Status:
TERMINATED
A Clinical Study of Melphalan Flufenamide (Melflufen) and Dexamethasone for Patients With Immunoglobulin Light Chain (AL) Amyloidosis
Lead Sponsor:
Oncopeptides AB
Collaborating Sponsors:
PRA Health Sciences
Conditions:
AL Amyloidosis
Eligibility:
All Genders
18+ years
Phase:
PHASE1
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...
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 transformati...
Eligibility Criteria
Inclusion
- (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
- (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
Key Trial Info
Start Date :
August 6 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 5 2022
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT04115956
Start Date
August 6 2020
End Date
January 5 2022
Last Update
March 28 2022
Active Locations (9)
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1
Boston University Medical Center
Boston, Massachusetts, United States, 02111
2
Fakultní Nemocnice Ostrava
Ostrava - Poruba, Czechia, 70852
3
Centre Hospitalier Universitaire de Limoges
Limoges, France, 87000
4
Universitätsklinikum Heidelberg
Heidelberg, Germany, 69120