Ignite Creation Date:
2025-12-24 @ 11:36 PM
Ignite Modification Date:
2026-01-04 @ 2:54 AM
Study NCT ID:
NCT04115956
Status:
TERMINATED
Last Update Posted:
2022-03-28
First Post:
2019-08-29
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Study of Melphalan Flufenamide (Melflufen) and Dexamethasone for Patients With Immunoglobulin Light Chain (AL) Amyloidosis
Sponsor:
Oncopeptides AB
Organization:
Study Overview
Official Title:
An Open-Label, Phase 1/2 Study of Melflufen and Dexamethasone for Patients With AL Amyloidosis Following at Least One Prior Line of Therapy
Status:
TERMINATED
Status Verified Date:
2022-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
The sponsor decided to terminate the study following an FDA request of a partial clinical hold.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a phase 1/2 open label study of melphalan flufenamide (melflufen) in combination with dexamethasone for participants with Al amyloidosis following at least one prior line of therapy. Melflufen will be administered on Day 1 of each 28-day cycle in combination with dexamethasone on days 1 and 2.
In both phases, treatment of each individual participant will continue for up to 8 cycles or until any stopping events occur.
Approximately 46 participants will be enrolled.
The study was intended to be a Phase 1/2 trial but was early terminated and never moved forward to Phase 2.
In both phases, treatment of each individual participant will continue for up to 8 cycles or until any stopping events occur.
Approximately 46 participants will be enrolled.
The study was intended to be a Phase 1/2 trial but was early terminated and never moved forward to Phase 2.
Detailed Description:
This is a clinical trial of melphalan flufenamide (melflufen), a peptide-conjugated alkylator which belongs to an novel class of drugs called peptidase-enhanced compounds, and targets the transformation process of tumor cells with a unique mechanism of action, as potential treatment option of AL amyloidosis.
AL amyloidosis is a rare progressive disease caused by proteotoxic light chain protein produced by small plasma cell clone. This plasma cell dyscrasia is characterized by monoclonal plasma cell's excessive production of monoclonal immunoglobulin light-chains that tends to misfold and subsequently deposit as amyloid fibrils in visceral organs. The plasma cell dyscrasia in AL amyloidosis is similar to that in multiple myeloma (MM) and therapies that are effective in MM are often used to treat AL amyloidosis.
Melphalan flufenamide is currently been evaluated in several ongoing clinical trials in patients with multiple myeloma, with observed efficacy. There are currently no therapies approved for treatment of AL amyloidosis and based on the efficacy of melphalan flufenamide and the demonstrated efficacy of melphalan (and other alkylators), it is anticipated that patients with AL amyloidosis may receive benefit from treatment with melphalan flufenamide.
This study consist of a screening period (up to 28 days), a treatment period (up to 8 cycles) and a follow-up period (up to 24 months).
Phase 1: Approximately 8-30 participants will be screened to achieve 7-23 enrolled participants.
Phase 2: Approximately 30 participants will be screened to achieve 23 enrolled participants.
The study was intended to be a Phase 1/2 trial but was early terminated and study never moved forward to Phase 2.
AL amyloidosis is a rare progressive disease caused by proteotoxic light chain protein produced by small plasma cell clone. This plasma cell dyscrasia is characterized by monoclonal plasma cell's excessive production of monoclonal immunoglobulin light-chains that tends to misfold and subsequently deposit as amyloid fibrils in visceral organs. The plasma cell dyscrasia in AL amyloidosis is similar to that in multiple myeloma (MM) and therapies that are effective in MM are often used to treat AL amyloidosis.
Melphalan flufenamide is currently been evaluated in several ongoing clinical trials in patients with multiple myeloma, with observed efficacy. There are currently no therapies approved for treatment of AL amyloidosis and based on the efficacy of melphalan flufenamide and the demonstrated efficacy of melphalan (and other alkylators), it is anticipated that patients with AL amyloidosis may receive benefit from treatment with melphalan flufenamide.
This study consist of a screening period (up to 28 days), a treatment period (up to 8 cycles) and a follow-up period (up to 24 months).
Phase 1: Approximately 8-30 participants will be screened to achieve 7-23 enrolled participants.
Phase 2: Approximately 30 participants will be screened to achieve 23 enrolled participants.
The study was intended to be a Phase 1/2 trial but was early terminated and study never moved forward to Phase 2.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: