NCT04304144CompletedPhase 2
A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
CAEL101-203: A Phase 2, Open-label, Multicenter Dose Selection Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
Source: ClinicalTrials.gov · Last updated 5 Mar 2025
Brief summary (registry, in English)
AL amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract. The primary purpose of this study is to determine the recommended dose of CAEL-101 to facilitate progression of further clinical trials and evaluate safety and tolerability of CAEL-101 in combination with the standard of care (SoC) cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy and daratumumab .
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 25 (actual)
- First submitted
- 28 Feb 2020
- Start date
- 18 Mar 2020
- Primary completion date
- 14 Nov 2023
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Diseases
- Amyloidosis
- NCT number
- NCT04304144
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 25
- From 18 Mar 2020 to the primary completion date, 14 Nov 2023
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Receiving dialysis
- Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or percutaneous cardiac intervention with recent stent, coronary artery bypass grafting or major cerebrovascular accident within 6 months prior to screening
Lab values
- Any form of secondary, hereditary, senile, localized, dialysis-related or leukocyte chemotactic factor 2-related (ALECT2) amyloidosis
- Left ventricular ejection fraction (LVEF) \< 45 percent by echocardiogram or multigated acquisition scan (MUGA)
Other
- Patients who meet any of the following criteria will not be permitted entry to the study.
- Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma. Patients with signs and/or symptoms attributable ONLY to amyloidosis and who do NOT meet IMWG definition of smoldering myeloma may be enrolled upon approval of the medical monitor.
- Supine systolic blood pressure \< 90 mmHg or symptomatic orthostatic hypotension, defined as a decrease in systolic blood pressure upon standing of \> 20 mmHg despite medical management (e.g., midodrine, fludrocortisones) in the absence of volume depletion
Who the study is looking for
- Each patient must meet the following criteria to be enrolled in this study.
- AL amyloidosis Mayo stage I, II or IIIa
- For Part A only, measurable hematologic disease defined by at least one of the following:
- involved/uninvolved free light chain difference (dFLC) \> 5mg/dL or
- free light chain (FLC) \> 5mg/dL with abnormal Kappa/Lambda ratio or
- serum protein electrophoresis (SPEP) m- spike \> 0.5 g/dL Patients with confirmed AL amyloid diagnosis without measurable disease may be enrolled with consultation and approval by the Sponsor Medical Monitor or their designee.
- a. For Part A, currently on and continuing OR planned to start concurrent chemotherapy with CyBorD administered weekly as SoC. b. For Part B, currently on and continuing OR planned to start concurrent chemotherapy with CyBorD and daratumumab administered as SoC.
Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Detroit | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Stanford | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.