled with appropriate therapy

6. Serologic evidence of HIV infection

7. Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary,
infectious, or metabolic disease of such severity that it would preclude the patient's
ability to tolerate protocol therapy, or that death within 30 days is likely

8. Subjects with cancer who are on active chemotherapeutic treatment or who take drugs
with hematological effects

9. Current pregnancy or unwillingness to take oral contraceptives or refrain from
pregnancy if of childbearing potential

10. Not able to understand the investigational nature of the study or to give informed
consent



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pancytopenia

Aplastic Anemia

Neutropenia

Severe Aplastic Anemia

Intervention(s)

Drug: Fludarabine

Drug: Cyclophosphamide

Primary Outcome(s)

Response Rate at 6 Months [Time Frame: 6 months]

Secondary Outcome(s)

Secondary Endpoints Will Evaluated for the Study to Include: (a) Hematologic Response at 3 and 12 Months and Yearly Thereafter; (b) Relapse (c) Clonal Evolution to Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplasia or Acute Leukemia; (e) Survival. [Time Frame: 12 months]

Secondary ID(s)

100177

10-H-0177

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 02/03/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01187017