NCT00298766

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.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
70

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Jun 2005

Longer than P75 for phase_1

Geographic Reach
1 country

4 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

Study Start

First participant enrolled

June 1, 2005

Completed
9 months until next milestone

First Submitted

Initial submission to the registry

March 1, 2006

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 3, 2006

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2009

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2009

Completed
12 months until next milestone

Results Posted

Study results publicly available

August 13, 2010

Completed
Last Updated

June 25, 2012

Status Verified

June 1, 2012

Enrollment Period

4.1 years

First QC Date

March 1, 2006

Results QC Date

July 16, 2010

Last Update Submit

June 19, 2012

Conditions

Amyloidosis

Outcome Measures

Primary Outcomes (5)

  • Maximum Tolerated Dose

    Maximum Tolerated Dose (MTD) was defined as the highest dose level that has 0/1 out of 6 patients experiences Dose Limited Toxicity (DLT). MTD is defined separately for QW and BIQ dose cohorts. DLT was defined as adverse events occurring during Cycle 1 and: (1) related to VELCADE, (2) Grade 4 thrombocytopenia or neutropenia, (3) Grade 3 or higher nonhematologic toxicity.

    5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts

  • Subjects With Treatment Emergent Adverse Events

    Treatment emergent adverse events observed during outcome measure time frame

    from first study-related procedure to 30 days after last dose of study medication

  • Subjects With Serious Treatment Emergent Adverse Events

    Serious treatment emergent adverse events observed during outcome measure time frame

    from first study-related procedure to 30 days after last dose of study medication

  • Subjects Grade 3/4/5 Treatment Emergent Adverse Events

    Grade 3/4/5 treatment emergent adverse events observed during outcome measure time frame. Grade is determined according to Common Terminology Criteria for Adverse Event (CTCAE) Version 3.0.

    from first study-related procedure to 30 days after last dose of study medication

  • Subjects With Treatment Emergent Adverse Events Leading to Treatment Termination

    Treatment emergent adverse events observed during outcome measure time frame leading to treatment termination

    from first study-related procedure to 30 days after last dose of study medication

Secondary Outcomes (1)

  • Best Confirmed Hematologic Responders

    from first dose of study medication to end of study visit

Study Arms (1)

1

EXPERIMENTAL

VELCADE

Drug: VELCADE

Interventions

VELCADEDRUG

Once weekly at: 0.7, 1.0, 1.3 or 1.6 mg/m2 Or Twice-weekly at: 0.7, 1.0, or 1.3 mg/m2

Also known as: Bortezomib
1

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

  • Hypersensitivity to boron or mannitol
  • Prior treatment with VELCADE
  • Patient requires other concomitant chemotherapy, radiotherapy or ancillary therapy considered investigational
  • Uncontrolled infection

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Cedars-Sinai Medical Center, Samuel Oschin Comprehensive Cancer Institute

Los Angeles, California, 90049, United States

Location

Winship Cancer Center - Emory Clinic School of Medicine

Atlanta, Georgia, 30322, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

MSKCC

New York, New York, 10017, United States

Location

Related Publications (3)

  • 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.

    PMID: 25202139DERIVED

  • 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.

    PMID: 21562045DERIVED

  • 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.

    PMID: 19498019DERIVED

MeSH Terms

Conditions

Amyloidosis

Interventions

Bortezomib

Condition Hierarchy (Ancestors)

Proteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

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

Results Point of Contact

Title
Dr. Helgi van de Velde
Organization
Johnson & Johnson Pharmaceutical Research & Development
HVDVELDE@ITS.JNJ.COM

Study Officials

  • Medical Monitor

    Millennium Pharmaceuticals, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 1, 2006

First Posted

March 3, 2006

Study Start

June 1, 2005

Primary Completion

July 1, 2009

Study Completion

September 1, 2009

Last Updated

June 25, 2012

Results First Posted

August 13, 2010

Record last verified: 2012-06

Locations

US(4)