NCT00017680

Brief Summary

OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation. II. Determine the toxicity of this regimen in these patients.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jul 1999

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

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

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 1999

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

June 6, 2001

Completed
Same day until next milestone

First Posted

Study publicly available on registry

June 6, 2001

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2004

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2004

Completed
Last Updated

June 9, 2008

Status Verified

June 1, 2008

Enrollment Period

4.8 years

First QC Date

June 6, 2001

Last Update Submit

June 6, 2008

Conditions

Amyloidosis

Keywords

amyloidosisarthritis & connective tissue diseasesgenetic diseases and dysmorphic syndromeshematopoietic/lymphoid canceroncologic disordersplasma cell neoplasmprimary systemic amyloidosisrare disease

Outcome Measures

Primary Outcomes (1)

  • Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction

Secondary Outcomes (1)

  • Toxicity of high dose chemotherapy regimen

Interventions

MelphalanDRUG

100 mg/m2 twice a day from Days -2 and -1 as induction therapy

Autologous Stem Cell TransplantationPROCEDURE

Bone marrow and peripheral blood stem cells harvested

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
PROTOCOL ENTRY CRITERIA: Disease Characteristics * Histologically confirmed primary amyloidosis * Ineligible for other high priority national or international study Prior/Concurrent Therapy * Biologic therapy: Concurrent participation in gene therapy trials allowed * Chemotherapy: Prior chemotherapy allowed No other concurrent chemotherapy * Endocrine therapy: No concurrent steroids unless given with amphotericin B, for adrenal failure, or for septic shock No concurrent hormones except for non-disease-related conditions (e.g., insulin for diabetes) * Other: No concurrent barbiturates or acetaminophen Concurrent participation in supportive care trials allowed Patient Characteristics * Performance status: ECOG 0-3 * Hepatic: Bilirubin less than 2 times normal * Renal: Creatinine less than 2.5 mg/dL OR On stable hemodialysis * Pulmonary: DLCO at least 60% predicted OR Clearance by pulmonologist * Other: HIV negative

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

AmyloidosisArthritisConnective Tissue DiseasesGenetic Diseases, InbornHematologic NeoplasmsNeoplasms, Plasma CellImmunoglobulin Light-chain AmyloidosisRare Diseases

Interventions

Melphalan

Condition Hierarchy (Ancestors)

Proteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesJoint DiseasesMusculoskeletal DiseasesSkin and Connective Tissue DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNeoplasms by SiteNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesNeoplasms by Histologic TypeLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesParaproteinemiasDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Nitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsAmino Acids, Peptides, and Proteins

Study Officials

  • Charles S. Hesdorffer

    Herbert Irving Comprehensive Cancer Center

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

June 6, 2001

First Posted

June 6, 2001

Study Start

July 1, 1999

Primary Completion

April 1, 2004

Study Completion

April 1, 2004

Last Updated

June 9, 2008

Record last verified: 2008-06

Locations

US(1)