A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis

Part of paid clinical trials in Stanford, California.

Sponsor
Alexion Pharmaceuticals, Inc.
Study ID
NCT04304144
Phase
PHASE2
Status
Completed

Conditions

  • AL Amyloidosis

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • CAEL-101 — DRUG
    The investigational product, CAEL-101, is formulated as a sterile liquid solution of protein plus excipients for dilution in a single-use, stoppered, glass vial. Each 10 mL vial contains 300 mg of CAEL-101 at a concentration of 30 mg/mL. CAEL-101 will be diluted with commercially available 0.9% Normal Saline.
  • SoC: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD) — DRUG
    According to institutional standard of care.
  • Daratumumab — DRUG
    Treatment for AL amyloidosis

Study Details

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 .

Key Dates

Start date
Mar 18, 2020
Status verified
Feb 2025
Primary completion
Nov 14, 2023
Completion
Nov 14, 2023

Study Design

Enrollment
25 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part A: CAEL-101 combined with SoC CyBorD
    CAEL-101 is administered as an intravenous (IV) infusion over approximately 2 hours. The initial cohort dose assignments of CAEL-101 will be: Cohort 1 - 500 mg/m\^2 Cohort 2 - 750 mg/m\^2 Cohort 3 - 1000 mg/m\^2. CAEL-101 will be administered weekly for the first 4 weeks, and then every other week until end of study, in combination with the SoC CyBorD chemotherapy. Patients will be treated until death, unacceptable toxicity, symptomatic deterioration, Investigator decision, patient decision or Sponsor decision to terminate the study. Patients from Part A who are in the Continued Treatment Period and who, in the Investigator's judgment, should have their SoC treatment complemented with daratumumab may do so (Part B).
  • Experimental: Part B: CAEL-101 combined with SoC CyBorD and daratumumab
    CAEL-101 is administered as an intravenous (IV) infusion at the RP3D dose level. CAEL-101 will be administered weekly for the first 4 weeks, and then every other week until end of study, in combination with the SoC CyBorD chemotherapy and daratumumab. After completing approximately 50 weeks of treatment, participants may switch to an alternative maintenance dosing regimen of every four weeks (q4wk), if agreed upon by the Investigator and the Sponsor Medical Monitor. Patients will be treated until death, unacceptable toxicity, symptomatic deterioration, Investigator decision, patient decision or Sponsor decision to terminate the study.

Primary Outcome Measure

Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation [ Time Frame: First dose of study drug until 140 days after last dose of study drug (Maximum exposure: 38.90 months for Part A and 32.50 months for Part B) ]

Locations (3)

FacilityCityStateZIPSite coordinators
Research SiteStanfordCalifornia94305-
Research SiteDetroitMichigan48201-
Research SiteClevelandOhio44195-

Find similar trials in Stanford, CA

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Research Site· Stanford, CAResearch Site· Detroit, MIResearch Site· Cleveland, OH

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