Clinical trial · Interventional
A Clinical Study of Melphalan Flufenamide (Melflufen) and Dexamethasone for Patients With Immunoglobulin Light Chain (AL) Amyloidosis
An Open-Label, Phase 1/2 Study of Melflufen and Dexamethasone for Patients With AL Amyloidosis Following at Least One Prior Line of Therapy
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): The sponsor decided to terminate the study following an FDA request of a partial clinical hold.
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| AL Amyloidosis | AL Amyloidosis | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Dexamethasone | Drug | Dexamethasone | ALIAS |
| Melphalan-Flufenamide (Melflufen) | Drug | Melphalan Flufenamide | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Melflufen and dexamethasone in combination
- description
- Intravenous infusion of melflufen Day 1 of 28 day cycles, in combination of dexamethasone on Days 1 and 2 of each 28-day cycle.
- interventionNames
- Drug: Melphalan-Flufenamide (Melflufen)
- Drug: Dexamethasone
Primary outcomes (3)
- measure
- The primary objective in Phase 1 is to explore safety and tolerability of melflufen
- timeFrame
- During phase 1 for up to 8 cycles of treatment of 28 days each (approx. up to 8 months)
- description
- Endpoints: * Frequency and grade of Adverse Events. The maximum grade for each type of AE will be recorded for each participant and frequency tables will be presented and reviewed to determine patterns * Laboratory values (laboratory abnormalities) for hematology, coagulation, blood chemistry, urinalysis
- measure
- The primary objective in Phase 1 is to identify recommended Phase 2 dose (RP2D)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: (For full list of inclusion criteria, see study protocol) * Male or female, age 18 years or older at the time of signing the informed consent * Proven histochemical diagnosis of AL amyloidosis based on tissue specimens with Congo red staining * At least one prior line of therapy, defined as either one non-transplant regimen, one ASCT (autologous stem cell transplantation), or one regimen of induction therapy followed by a single ASCT. No more that 4 cycles of melphalan containing chemotherapy is allowed. * Measurable hematologic disease * Objectively measurable organ amyloid involvement * ECOG performance status ≤ 2 (ECOG = Eastern cooperative oncology group) * Women of child bearing potential must have a negative serum or urine pregnancy test * Less than 30% plasma cells in bone marrow aspirate or biopsy * Acceptable laboratory results met (absolute neutrophil count (ANC), platelet count, hemoglobin, total bilirubin,alkaline phosphatase, AST (aspartate aminotransferase) and ALT (alanine aminotransferase), renal function) * Male participant agrees to use contraception during treatment and 90 days after last dose of melflufen Exclusion Criteria: (For full list of exclusion criteria, see study protocol) * Amyloidosis due to known mutations of the transthyretin gene or presence of another non-AL amyloidosis * Evidence of gastro-intestinal bleeding * Cardiac risk stage 3 * Low platelets value with evidence of mucosal or internal bleeding * Medical documented cardiac syncope, NYHA Class 3 or 4 congestive heart failure, myocardial infarction, unstable angina pectoris, clinically significant ventricular arrhythmias (NYHA=New York Heart Association Functional Classification) * Clinically significant finding on 24 h Holter recording * Severe orthostatic hypotension * Clinically significant factor X deficiency * Clinically significant autonomic disease * Any medical condition that would impose excessive risk to the patient * Serious psychiatric illness, active alcoholism or drug addiction that may hinder or confuse compliance * Known HIV or active hepatitis B or C viral infections * Previous cytotoxic therapies, including cytotoxic investigational agents within 3 weeks prior to start of study treatment. Monoclonal antibodies within 4 weeks. Concomitant immunotherapy, investigational therapy and anticoagulation therapy are not permitted * Prior autologous or allogenic stem cell transplant within 12 weeks of initiation of therapy * Prior allogeneic stem cell transplant with active graft-host-disease * Prior major surgical procedure or radiation therapy within 4 weeks of the first dose of study treatment
References
Publications (0)
Data not yet available