Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04512235 | A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES) | PHASE3 | ACTIVE NOT_RECRUITING | 281 | — | — | Nov 3, 2020 | Apr 8, 2027 | Apr 7, 2026 | 110 | United States, Australia +17 |
| NCT04504825 | A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES) | PHASE3 | ACTIVE NOT_RECRUITING | 125 | — | — | Aug 25, 2020 | Oct 22, 2027 | Mar 17, 2026 | 90 | United States, Australia +17 |
| NCT04304144 | A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis | PHASE2 | COMPLETED | 25 | — | — | Mar 18, 2020 | Nov 14, 2023 | Mar 5, 2025 | 3 | United States |
An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, resulted in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), or an important medical event or reaction. A TEAE was defined as an AE that started after the first dose of treatment and before the last dose of study drug +140 days. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
A DLT was defined as any Grade 3 or greater study intervention-related AE that was clinically significant.
| Arm | Type | Description |
|---|---|---|
| CAEL-101 combined with SoC plasma cell dyscrasia | EXPERIMENTAL | The study is divided into 2 parts, the Primary Study and the Open-Label Extension Study. CAEL-101 is administered as an intravenous (IV) infusion over approximately 2 hours. It is planned that all patients will continue their double-blind treatment until the last patient is randomized in the study plus 18 months. |
| Placebo combined with SoC plasma cell dyscrasia | PLACEBO_COMPARATOR | Patients randomized to receive placebo will receive 0.9% normal saline in an equivalent volume to a CAEL-101 infusion (approximately 250 cc). It is planned that all patients will continue their double-blind treatment until the last patient is randomized in the study plus 18 months. |
| Part A: CAEL-101 combined with SoC CyBorD | EXPERIMENTAL | 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). |
| Part B: CAEL-101 combined with SoC CyBorD and daratumumab | EXPERIMENTAL | 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. |
| Name | Type | Description |
|---|---|---|
| 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. |
| Placebo | OTHER | Commercially available 0.9% Normal Saline will be used as the placebo. |
| cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen | DRUG | According to institutional standard of care. |
| SoC: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD) | DRUG | According to institutional standard of care. |
| Daratumumab | DRUG | Treatment for AL amyloidosis |
Key Inclusion Criteria: * AL amyloidosis stage IIIa based on the European Modification of the 2004 Standard Mayo Clinic Staging who also have NT-proBNP \> 650 ng/L at the time of Screening * Measurable hematologic disease at Screening as defined by at least one of the following: 1. Involved/unin...