Clinical trial NCT04398459: The Safety and Efficacy of Ibrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia
Official title: A Phase II, Multicenter, Open-label Trial to Determine the Safety and Efficacy of Ibrutinib in Refractory/Relapsed Autoimmune Hemolytic
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Refractory/Relapsed Autoimmune Hemolytic Anemia
Keywords: Ibrutinib
BTK Inhibitor
autoimmune hemolytic anemia
AIHA
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2020-05-21
Enrollment: 18
Sex: ALL
Minimum age (years): 6
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is an open-label, single-arm study to evaluate the safety and efficacy of Ibrutinib in subjects with refractory/relapsed autoimmune hemolytic anemia.


Eligibility criteria: Inclusion Criteria:

* ECOG ≤ 3
* Age from 6 to 70.
* Diagnosed with WAIHA or MAIHA.
* Meets the criteria of relapsed / refractory AIHA.
* Meets the criteria of DAT-negative AIHA without any other inherited or acquired hemolytic diseases, and previously treated effectively with glucocorticoids and rituximab.
* Signed informed consent.
* Organs in good function.

Exclusion Criteria:

* Nursing woman
* Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, etc.
* Diagnosed with cold agglutinin disease or cold agglutinin syndrome or paroxysmal cold hemoglobinuria.
* Secondary AIHA caused by drugs or infection.
* Received rituximab in 8 weeks before enrollment.
* Previously received treatment with BTK inhibitor.
* Previously received organ or stem cell transplantation.
* Have a history of thrombosis or organ infarction.
* Diagnosed with an active stage of connective tissue disease.
* Have a history of lymphoproliferative tumors or any other malignant tumors.
* Have other inherited or acquired hemolytic diseases.
* Received low-molecular-weight heparin or warfarin within 1 week before enrollment or during the Ibrutinib treatment.
* Received CYP3A4 Enzyme Inhibitors or Inducers within 1 week before enrollment or during the Ibrutinib treatment.
* Have a history of mental illness.
* Inability to understand or to follow study procedures.

Primary outcomes:
measure: Overall response rate
timeFrame: within 12 weeks
Secondary outcomes:
measure: Incidence of adverse events and severe adverse events
timeFrame: within 48 weeks
measure: Relapse free survival rate
timeFrame: within 48 weeks
Other outcomes:
Locations:
city: Zhoukou
country: China
facility: Zhoukou Central Hospital
state: Henan
city: Tai'an
country: China
facility: The Second Affilated Hospital of Shandong First Medical University
state: Shandong
city: Tianjin
country: China
facility: Regenerative Medicine Center
state: Tianjin
Study officials:
References:
citation: Fang LW, Pan H, Shi J. [Ibrutinib treatment for 2 cases of relapsed/refractory autoimmune hemolytic anemia: a pilot study]. Zhonghua Xue Ye Xue Za Zhi. 2020 May 14;41(5):412-416. doi: 10.3760/cma.j.issn.0253-2727.2020.05.009. Chinese.