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Risk-adapted Therapy for Primary Systemic (AL) Amyloidosis

NCT01527032 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2015-03-25

No results posted yet for this study

Summary

High-dose melphalan (MEL) with autologous stem cell transplant (SCT) is an effective therapy for systemic AL amyloidosis (AL), but treatment-related mortality (TRM) has historically been high. The investigators performed a phase II trial of risk-adapted SCT followed by adjuvant dexamethasone (dex) and thalidomide (thal) in an attempt to reduce TRM and improve response rates. Patients with newly diagnosed AL involving £2 organ systems were assigned to MEL 100, 140, or 200 mg/m2 with SCT, based on age, renal function and cardiac involvement. Patients with persistent clonal plasma cell disease 3 months post-SCT received 9 months of adjuvant thal/dex (or dex if there was a history of deep vein thrombosis or neuropathy). TRM was 4.4%. Thirty-one patients began adjuvant therapy, with 16 (52%) completing 9 months of treatment and 13 (42%) achieving an improvement in hematological response. By intention-to-treat, overall hematological response rate was 71% (36% complete response) with 44% having organ responses. With a median follow-up of 31 months, 2-year survival was 84% (95% confidence interval: 73%, 94%). Risk-adapted SCT with adjuvant thal/ dex is feasible and results in low TRM and high hematological and organ response rates in AL patients.

Conditions

  • Amyloidosis

Interventions

DRUG

melphalan, thalidomide and dexamethasone

Sponsors & Collaborators

  • FDA Office of Orphan Products Development

    lead FED

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
Healthy Volunteers
No

Timeline & Regulatory

Start
2002-09-30
Completion
2005-09-30

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01527032 on ClinicalTrials.gov