AL Amyloidosis
Conditions
Keywords
Anselamimab
Brief summary
This is an exploratory study to assess the binding of CAEL-101/anselamimab to amyloid in vivo, recruitment of inflammatory cells and reduction of the amyloid mass.
Interventions
DRUGAnselamimab
Patients will receive anselamimab administered by intravenous infusion. The dose will be 1000 mg/m² based on body surface area (BSA) calculated from height and weight obtained during screening. BSA will not be recalculated unless body weight changes by ≥20% from screening. Study drug will be administered over approximately 2 hours. Participants will receive infusions every 7 (±1) days for the first 4 infusions, followed by every 14 (±2) days thereafter for a total treatment duration of 48 weeks. The maximum single dose is 2700 mg. Participants will be monitored for infusion-related reactions and overall tolerability for approximately 90 minutes following completion of the first 4 infusions, or longer at the investigator's discretion. Premedication with diphenhydramine (25-50 mg PO/IV), acetaminophen/paracetamol (325-650 mg PO/IV), and/or similar agents may be administered per institutional standards to reduce the risk of infusion-related reactions.
Sponsors
Stanford University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. AL amyloid deposit confirmed by biopsy and IHC or mass spectrometry 2. Amyloid deposits are measurable by imaging (ultrasound or cross-sectional) 3. 18 years or older 4. ECOG performance status 0-3 5. Adequate bone marrow reserve, hepatic and renal function as demonstrated by: 1. Absolute neutrophil count ≥ 1.0 × 109/L 2. Platelet count ≥ 75 × 109/L 3. Hemoglobin ≥ 9 g/dL 4. Total bilirubin ≤ 2 times the upper limit of normal (× ULN) unless due to Gilbert's syndrome. 5. Aspartate aminotransferase (AST) ≤ 3 × ULN 6. Alanine aminotransferase (ALT) ≤ 3 × ULN 6. No evidence of cardiac, renal or hepatic involvement by amyloidosis 1. Echocardiogram with mean wall thickness \</= 12mm unless other cardiac cause 2. 24 hour urine protein \<500mg AND estimated glomerular filtration rate (eGFR) \>50mL/min/1.73 sqm (Cockcroft-Gault formula) 3. Alkaline phosphatase below upper limit of normal and total liver span \</=15cm 7. Participants of childbearing potential agree to use contraception throughout study an 8. Ability to understand and willingness to provide written informed consent.
Exclusion criteria
1. Use of other investigational agents within 30 days of screening 2. Taking doxycycline within 30 days of screening 3. Current significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, or neurological disorders, or psychiatric disorder. 4. Major surgery within 4 weeks of enrollment 5. Pregnant 6. Breast feeding 7. Participant is eligible and agreeable to standard of care chemotherapy. 8. Presence of active infection at the time of screening 9. Participant with a monoclonal protein or isotypic light chain predominance (increased level of the involved light chain and abnormal free light chain ratio (\<0.26 or \>1.65)) ☐ ☐ 10. Participant with known or suspected systemic AL amyloidosis, or suspicion of other organ involvement. 11. Lymph node involvement 12. Involvement of amyloidoma in more than one organ. 13. AL amyloidoma involving other disease locations except those specified in the protocol 14. Presence of solitary plasmacytoma 15. Participant with clinically significant lung disorder or disease 16. Not a candidate for definitive surgical treatment (i.e., complete resection) of amyloidoma.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Amyloid Target Lesion Size | Baseline through Week 72 | Amyloid target lesion size will be assessed using RECIST v1.1 criteria. Measurements will be obtained at baseline and at Weeks 12, 24, 36, 48, and 72. The outcome will evaluate change from baseline in target lesion size. |
Countries
United States
Contacts
CONTACTMani Gupta
PRINCIPAL_INVESTIGATORMichaela Liedtke, MD
Stanford University
Outcome results
None listed