Alefacept in Patients With Relapsed/Refractory Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01267643
• Date of registration: 20/12/2010
• Prospective Registration: Yes
• Primary sponsor: The Cleveland Clinic
• Public title: Alefacept in Patients With Relapsed/Refractory Aplastic Anemia
• Scientific title: A Phase 1/2 Study of Alefacept, a CD2 Receptor Antagonist in Patients With Relapsed/Refractory Aplastic Anemia
• Date of first enrolment: May 2011
• Target sample size: 4
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01267643
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Ramon V. Tiu, M.D.
• Affiliation: Cleveland Clinic Taussig Cancer Center, Case Comprehensive Cancer Center

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Fulfilled criteria for diagnosis of either moderate (mAA) or severe aplastic anemia
(sAA) at the time of initial diagnosis defined per protocol.

2. Patient with a history sAA must have had an incomplete response at least 3 months
following treatment with ATG/CsA, or they must have relapsed following an initial
response to treatment.

3. Patient must not be receiving any cyclosporine or any other T cell immunosuppressive
agents within 4 weeks of study entry.

4. Patients must have organ function as defined below:

- total bilirubin within normal institutional limits (NV: 0.0-1.5 mg/dL)

- AST(SGOT)/ALT(SGPT) <2.5 X institutional upper limit of normal AST (NV: 7-40
U/L); ALT (NV: Male 5-50 U/L; Female 0-45 U/L)

- creatinine within normal institutional limits (NV: Age 18 0.4-1.3 mg/dL; 19-99
years old 0.7-1.4 mg/dL) OR

- creatinine clearance >60 mL/min/1.73 m2 for patients with creatinine levels above
institutional normal.

5. Peripheral blood counts at the time of enrollment must include at least one of the
following: Hgb <9 g/dL or red blood cell (RBC) transfusion dependence, ANC <1000/µl,
or platelet count of <60,000/µL.

6. Women of child-bearing potential and men must agree to use adequate contraception
defined per protocol.

7. Ability to understand and the willingness to sign a written informed consent document.

Exclusion Criteria:

1. Patients may not be receiving any other investigational agents (other than
hematopoietic growth factors) within 4 weeks of study entry.

2. History of allergic reactions attributed to compounds of similar chemical or biologic
composition to Alefacept.

3. Current diagnosis of acute myeloid leukemia (AML), myelodysplastic syndromes (MDS),
Fanconi's anemia, Dyskeratosis Congenita (DC) or other hereditary forms of AA.

4. Psychiatric, addictive or any other disorder that compromises ability to give a truly
informed consent.

5. Age <18 years.

6. ECOG performance status >2 (Karnofsky <60%, see Appendix A).

7. Malignancy other than non-melanoma skin cancer within the last 2 years.

8. Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection (defined as uncontrolled infection requiring IV antibiotics, invasive fungal
infection and progressive CMV viremia), symptomatic congestive heart failure (NYH
class III and IV), unstable angina pectoris, or cardiac arrhythmia.

9. Pregnant or breastfeeding women.

10. HIV-positive patients on combination antiretroviral therapy.

11. Patients who have previously received systemic chemotherapy and/ or radiation therapy.

12. Patients who previously underwent allogeneic hematopoietic stem cell transplant.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: Alefacept

Primary Outcome(s)

To evaluate the efficacy of alefacept in refractory/ relapsed AA by determining overall response rates (ORR) which includes complete remission [CR] and partial remission (PR) rates. [Time Frame: Every 12 weeks]

To measure the number of dose limiting toxicities (DLTs) as a determination of the maximum tolerable dose (MTD). [Time Frame: Every 12 weeks]

Secondary Outcome(s)

To analyze bood samples to evaluate for predictive markers for response to Alefacept with relapsed/ refractory AA and evaluate its effect on the PNH clone. [Time Frame: Every 2 weeks for the first 4 weeks, then monthly after that]

Secondary ID(s)

CASE5Z10

CASE 5Z10

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Astellas Pharma US, Inc.