Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00319878
• Date of registration: 28/04/2006
• Prospective Registration: Yes
• Primary sponsor: Office of Rare Diseases (ORD)
• Public title: Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia
• Scientific title: A Phase I/II Trial of Sirolimus (Rapamune) and Cyclosporine in Patients With Refractory Aplastic Anemia
• Date of first enrolment: May 2006
• Target sample size: 52
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT00319878
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Lynn Tihopu

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of moderate or severe aplastic anemia with bone marrow cellularity of less
than 25%

- Falls within one of the following descriptions at the time of the original diagnosis:

1. For severe aplastic anemia, fulfills any two of the following three criteria:
absolute neutrophil count less than 500/uL; absolute reticulocyte count less
than 60,000/uL; and platelet count less than 20,000/uL

2. For moderate aplastic anemia, fulfills any two of the following three criteria:
absolute neutrophil count less than 1200/ul; hemoglobin less than 8 g/dL with
corrected reticulocyte count less than 1%; and platelet count less than
60,000/uL (Note: Participants who have progressed from moderate to severe
aplastic anemia prior to study entry will be classified as having severe
aplastic anemia)

- Diagnosis of refractory aplastic anemia, as defined by a failure to achieve at least
a partial response to ATG within 6 months of treatment. Individuals who had a prior
response to ATG but who have relapsed and not responded to salvage ATG are eligible.
Individuals with relapsed disease who are not candidates for salvage ATG because they
experienced a serious or life-threatening complication prior to ATG are also
eligible.

- A Karnofsky performance status of at least 60%

- Adequate organ function, as defined by creatine levels less than 1.5 times the upper
limit normal (ULN), and liver function tests (AST, bilirubin) less than 2 times the
ULN

- Women of childbearing age must be willing to use effective contraception throughout
the study

Exclusion Criteria:

- Received ATG treatment less than 6 months prior to study entry

- Candidate for related allogeneic stem cell transplantation

- Active uncontrolled infection

- History of myelodysplastic syndrome or bone marrow cytogenetic abnormalities

- History of Fanconi's anemia or other congenital form of aplastic anemia

- Treatment with an investigational agent within 1 month of study entry

- HIV infection

- Pregnant or breastfeeding



Age minimum: 21 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Anemia, Aplastic

Intervention(s)

Drug: Cyclosporine

Drug: Sirolimus

Primary Outcome(s)

Safety and tolerability of sirolimus and cyclosporine in each stratum of participants [Time Frame: Measured at Month 6]

Secondary Outcome(s)

Rate of clonal disease evolution [Time Frame: Measured at Month 6]

Survival [Time Frame: Measured at Month 6]

Duration of hematologic response [Time Frame: Measured at Month 6]

Response rate [Time Frame: Measured at Months 3 and 6]

Secondary ID(s)

U54 RR19397-01

BMF 5403

RDCRN 5403

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Rare Diseases Clinical Research Network