Trial record 11 of 185 for:    Amyloidosis

Risk-adapted Therapy for Primary Systemic (AL) Amyloidosis

This study has been completed.
Sponsor:
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT01527032
First received: July 28, 2011
Last updated: February 2, 2012
Last verified: July 2011
  Purpose

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.


Condition Intervention Phase
Amyloidosis
Drug: melphalan, thalidomide and dexamethasone
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Risk-adapted Therapy for AL Amyloidosis

Resource links provided by NLM:


Further study details as provided by FDA Office of Orphan Products Development:

Study Start Date: September 2002
Study Completion Date: September 2005
Detailed Description:

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 (n=45) 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). Organ involvement was kidney (67%), heart (24%), liver/GI (22%) and peripheral nervous system (18%), with 31% having two organs involved. 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.(British Journal of Haematology 2007;139:224-33)

  Eligibility

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  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT01527032     History of Changes
Other Study ID Numbers: 2174
Study First Received: July 28, 2011
Last Updated: February 2, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by FDA Office of Orphan Products Development:
amyloidosis
stem cell transplant

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Dexamethasone acetate
Dexamethasone
Dexamethasone 21-phosphate
Melphalan
Thalidomide
BB 1101
Anti-Inflammatory Agents
Therapeutic Uses
Pharmacologic Actions
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Central Nervous System Agents
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Antineoplastic Agents, Alkylating

ClinicalTrials.gov processed this record on July 29, 2014