NCT01696474

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2012

Typical duration for phase_2

Geographic Reach
1 country

8 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 27, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

October 1, 2012

Completed
2 months until next milestone

Study Start

First participant enrolled

December 1, 2012

Completed
4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 27, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 27, 2017

Completed
Last Updated

October 27, 2017

Status Verified

October 1, 2017

Enrollment Period

4.3 years

First QC Date

September 27, 2012

Last Update Submit

October 26, 2017

Conditions

Refractory Cold Agglutinin Disease

Keywords

Refractory cold agglutinin diseaseBortezomibblood-transfusion

Outcome Measures

Primary Outcomes (2)

  • Number of patients who become transfusion-free after Bortezomib therapy.

    Cumulative proportion of patients transfusion-free at three months after Bortezomib therapy.

    After 3 months from study entry.

  • Number of patients who have never been transfused with a >2g hemoglobin rise compared to baseline.

    After 3 months from study entry.

Secondary Outcomes (3)

  • Number of CTC grade 3 and 4 adverse events.

    After 12 months from study entry.

  • Duration in months of transfusion independence.

    At 12 months from study entry.

  • Effect of treatment on the underlying clonal B cell disorder.

    At 3 months from study entry.

Study Arms (1)

Bortezomib therapy

EXPERIMENTAL

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

Interventions

BortezomibDRUG
Bortezomib therapy

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Struttura Complessa di Ematologia Ospedali Riuniti Foggia - Azienda Ospedaliero-Universitaria

Foggia, Italy

Location

Centro Trapianti di Midollo - IRCCS Ospedale Maggiore Policlinico

Milan, Italy

Location

Cattedra di Ematologia CTMO Università degli Studi di Parma

Parma, Italy

Location

U.O. Ematologia Clinica - Azienda USL di Pescara

Pescara, Italy

Location

Azienda Ospedaliera Bianchi Melacrino Morelli

Reggio Calabria, 89100, Italy

Location

Università degli Studi "Sapienza" - Dip Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia

Roma, Italy

Location

Clinica Ematologica - Policlinico Universitario

Udine, Italy

Location

Ospedale San Bortolo

Vicenza, 36100, Italy

Location

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Interventions

Bortezomib

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

Boronic AcidsAcids, NoncarboxylicAcidsInorganic ChemicalsBoron CompoundsOrganic ChemicalsPyrazinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • Giuseppe Rossi, Dr.

    S.C. Ematologia e Dipartimento di Oncologia Medica Spedali Civili - Brescia

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 27, 2012

First Posted

October 1, 2012

Study Start

December 1, 2012

Primary Completion

March 27, 2017

Study Completion

March 27, 2017

Last Updated

October 27, 2017

Record last verified: 2017-10

Data Sharing

IPD Sharing
Will not share

Locations

IT(8)