Efficacy and Safety of Ixazomib and Dexamethasone Refractory Autoimmune Cytopenia (NCT03965624) | Clinical Trial Compass
WithdrawnPhase 2
Efficacy and Safety of Ixazomib and Dexamethasone Refractory Autoimmune Cytopenia
Stopped: Decision of sponsor: impossibility of contract with Takeda
France0Started 2019-09-01
Plain-language summary
Some patients with antibody-mediated autoimmune hematological diseases (warm autoimmune hemolytic anemia (wAIHA), cold agglutinin disease (cAIHA) and immune thrombocytopenia (ITP)) shows no or only minor and transient response to therapy despite several treatment-lines. Such patients are more likely to have a severe disease, with a higher morbidity and mortality.
Hypothesis Effective depletion of autoreactive plasma cells might be the key for a curative approach of these diseases. Therefore, there is a rationale for using proteasome inhibitors (PIs) in these refractory patients.
The rationale is that non-tumoral autoreactive plasma cells are rapidly targeted by proteasome inhibitors. It led us to propose a short course of dexamethasone and ixazomib (5 cycles), to evaluate the safety/efficacy of this innovative strategy of treatment.
Method Prospective interventional uncontrolled single arm open study evaluating the rate of patients achieving 5 cycles of ixazomib and dexamethasone without severe toxicity and response on therapy.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Age \>= 18 years
✓. Diagnosis of ITP according to the international definition (Rodeghiero et al Blood 2009)
✓. Platelets count \< 30 x 109/L or \<50 x 109/L if presence of hemorrhagic events or other reason left up to investigator discretion within the months preceding inclusion.
✓. Multirefractory ITP defined as patients who have previously failed to maintain a response after rituximab (anti-CD20), splenectomy, and romiplostim and eltrombopag, except if patients have any contraindications or refused these treatments
✓. Age \>= 18 years
✓. Diagnosis of wAIHA , symptomatic anemia and a positive direct antiglobulin test
✓. Refractory AIHA who have previously failed to maintain a sustained response after rituximab (anti-CD20) and splenectomy except if patients have any contraindications or refused these treatments.
✓. Absolute neutrophil count (ANC) \>=1,000/mm3
Exclusion criteria
What they're measuring
1
Rate of patients achieving a response (CR+R) with 5 cycles without severe toxicity (grade III/IV)
✕. Infection requiring systemic antibiotic therapy or other serious infection within 14 days before study enrollment.
✕. Female patients who are lactating or have a positive serum pregnancy test during the screening period.
✕. Evidence of current uncontrolled cardiovascular conditions, including uncontrolled hypertension, uncontrolled cardiac arrhythmias, symptomatic congestive heart failure, unstable angina, or myocardial infarction within the past 6 months.
✕. Systemic treatment, within 14 days before the first dose of ixazomib, with strong CYP3A inducers (rifampin, rifapentine, rifabutin, carbamazepine, phenytoin, phenobarbital, azathioprine), or use of St. John's wort.
✕. ) Positive HIV test and/or hepatitis virus C infection and/or positive hepatitis B virus surface antigen or core antibody (HbsAg or HBcAb) and/or Active Varicella or Herpes and zoster infection.
✕. Any serious medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with the completion of treatment according to this protocol.
✕. Known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent.