TriangleNCT04304144 · STUDY IN CONTEXT
A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
Read the full study record and all posted results →
Linked outcomes
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.
Maximum Observed Plasma Concentration (Cmax) of CAEL-101
Area Under Plasma Concentration-time Curve Over Dosing Interval (AUCtau) of CAEL-101
Reported safety
Quality of life and patient-reported measures
No linked quality-of-life measure is recorded for this setting and study.
Full draft applicability and source-link record
- trial
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- trial label
- A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis
- applicability
- narrower
- applicability note
- Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- provenance class
- registry-condition-search
- discovery note
- Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
- outcome Count
- 4
- harms Count
- 5
- qol Count
- 0
- outcomes
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0000
- source section id
- NCT04304144-om-0000
- source outcome index
- 0
- endpoint
- Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs), and AEs Leading to Treatment Discontinuation
- endpoint definition
- 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.
- time origin
- 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)
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0001
- source section id
- NCT04304144-om-0001
- source outcome index
- 1
- endpoint
- Number of Participants With Dose-limiting Toxicity (DLT) During the First 4 Weeks of Therapy
- endpoint definition
- A DLT was defined as any Grade 3 or greater study intervention-related AE that was clinically significant.
- time origin
- 4 weeks
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Safety Set included all participants who were treated with at least 1 dose of CAEL-101.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0002
- source section id
- NCT04304144-om-0002
- source outcome index
- 2
- endpoint
- Maximum Observed Plasma Concentration (Cmax) of CAEL-101
- endpoint definition
- Maximum Observed Plasma Concentration (Cmax) of CAEL-101
- time origin
- Predose through Week 1 and through Week 20
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- The Pharmacokinetic Set (PKS) included all participants who had at least 1 measurable plasma concentration. Number analyzed = participants evaluable at specified timepoint.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0003
- source section id
- NCT04304144-om-0003
- source outcome index
- 3
- endpoint
- Area Under Plasma Concentration-time Curve Over Dosing Interval (AUCtau) of CAEL-101
- endpoint definition
- Area Under Plasma Concentration-time Curve Over Dosing Interval (AUCtau) of CAEL-101
- time origin
- Predose through Week 1 and through Week 20
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- The PKS included all participants who had at least 1 measurable plasma concentration. Number analyzed = participants evaluable at specified timepoint.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- arms
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0000
- arm key
- OG000
- arm label
- DLT Period (Part A): CAEL-101 500 mg/m^2
- regimen
- Participants received CAEL-101 500 mg/m\^2 administered weekly as an IV infusion the first 4 weeks (DLT observation period).
- analyzed count
- 4
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0000#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0000
- arm key
- OG001
- arm label
- DLT Period (Part A): CAEL-101 750 mg/m^2
- regimen
- Participants received CAEL-101 750 mg/m\^2 administered weekly as an IV infusion the first 4 weeks (DLT observation period).
- analyzed count
- 3
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0000#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0000
- arm key
- OG002
- arm label
- DLT Period (Part A): CAEL-101 1000 mg/m^2
- regimen
- Participants received CAEL-101 1000 mg/m\^2 administered weekly as an IV infusion the first 4 weeks (DLT observation period).
- analyzed count
- 6
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0000#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0000
- arm key
- OG003
- arm label
- Overall (Part A): DLT + Continued Treatment Period: CAEL-101 Combined With SoC CyBorD
- regimen
- Participants received CAEL-101 during the DLT period weekly as an IV infusion the first 4 weeks, and thereafter received CAEL-101 at the RP3D (1000 mg/m\^2) every other week until the end of study, in combination with the SoC CyBorD (cyclophosphamide, bortezomib, dexamethasone) chemotherapy.
- analyzed count
- 13
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0000#groups/OG003
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0000
- arm key
- OG004
- arm label
- Continued Treatment Period (Part B): CAEL-101 Combined With SoC CyBorD and Daratumumab
- regimen
- Participants received CAEL-101 at the RP3D (1000 mg/m\^2) administered weekly as an IV infusion for the first 4 weeks, and then every other week until end of study, in combination with the SoC CyBorD chemotherapy and daratumumab.
- analyzed count
- 12
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0000#groups/OG004
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0001
- arm key
- OG000
- arm label
- Part A: CAEL-101 500 mg/m^2 Combined With SoC CyBorD
- regimen
- Participants received CAEL-101 500 mg/m\^2 administered weekly as an IV infusion the first 4 weeks (DLT observation period), and thereafter received CAEL-101 at the RP3D (1000 mg/m\^2) every other week until the end of study, in combination with the SoC CyBorD.
- analyzed count
- 4
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0001#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0001
- arm key
- OG001
- arm label
- Part A: CAEL-101 750 mg/m^2 Combined With SoC CyBorD
- regimen
- Participants received CAEL-101 750 mg/m\^2 administered weekly as an IV infusion the first 4 weeks (DLT observation period), and thereafter received CAEL-101 at the RP3D (1000 mg/m\^2) every other week until the end of study, in combination with the SoC CyBorD chemotherapy.
- analyzed count
- 3
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0001#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0001
- arm key
- OG002
- arm label
- Part A: CAEL-101 1000 mg/m^2 Combined With SoC CyBorD
- regimen
- Participants received CAEL-101 1000 mg/m\^2 administered weekly as an IV infusion the first 4 weeks, and then every other week until the end of study, in combination with the SoC CyBorD chemotherapy.
- analyzed count
- 6
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0001#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0001
- arm key
- OG003
- arm label
- Part B: CAEL-101 Combined With SoC CyBorD and Daratumumab
- regimen
- Participants received CAEL-101 at the RP3D (1000 mg/m\^2) administered weekly as an IV infusion for the first 4 weeks, and then every other week until end of study, in combination with the SoC CyBorD chemotherapy and daratumumab.
- analyzed count
- 12
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0001#groups/OG003
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0002
- arm key
- OG000
- arm label
- Part A: CAEL-101 500 mg/m^2
- regimen
- Participants received CAEL-101 500 mg/m\^2 administered weekly as an IV infusion the first 4 weeks (DLT observation period), and thereafter received CAEL-101 at the RP3D (1000 mg/m\^2) every other week until the end of study.
- analyzed count
- 4
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0002#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0002
- arm key
- OG001
- arm label
- Part A: CAEL-101 750 mg/m^2
- regimen
- Participants received CAEL-101 750 mg/m\^2 administered weekly as an IV infusion the first 4 weeks (DLT observation period), and thereafter received CAEL-101 at the RP3D (1000 mg/m\^2) every other week until the end of study.
- analyzed count
- 3
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0002#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- outcome key
- om-0002
- arm key
- OG002
- arm label
- Part A: CAEL-101 1000 mg/m^2
- regimen
- Participants received CAEL-101 1000 mg/m\^2 administered weekly as an IV infusion the first 4 weeks, and then every other week until the end of study.
- analyzed count
- 6
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04304144-om-0002#groups/OG002
- harms
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- harm key
- EG000
- source section id
- NCT04304144-ae
- event group key
- EG000
- event group label
- DLT Period (Part A): CAEL-101 500 mg/m^2
- serious affected
- 0
- serious at risk
- 4
- other affected
- 4
- other at risk
- 4
- frequency threshold
- 5
- collection period
- 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)
- event definition
- 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.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- harm key
- EG001
- source section id
- NCT04304144-ae
- event group key
- EG001
- event group label
- DLT Period (Part A): CAEL-101 750 mg/m^2
- serious affected
- 0
- serious at risk
- 3
- other affected
- 3
- other at risk
- 3
- frequency threshold
- 5
- collection period
- 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)
- event definition
- 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.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- harm key
- EG002
- source section id
- NCT04304144-ae
- event group key
- EG002
- event group label
- DLT Period (Part A): CAEL-101 1000 mg/m^2
- serious affected
- 1
- serious at risk
- 6
- other affected
- 6
- other at risk
- 6
- frequency threshold
- 5
- collection period
- 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)
- event definition
- 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.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- harm key
- EG003
- source section id
- NCT04304144-ae
- event group key
- EG003
- event group label
- Overall (Part A): DLT + Continued Treatment Period: CAEL-101 Combined With SoC CyBorD
- serious affected
- 9
- serious at risk
- 13
- other affected
- 13
- other at risk
- 13
- frequency threshold
- 5
- collection period
- 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)
- event definition
- 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.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04304144
- harm key
- EG004
- source section id
- NCT04304144-ae
- event group key
- EG004
- event group label
- Continued Treatment Period (Part B): CAEL-101 Combined With SoC CyBorD and Daratumumab
- serious affected
- 7
- serious at risk
- 12
- other affected
- 11
- other at risk
- 12
- frequency threshold
- 5
- collection period
- 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)
- event definition
- 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.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- quality of life
- setting source links
Preserved source evidence · Not medical advice