Ibrutinib in Steroid Refractory Autoimmune Hemolytic Anemia

• Status: Active, not recruiting
• Trial end date: 2022-03-31
• Target enrollment: 0
• Participant gender: All

Summary

This is an open-labeled, single arm, Phase II, two staged study of combination of ibrutinib and rituximab in patients with autoimmune cytopenia and underlying CLL. In stage I (remission induction) patients will receive ibrutinib and rituximab for 6 months; in stage II (maintenance) - only ibrutinib until relapse, progression or unacceptable toxicity. A total of 50 patients will be enrolled into the trial. The results will be compared to historical control, efficacy of rituximab as monotherapy or in combination with chemotherapy in patients with AIC and underlying CLL.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Eugene Nikitin

Criteria

Inclusion Criteria

1. Flow cytometry confirmation of immunophenotype with CD5, CD19, CD20, CD23, CD79b, and surface Ig prior to Visit 2

2. Proven diagnosis autoimmune hemolytic anemia or pure red cell aplasia

3. Relapse of AIHA/PRCA after steroids, splenectomy or rituximab or refractoriness to glucocorticosteroid hormones

4. No more than 2 lines of antileukemia treatment

5. Hematology values must be within the following limits:

1. Absolute neutrophil count 1000/mm3 independent of growth factor support

2. Platelets 100.000/mm3 or 50.000/mm3 if bone marrow involvement independent of transfusion support in either situation

6. Biochemical values within the following limits:

1. Alanine aminotransferase and aspartate aminotransferase less 3 upper limit of normal (ULN)

2. Total bilirubin less 1.5x ULN unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin

3. Serum creatinine less 2 x ULN or estimated Glomerular Filtration Rate more 40 mL/min/1.73 m2

7. Age ≥ 18 years

8. ECOG Performance Status of 0-2

9. Life expectancy of at least 6 months

10. Women of childbearing potential and men who are sexually active must be practicing a highly effective method of birth control during and after the study consistent with local regulations regarding the use of birth control methods for subjects participating in clinical trials. Men must agree to not donate sperm during and after the study. For females, these restrictions apply for 1 month after the last dose of study drug. For males, these restrictions apply for 3 months after the last dose of study drug.

11. Women who are pregnant or breastfeeding are ineligible for this study.

12. Sign (or their legally-acceptable representatives must sign) an informed consent document indicating that they understand the purpose of and procedures required for the study, including biomarkers, and are willing to participate in the study.