Refractory Cold Agglutinin Disease | Short Course of Bortezomib in Anemic Patients With Refractory Cold Agglutinin Disease
Refractory Cold Agglutinin Disease research study
What is the primary objective of this study?
This multicenter, phase II, open label study will enroll patients with chronic cold agglutinin disorder. A single course of Bortezomib will be given at the dose of 1,3 mg/sqm iv on days 1, 4, 8, 11.
Who is eligible to participate?
Inclusion Criteria: - Patients with chronic cold agglutinin disorder requiring or with a hemoglobin concentration below 10g/L determined at least monthly during the two months before entering the trial; - Failure of at least one previous treatment attempt; - Hemoglobin level assessment; - Presence of serum cold agglutinin (CA). If an overnight incubation is used for CA detection, a titer at 4°C of 64 or higher is required; - Written informed consent. Exclusion Criteria: - Presence of a concomitant lymphoproliferative disorder requiring specific treatment for reasons other than cold agglutinin related hemolytic anemia; - Preexisting peripheral neuropathy; - Known hypersensitivity to Bortezomib; - Non-cooperative behaviour or non-compliance; - Psychiatric diseases or conditions that might impair the ability to give informed consent; - Patients who are pregnant (women of childbearing potential must have a negative serum pregnancy test). Post-menopausal women must be amenorrhoic for at least 24 months to be considered of non-childbearing potential. Male and female patients must agree to employ an effective barrier method of birth control throughout the study and for up to 3 months following discontinuation of study drug.
Which medical condition, disease, disorder, syndrome, illness, or injury is researched?
Refractory Cold Agglutinin Disease
Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.
Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.
Bortezomib therapyA single course of Bortezomib will be given at the dose of 1,3 mg/sqm iv on days 1, 4, 8, 11. Prophylaxis of HZ reactivation will be given with oral acyclovir at the dosage of 400 mg twice daily for one month after the end of Bortezomib.
Start Date: December 2012
Completed Date: March 27, 2017
Phase: Phase 2
Primary Outcome: Number of patients who become transfusion-free after Bortezomib therapy.
Secondary Outcome: Number of CTC grade 3 and 4 adverse events.
Study sponsors, principal investigator, and references
Principal Investigator: Giuseppe Rossi, Dr.
Lead Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto