Trial · NCT04304144

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

NCT04304144, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2025-03-05

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis. Trials, record NCT04304144. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04304144

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
NCT number: NCT04304144
Status: Completed
Phases: Phase 2
Enrollment: 25 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: AL Amyloidosis
Interventions: CAEL-101, SoC: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD), Daratumumab
First posted: 2020-03-11
Last updated: 2025-03-05
Results first posted: 2025-03-05

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	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)	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.
Primary	Number of Participants With Dose-limiting Toxicity (DLT) During the First 4 Weeks of Therapy	4 weeks	A DLT was defined as any Grade 3 or greater study intervention-related AE that was clinically significant.
Secondary	Maximum Observed Plasma Concentration (Cmax) of CAEL-101	Predose through Week 1 and through Week 20
Secondary	Area Under Plasma Concentration-time Curve Over Dosing Interval (AUCtau) of CAEL-101	Predose through Week 1 and through Week 20

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	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)	Safety Set included all participants who were treated with at least 1 dose of CAEL-101. AE data were collected for the participants in the DLT period (Part A) by dose separately. Additionally, the AE data for participants in the DLT period were also collected regardless of dose level received along with the AE data collected during the Part A Continued Treatment (Part A) period as an Overall arm.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Dose-limiting Toxicity (DLT) During the First 4 Weeks of Therapy	4 weeks	Safety Set included all participants who were treated with at least 1 dose of CAEL-101.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Maximum Observed Plasma Concentration (Cmax) of CAEL-101	Predose through Week 1 and through Week 20	The Pharmacokinetic Set (PKS) included all participants who had at least 1 measurable plasma concentration. Number analyzed = participants evaluable at specified timepoint.	micrograms (µg)/milliliter (mL)	MEAN
SECONDARY	Area Under Plasma Concentration-time Curve Over Dosing Interval (AUCtau) of CAEL-101	Predose through Week 1 and through Week 20	The PKS included all participants who had at least 1 measurable plasma concentration. Number analyzed = participants evaluable at specified timepoint.	hours*µg/mL	MEAN

Source ClinicalTrials.gov

Results by group31

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	DLT Period (Part A): CAEL-101 500 mg/m^2	Any TEAEs	4	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	DLT Period (Part A): CAEL-101 750 mg/m^2	Any TEAEs	3	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	DLT Period (Part A): CAEL-101 1000 mg/m^2	Any TEAEs	6	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	Overall (Part A): DLT + Continued Treatment Period: CAEL-101 Combined With SoC CyBorD	Any TEAEs	13	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	Continued Treatment Period (Part B): CAEL-101 Combined With SoC CyBorD and Daratumumab	Any TEAEs	12	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	DLT Period (Part A): CAEL-101 500 mg/m^2	Treatment-emergent SAEs	0	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	DLT Period (Part A): CAEL-101 750 mg/m^2	Treatment-emergent SAEs	0	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	DLT Period (Part A): CAEL-101 1000 mg/m^2	Treatment-emergent SAEs	2	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	Overall (Part A): DLT + Continued Treatment Period: CAEL-101 Combined With SoC CyBorD	Treatment-emergent SAEs	9	Participants
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation	Continued Treatment Period (Part B): CAEL-101 Combined With SoC CyBorD and Daratumumab	Treatment-emergent SAEs	7	Participants

1 to 10 of 31Source ClinicalTrials.gov

See all 31 results by group

Participant flow28

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
DLT Observation Period	STARTED	Part A: CAEL101 500 mg/m^2 Combined With SoC CyBorD	4
DLT Observation Period	STARTED	Part A: CAEL101 750 mg/m^2 Combined With SoC CyBorD	3
DLT Observation Period	STARTED	Part A: CAEL101 1000 mg/m^2 Combined With SoC CyBorD	6
DLT Observation Period	STARTED	Part B: CAEL-101 Combined With SoC CyBorD and Daratumumab	0
DLT Observation Period	Received At Least 1 Dose of Study Drug	Part A: CAEL101 500 mg/m^2 Combined With SoC CyBorD	4
DLT Observation Period	Received At Least 1 Dose of Study Drug	Part A: CAEL101 750 mg/m^2 Combined With SoC CyBorD	3
DLT Observation Period	Received At Least 1 Dose of Study Drug	Part A: CAEL101 1000 mg/m^2 Combined With SoC CyBorD	6
DLT Observation Period	Received At Least 1 Dose of Study Drug	Part B: CAEL-101 Combined With SoC CyBorD and Daratumumab	0
DLT Observation Period	COMPLETED	Part A: CAEL101 500 mg/m^2 Combined With SoC CyBorD	4
DLT Observation Period	COMPLETED	Part A: CAEL101 750 mg/m^2 Combined With SoC CyBorD	2

1 to 10 of 28Source ClinicalTrials.gov

See all 28 participant flow

Adverse events690

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Anaemia	Blood and lymphatic system disorders	DLT Period (Part A): CAEL-101 500 mg/m^2	0	4
other	Anaemia	Blood and lymphatic system disorders	DLT Period (Part A): CAEL-101 750 mg/m^2	0	3
other	Anaemia	Blood and lymphatic system disorders	DLT Period (Part A): CAEL-101 1000 mg/m^2	2	6
other	Anaemia	Blood and lymphatic system disorders	Overall (Part A): DLT + Continued Treatment Period: CAEL-101 Combined With SoC CyBorD	7	13	11
other	Anaemia	Blood and lymphatic system disorders	Continued Treatment Period (Part B): CAEL-101 Combined With SoC CyBorD and Daratumumab	4	12	7
other	Thrombocytopenia	Blood and lymphatic system disorders	DLT Period (Part A): CAEL-101 500 mg/m^2	0	4
other	Thrombocytopenia	Blood and lymphatic system disorders	DLT Period (Part A): CAEL-101 750 mg/m^2	0	3
other	Thrombocytopenia	Blood and lymphatic system disorders	DLT Period (Part A): CAEL-101 1000 mg/m^2	0	6
other	Thrombocytopenia	Blood and lymphatic system disorders	Overall (Part A): DLT + Continued Treatment Period: CAEL-101 Combined With SoC CyBorD	0	13	0
other	Thrombocytopenia	Blood and lymphatic system disorders	Continued Treatment Period (Part B): CAEL-101 Combined With SoC CyBorD and Daratumumab	2	12	2

1 to 10 of 690Source ClinicalTrials.gov

See all 690 adverse events

Adverse event totals15

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	DLT Period (Part A): CAEL-101 500 mg/m^2	0	4
Total, all-cause mortality	DLT Period (Part A): CAEL-101 750 mg/m^2	0	3
Total, all-cause mortality	DLT Period (Part A): CAEL-101 1000 mg/m^2	0	6
Total, all-cause mortality	Overall (Part A): DLT + Continued Treatment Period: CAEL-101 Combined With SoC CyBorD	1	13
Total, all-cause mortality	Continued Treatment Period (Part B): CAEL-101 Combined With SoC CyBorD and Daratumumab	2	12
Total, other adverse events	DLT Period (Part A): CAEL-101 500 mg/m^2	4	4
Total, other adverse events	DLT Period (Part A): CAEL-101 750 mg/m^2	3	3
Total, other adverse events	DLT Period (Part A): CAEL-101 1000 mg/m^2	6	6
Total, other adverse events	Overall (Part A): DLT + Continued Treatment Period: CAEL-101 Combined With SoC CyBorD	13	13
Total, other adverse events	Continued Treatment Period (Part B): CAEL-101 Combined With SoC CyBorD and Daratumumab	11	12

1 to 10 of 15Source ClinicalTrials.gov

See all 15 adverse event totals

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

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