NCT00030381

Brief Summary

Iododoxorubicin may dissolve protein deposits and be an effective treatment for primary systemic amyloidosis. Phase I trial to determine the effectiveness of iododoxorubicin in treating patients who have primary systemic amyloidosis

Trial Health

35
At Risk

Trial Health Score

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

Enrollment
22

participants targeted

Target at P25-P50 for phase_1

Geographic Reach
1 country

1 active site

Status
terminated

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

December 1, 2001

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

February 14, 2002

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2003

Completed
9 months until next milestone

First Posted

Study publicly available on registry

September 17, 2003

Completed
Last Updated

January 16, 2013

Status Verified

January 1, 2013

Enrollment Period

1.1 years

First QC Date

February 14, 2002

Last Update Submit

January 15, 2013

Conditions

Primary Systemic Amyloidosis

Outcome Measures

Primary Outcomes (1)

  • MTD of IDOX defined as the highest safely-tolerated dose where =< 1 patient experiences DLT with the next higher dose having at least 2 patients who experience DLT

    The number and severity of toxicity incidents will indicate the level of tolerance of IDOX in the treatment of primary amyloidosis. Non-hematologic toxicities will be evaluated via the ordinal CTC standard toxicity grading. Hematologic toxicity measures of thrombocytopenia, neutropenia and leukopenia will be assessed using continuous variables as the outcome measures (primarily nadir and percent change from baseline values) as well as categorization via CTC standard toxicity grading.

    12 weeks

Secondary Outcomes (1)

  • Laboratory correlates

    Up to 3 months post treatment

Study Arms (1)

Treatment (iododoxorubicin)

EXPERIMENTAL

Patients receive iododoxorubicin IV over 15 minutes on days 1, 8, 15, and 22. Treatment repeats every 12 weeks for a total of 4 courses or a cumulative dose of 400 mg/m\^2 in the absence of disease progression or unacceptable toxicity.

Drug: 4'-iodo-4'-deoxydoxorubicinOther: pharmacological study

Interventions

4'-iodo-4'-deoxydoxorubicinDRUG

Given IV

Also known as: IDOX, iododoxorubicin
Treatment (iododoxorubicin)
pharmacological studyOTHER

Correlative studies

Also known as: pharmacological studies
Treatment (iododoxorubicin)

Eligibility Criteria

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

You may qualify if:

  • Histochemically confirmed amyloidosis by polarizing microscopy of greenbirefringent material in Congo red-stained tissue specimens
  • At least one of the following:
  • Demonstrable M-protein in serum or urine
  • Clonal population of plasma cells in bone marrow
  • Immunohistochemical stain with anti-light chain antisera of amyloid fibrils
  • Symptomatic organ involvement, including liver involvement, mild cardiac involvement, renal involvement, grade 1 or 2 peripheral neuropathy, or soft tissue involvement (including tongue)
  • No purpura or carpal tunnel syndrome as sole manifestation of disease
  • No clinically overt multiple myeloma defined as monoclonal bone marrow platelet concentration greater than 20% and at least one of the following:
  • Bone lesions
  • Anemia
  • Hypercalcemia
  • Performance status - ECOG 0-3 (3 allowed only if related to muscular infiltration by amyloid or peripheral neuropathy)
  • Platelet count at least 100,000/mm\^3
  • Absolute neutrophil count at least 1,500/mm\^3
  • Total bilirubin no greater than 2.0 mg/dL
  • +27 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis

Interventions

4'-deoxy-4'-iododoxorubicin

Condition Hierarchy (Ancestors)

Neoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsAmyloidosisProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesParaproteinemias

Study Officials

  • Angela Dispenzieri

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 14, 2002

First Posted

September 17, 2003

Study Start

December 1, 2001

Primary Completion

January 1, 2003

Last Updated

January 16, 2013

Record last verified: 2013-01

Locations

US(1)