Clinical trial NCT00298766: Open-Label Phase 1/2 Study of VELCADE for Injection in Patients With Light-chain (AL)-Amyloidosis
Official title: An Open-Label Phase 1/2 Study of VELCADE for Injection in Subjects With Light-Chain (AL)-Amyloidosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Amyloidosis
Lead sponsor: Millennium Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
First posted: 2006-03-03
Enrollment: 70
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a phase 1/2 open-label, dose-escalation study investigating single-agent therapy with VELCADE in patients with previously treated systemic AL-amyloidosis who require further treatment.


Eligibility criteria: Inclusion Criteria:

1. Male or Female 18 y/o and older
2. Female patients must be practicing an effective method of birth control
3. Biopsy-proven AL-amyloidosis
4. Must have been previously treated (failed at least 1 previous treatment) and in the opinion of the physician, patient requires further treatment

Exclusion Criteria:

1. Hypersensitivity to boron or mannitol
2. Prior treatment with VELCADE
3. Patient requires other concomitant chemotherapy, radiotherapy or ancillary therapy considered investigational
4. Uncontrolled infection

Primary outcomes:
measure: Maximum Tolerated Dose
timeFrame: 5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts
measure: Subjects With Treatment Emergent Adverse Events
timeFrame: from first study-related procedure to 30 days after last dose of study medication
measure: Subjects With Serious Treatment Emergent Adverse Events
timeFrame: from first study-related procedure to 30 days after last dose of study medication
measure: Subjects Grade 3/4/5 Treatment Emergent Adverse Events
timeFrame: from first study-related procedure to 30 days after last dose of study medication
measure: Subjects With Treatment Emergent Adverse Events Leading to Treatment Termination
timeFrame: from first study-related procedure to 30 days after last dose of study medication
Secondary outcomes:
measure: Best Confirmed Hematologic Responders
timeFrame: from first dose of study medication to end of study visit
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center, Samuel Oschin Comprehensive Cancer Institute
state: California
city: Atlanta
country: United States
facility: Winship Cancer Center - Emory Clinic School of Medicine
state: Georgia
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: New York
country: United States
facility: MSKCC
state: New York
Study officials:
affiliation: Millennium Pharmaceuticals, Inc.
name: Medical Monitor
role: STUDY_DIRECTOR
References:
citation: Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Blade J, Fermand JP, Hassoun H, Heffner L, Kukreti V, Vescio RA, Pei L, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Long-term follow-up from a phase 1/2 study of single-agent bortezomib in relapsed systemic AL amyloidosis. Blood. 2014 Oct 16;124(16):2498-506. doi: 10.1182/blood-2014-04-568329. Epub 2014 Sep 8.
citation: Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Blade J, Fermand JP, Hassoun H, Heffner L, Vescio RA, Liu K, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study. Blood. 2011 Jul 28;118(4):865-73. doi: 10.1182/blood-2011-02-334227. Epub 2011 May 11.
citation: Reece DE, Sanchorawala V, Hegenbart U, Merlini G, Palladini G, Fermand JP, Vescio RA, Liu X, Elsayed YA, Cakana A, Comenzo RL; VELCADE CAN2007 Study Group. Weekly and twice-weekly bortezomib in patients with systemic AL amyloidosis: results of a phase 1 dose-escalation study. Blood. 2009 Aug 20;114(8):1489-97. doi: 10.1182/blood-2009-02-203398. Epub 2009 Jun 4.