Short Course of Bortezomib in Anemic Patients With Refractory Cold Agglutinin Disease (CAD0111)
This study is not yet open for participant recruitment.
Verified January 2013 by Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor:
Gruppo Italiano Malattie EMatologiche dell'Adulto
Information provided by (Responsible Party):
Gruppo Italiano Malattie EMatologiche dell'Adulto
ClinicalTrials.gov Identifier:
NCT01696474
First received: September 27, 2012
Last updated: January 21, 2013
Last verified: January 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Refractory Cold Agglutinin Disease |
Drug: Bortezomib |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Short Course of Bortezomib in Anemic Patients With Refractory Cold Agglutinin Disease |
Resource links provided by NLM:
Further study details as provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:
Primary Outcome Measures:
- Number of patients who become transfusion-free after Bortezomib therapy. [ Time Frame: After 3 months from study entry. ] [ Designated as safety issue: No ]Cumulative proportion of patients transfusion-free at three months after Bortezomib therapy.
- Number of patients who have never been transfused with a >2g hemoglobin rise compared to baseline. [ Time Frame: After 3 months from study entry. ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Number of CTC grade 3 and 4 adverse events. [ Time Frame: After 12 months from study entry. ] [ Designated as safety issue: Yes ]Evaluation of the incidence of CTC grade 3 and 4 adverse events during and after treatment.
- Duration in months of transfusion independence. [ Time Frame: At 12 months from study entry. ] [ Designated as safety issue: No ]
- Effect of treatment on the underlying clonal B cell disorder. [ Time Frame: At 3 months from study entry. ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 25 |
| Study Start Date: | October 2012 |
| Estimated Study Completion Date: | March 2014 |
| Estimated Primary Completion Date: | March 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Bortezomib therapy
A 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.
|
Drug: Bortezomib |
Detailed Description:
Based on its activity in MM, single agent Bortezomib was tested in phase II trials in lymphoplasmacytic lymphoma, the disorder most frequently associated with CAD, and achieved 40-80% responses. These striking clinical responses indicate that proteasome activity is critical for the survival of immunoglobulin-secreting cells. The resolution of transfusion requirement in two patients with refractory CAD associated with IgMk monoclonal gammopathy has been reported after treatment with a short course of Bortezomib. It may be interesting to test the efficacy of Bortezomib in a larger series of patients with refractory CAD, idiopathic or associated with an otherwise asymptomatic B cell clonal disorder, and to evaluate the duration of clinical benefit.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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;
- Cold agglutinin (CA) titer at 4°C of 64 or higher;
- 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01696474
Contacts
| Contact: Paola Fazi, Dr. | p.fazi@gimema.it | |
| Contact: Enrico Crea | e.crea@gimema.it |
Locations
| Italy | |
| Sezione di Ematologia e Trapianti Spedali Civili | |
| Brescia, Italy, 21125 | |
| US Dipartimentale Centro per le Malattie del Sangue | |
| Castelfranco Veneto, Italy | |
| Azienda Ospedaliera di Firenze | |
| Firenze, Italy, 50011 | |
| Struttura Complessa di Ematologia Ospedali Riuniti Foggia - Azienda Ospedaliero-Universitaria | |
| Foggia, Italy | |
| Centro Trapianti di Midollo - IRCCS Ospedale Maggiore Policlinico | |
| Milano, Italy | |
| Azienda ospedaliera S. Gerardo di Monza | |
| Monza, Italy | |
| S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro | |
| Novara, Italy | |
| Div. di Ematologia IRCCS Policlinico S. Matteo | |
| Pavia, Italy, 27100 | |
| Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza | |
| Piacenza, Italy | |
| Azienda Ospedaliera Bianchi Melacrino Morelli | |
| Reggio Calabria, Italy, 89100 | |
| Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova | |
| Reggio Emilia, Italy | |
| UO di Ematologia Centro Oncologico Basilicata | |
| Rionero in Vulture, Italy | |
| Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore | |
| Rome, Italy, 00168 | |
| Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza | |
| S. G. Rotondo, Italy | |
| SCDO Ematologia 2 AOU S.Giovanni Battista | |
| Torino, Italy | |
| Azienda U.L.S.S.9 - U.O. di Ematologia | |
| Treviso, Italy | |
| Clinica Ematologica - Policlinico Universitario | |
| Udine, Italy | |
| Policlinico G. B. Rossi - Borgo Roma | |
| Verona, Italy, 37134 | |
| Ospedale San Bortolo | |
| Vicenza, Italy, 36100 | |
Sponsors and Collaborators
Gruppo Italiano Malattie EMatologiche dell'Adulto
Investigators
| Principal Investigator: | Giuseppe Rossi, Dr. | S.C. Ematologia e Dipartimento di Oncologia Medica Spedali Civili - Brescia |
More Information
No publications provided
| Responsible Party: | Gruppo Italiano Malattie EMatologiche dell'Adulto |
| ClinicalTrials.gov Identifier: | NCT01696474 History of Changes |
| Other Study ID Numbers: | CAD0111, EudraCT number 2011-006329-42 |
| Study First Received: | September 27, 2012 |
| Last Updated: | January 21, 2013 |
| Health Authority: | Italy: Ethics Committee |
Keywords provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:
|
Refractory cold agglutinin disease Bortezomib blood-transfusion |
Additional relevant MeSH terms:
|
Anemia, Hemolytic, Autoimmune Anemia, Hemolytic Anemia Hematologic Diseases Autoimmune Diseases Immune System Diseases Agglutinins Cold agglutinins Bortezomib |
Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Hemagglutinins Antineoplastic Agents Therapeutic Uses Protease Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |
ClinicalTrials.gov processed this record on May 19, 2013