A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
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|ClinicalTrials.gov Identifier: NCT04304144|
Recruitment Status : Recruiting
First Posted : March 11, 2020
Last Update Posted : March 25, 2020
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.
|Condition or disease||Intervention/treatment||Phase|
|AL Amyloidosis||Drug: CAEL-101||Phase 2|
This is a multicenter, open-label, sequential cohort, dose-selection study of CAEL-101 in Mayo Stage I, Stage II and Stage IIIa AL amyloidosis patients. The primary objective is to define the safety and tolerability of CAEL-101 and determine the recommended Phase 3 dose for patients with AL amyloidosis. The secondary objective is to describe the pharmacokinetic (PK) profile of CAEL-101.
The study will employ a 3+3 dose escalation design. At least 3 patients will be enrolled in each dose cohort unless adverse events (AE) preventing further dosing are observed. CAEL-101 will be administered in combination with the standard of care (SoC) cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Intervention Model:||Sequential Assignment|
|Intervention Model Description:||
The initial cohort dose assignments will be: Cohort 1 500 mg/m^2, Cohort 2 750 mg/m^2 and Cohort 3 1000 mg/m^2.
The study will employ a 3+3 dose escalation design. At least 3 patients will be enrolled in each dose cohort unless adverse events (AE) preventing further dosing are observed.
The Sponsor and at least one Investigator will jointly decide the following:
The Sponsor may choose to evaluate CAEL-101 in additional patients and at other/higher doses and dosing schedules to further assess the PK profile, the benefit/risk profile and/or based on the safety findings from ongoing CAEL-101 clinical trials.
|Masking:||None (Open Label)|
|Official Title:||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|
|Actual Study Start Date :||March 18, 2020|
|Estimated Primary Completion Date :||July 27, 2020|
|Estimated Study Completion Date :||January 23, 2023|
Stage I, II and IIIa AL amyloidosis
Treatment will continue until development of significant treatment-related toxicities
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 standard of care (SoC) cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy.
- Dose Toxicity [ Time Frame: 4 weeks ]Occurrence of dose limiting toxicity (DLT) during the first 4 weeks of therapy
- Area under the plasma concentration versus time curve (AUC) [ Time Frame: 3 weeks ]For each subject, variation of concentration of CAEL-101 in the plasma will be measured
- Peak Plasma Concentration (Cmax) [ Time Frame: 3 weeks ]For each subject, the maximum concentration of CAEL-101 in the plasma will be measured
- Systemic clearance [ Time Frame: 3 weeks ]For each subject, clearance of CAEL-101 from the body will be measured
- Determination of immunogenicity [ Time Frame: 3 weeks ]The presence of anti-drug antibodies will be assayed and, if present, further evaluation will confirm positivity for anti-drug antibodies and determine specificity, neutralizing ability, cell-mediated immune response and correlation with clinical responses.
- Measure of B-type natriuretic peptide in blood serum [ Time Frame: Up to 3 years ]For each subject, blood plasma of B-type natriuretic peptide will be assayed, comparing the subject's baseline value over time.
- Measure of N-terminal pro b-type natriuretic peptide (NT-proBNP) in blood serum [ Time Frame: Up to 3 years ]For each subject, blood serum will be assayed for NT-proBNP, comparing the subject's baseline value over time.
- Measure of Cardiac troponin (cTnT) in blood serum [ Time Frame: Up to 3 years ]For each subject, blood serum will be assayed for Cardiac troponin (cTnT), comparing the subject's baseline value over time.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04304144
|Contact: Caelum Medical Monitor||+1 609 337 firstname.lastname@example.org|
|United States, Ohio|
|Cleveland, Ohio, United States, 44195|