TriangleNCT04512235 · STUDY IN CONTEXT
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
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
Time to all-cause mortality was defined as the number of weeks from the date of randomization to the date of death if it is on or before the end of PETP. Participants alive at the end of PETP (LPI+18 months) were censored at their last known alive date recorded within the PETP. CVHs were events adjudicated and confirmed as such by the Clinical Event Adjudication Committee (CEAC). Hypothesis testing with the Finkelstein-Schoenfeld test and treatment effect estimation with the win-ratio method based on pairwise comparisons of participant outcomes with comparisons performed in a hierarchical manner. To calculate win ratio, participant outcomes based on time to all-cause mortality were first compared and if there was no 'winner' due to censoring then a comparison based on frequency of cardiovascular hospitalization was made. A win ratio \>1 represents a more favorable outcome for CAEL-101 over placebo.
An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product, at any dose, that was not necessarily related to the treatment. A TEAE was an AE with an occurrence defined as follows: last study intervention day-first study intervention day + 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.
The KCCQ-OS is a 23-item self-administered questionnaire that quantifies physical function, symptoms, social function, self-efficacy and knowledge and quality of life. It uses an ordinal, adjectival (Likert) scale. Participants provide their level of agreement or disagreement with an agree-disagree scale for a series of statements. The questionnaire captures how the participants feel physically. The overall score was calculated as the sum from all 23 items and ranges from 0 to 100, where higher scores reflected better health status (fewer symptoms, fewer social or physical limitations, and better quality of life).
The 6MWT measures the distance a participant can quickly walk on a flat, hard surface in a period of 6 minutes. It evaluates the global and integrated response of all the systems involved during exercise. This is a self-paced test; participants choose their own intensity of exercise and are allowed to stop and rest during the test.
All-cause mortality was defined as death on or before the end of the PETP. Otherwise, participants who were alive at the end of the PETP (last participant randomized plus 18 months) were censored at their last known alive date recorded within the PETP. All deaths, regardless of causality or whether they were reported as a reason for study discontinuation, are reported in the All-Cause Mortality section.
CVHs were those events that were adjudicated and confirmed as such by the CEAC. Frequency of CVH was calculated using negative binomial regression analysis with study intervention, study, geographic region, daratumumab usage at Baseline, and offset term equal to log of each participant's follow-up time for cardiovascular hospitalization included in the model.
Reported safety
Quality of life and patient-reported measures
Unnamed quality-of-life measure · Change From Baseline in the Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)
Score direction: Higher is better, according to the source. Direction is never inferred from an instrument name.
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- qol key
- qol-0002
- source section id
- NCT04512235-om-0002
- source outcome index
- 2
- instrument
- Unnamed quality-of-life measure
- instrument basis
- matched 'quality of life' in the study's own wording
- scale title
- Change From Baseline in the Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)
- measure definition
- The KCCQ-OS is a 23-item self-administered questionnaire that quantifies physical function, symptoms, social function, self-efficacy and knowledge and quality of life. It uses an ordinal, adjectival (Likert) scale. Participants provide their level of agreement or disagreement with an agree-disagree scale for a series of statements. The questionnaire captures how the participants feel physically. The overall score was calculated as the sum from all 23 items and ranges from 0 to 100, where higher scores reflected better health status (fewer symptoms, fewer social or physical limitations, and better quality of life).
- time frame
- Baseline, Week 50
- analysis role
- secondary
- reporting status
- POSTED
- higher is better
- 1
- direction quote
- higher scores reflected better
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'. Here, 'Overall Number of Participants Analyzed' and 'Number Analyzed' signifies those participants evaluable for this outcome measure.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
SF-36 / SF-12 · Change From Baseline in the Short Form-36 (SF-36) Version 2 (v2) Physical Component Score (PCS)
Score direction: Not stated by the study. Direction is never inferred from an instrument name.
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- qol key
- qol-0007
- source section id
- NCT04512235-om-0007
- source outcome index
- 7
- instrument
- SF-36 / SF-12
- instrument basis
- matched 'sf-36' in the study's own wording
- scale title
- Change From Baseline in the Short Form-36 (SF-36) Version 2 (v2) Physical Component Score (PCS)
- measure definition
- The SF-36v2 is a self-administered questionnaire containing 36 items that measure health on functional status, well-being, and overall evaluation of health in 8 domains, including vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health, using scaled, ordinal responses (for example, All of the time, Most of the time, A good bit of the time). The SF-36v2 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale, with higher scores indicating an increased quality of life. The 8 multi-item scales are aggregated and summed to give the final PCS. The final score ranges from 0-100, with higher scores indicating an increased quality of life.
- time frame
- Baseline, Week 50
- analysis role
- secondary
- reporting status
- POSTED
- higher is better
- Source null
- direction quote
- Source null
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported for Intent-to-Treat Set. Here, 'Overall Number of Participants Analyzed' signifies those participants evaluable for this outcome measure.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
Full draft applicability and source-link record
- trial
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- trial label
- A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
- 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
- 6
- harms Count
- 2
- qol Count
- 2
- outcomes
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0000
- source section id
- NCT04512235-om-0000
- source outcome index
- 0
- endpoint
- A Hierarchical Combination of Time to All-cause Mortality and Frequency of Cardiovascular Hospitalizations (CVHs) Analyzed by Win Ratio
- endpoint definition
- Time to all-cause mortality was defined as the number of weeks from the date of randomization to the date of death if it is on or before the end of PETP. Participants alive at the end of PETP (LPI+18 months) were censored at their last known alive date recorded within the PETP. CVHs were events adjudicated and confirmed as such by the Clinical Event Adjudication Committee (CEAC). Hypothesis testing with the Finkelstein-Schoenfeld test and treatment effect estimation with the win-ratio method based on pairwise comparisons of participant outcomes with comparisons performed in a hierarchical manner. To calculate win ratio, participant outcomes based on time to all-cause mortality were first compared and if there was no 'winner' due to censoring then a comparison based on frequency of cardiovascular hospitalization was made. A win ratio \>1 represents a more favorable outcome for CAEL-101 over placebo.
- time origin
- Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, the primary analysis for the study was conducted 18-49 months after the study randomization.)
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'. For the win-ratio analysis, comparisons were made between participants in the 'CAEL-101 + Plasma Cell Dyscrasia Treatment' arm and the 'Placebo + Plasma Cell Dyscrasia Treatment' arm of each reporting group.
- 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
- NCT04512235
- outcome key
- om-0001
- source section id
- NCT04512235-om-0001
- source outcome index
- 1
- endpoint
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)
- endpoint definition
- An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product, at any dose, that was not necessarily related to the treatment. A TEAE was an AE with an occurrence defined as follows: last study intervention day-first study intervention day + 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
- Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, the primary analysis for the study was conducted 18-49 months after the study randomization.)
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Safety Set: all randomized participants who received at least 1 dose of the study intervention. As prespecified, data collected and reported as 'Overall (All Safety Set Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'.
- 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
- NCT04512235
- outcome key
- om-0002
- source section id
- NCT04512235-om-0002
- source outcome index
- 2
- endpoint
- Change From Baseline in the Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)
- endpoint definition
- The KCCQ-OS is a 23-item self-administered questionnaire that quantifies physical function, symptoms, social function, self-efficacy and knowledge and quality of life. It uses an ordinal, adjectival (Likert) scale. Participants provide their level of agreement or disagreement with an agree-disagree scale for a series of statements. The questionnaire captures how the participants feel physically. The overall score was calculated as the sum from all 23 items and ranges from 0 to 100, where higher scores reflected better health status (fewer symptoms, fewer social or physical limitations, and better quality of life).
- time origin
- Baseline, Week 50
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'. Here, 'Overall Number of Participants Analyzed' and 'Number Analyzed' signifies those participants evaluable for this outcome measure.
- 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
- NCT04512235
- outcome key
- om-0003
- source section id
- NCT04512235-om-0003
- source outcome index
- 3
- endpoint
- Change From Baseline in Distance Walked During a Six-minute Walk Test (6MWT)
- endpoint definition
- The 6MWT measures the distance a participant can quickly walk on a flat, hard surface in a period of 6 minutes. It evaluates the global and integrated response of all the systems involved during exercise. This is a self-paced test; participants choose their own intensity of exercise and are allowed to stop and rest during the test.
- time origin
- Baseline, Week 50
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'. Here, 'Overall Number of Participants Analyzed' and 'Number Analyzed' signifies those participants evaluable for this outcome measure.
- 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
- NCT04512235
- outcome key
- om-0004
- source section id
- NCT04512235-om-0004
- source outcome index
- 4
- endpoint
- All-cause Mortality
- endpoint definition
- All-cause mortality was defined as death on or before the end of the PETP. Otherwise, participants who were alive at the end of the PETP (last participant randomized plus 18 months) were censored at their last known alive date recorded within the PETP. All deaths, regardless of causality or whether they were reported as a reason for study discontinuation, are reported in the All-Cause Mortality section.
- time origin
- Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, analysis for the study was conducted 18-49 months after the study randomization.)
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'.
- 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
- NCT04512235
- outcome key
- om-0005
- source section id
- NCT04512235-om-0005
- source outcome index
- 5
- endpoint
- Frequency of CVHs
- endpoint definition
- CVHs were those events that were adjudicated and confirmed as such by the CEAC. Frequency of CVH was calculated using negative binomial regression analysis with study intervention, study, geographic region, daratumumab usage at Baseline, and offset term equal to log of each participant's follow-up time for cardiovascular hospitalization included in the model.
- time origin
- Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, analysis for the study was conducted 18-49 months after the study randomization.)
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'.
- 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
- NCT04512235
- outcome key
- om-0000
- arm key
- OG000
- arm label
- Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment
- regimen
- In addition to SoC plasma cell dyscrasia treatment, participants were administered either CAEL-101 or placebo as an IV infusion over approximately 2 hours until LPI+18 months.
- analyzed count
- 281
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0000#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0001
- arm key
- OG000
- arm label
- CAEL-101 + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered CAEL-101 as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 187
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0001#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0001
- arm key
- OG001
- arm label
- Placebo + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered placebo as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 94
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0001#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0002
- arm key
- OG000
- arm label
- CAEL-101 + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered CAEL-101 as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 120
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0002#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0002
- arm key
- OG001
- arm label
- Placebo + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered placebo as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 58
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0002#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0003
- arm key
- OG000
- arm label
- CAEL-101 + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered CAEL-101 as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 123
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0003#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0003
- arm key
- OG001
- arm label
- Placebo + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered placebo as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 56
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0003#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0004
- arm key
- OG000
- arm label
- CAEL-101 + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered CAEL-101 as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 187
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0004#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0004
- arm key
- OG001
- arm label
- Placebo + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered placebo as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 94
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0004#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0005
- arm key
- OG000
- arm label
- CAEL-101 + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered CAEL-101 as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 187
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0005#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- outcome key
- om-0005
- arm key
- OG001
- arm label
- Placebo + Plasma Cell Dyscrasia Treatment
- regimen
- During the PETP, participants were administered placebo as an IV infusion over approximately 2 hours, in addition to SoC plasma cell dyscrasia treatment. All participants continued their double-blind treatment until LPI+18 months. All participants had the option to enter into the OLEP, where they continued to receive CAEL-101 in addition to any SoC prescribed by a physician.
- analyzed count
- 94
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT04512235-om-0005#groups/OG001
- harms
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- harm key
- EG000
- source section id
- NCT04512235-ae
- event group key
- EG000
- event group label
- CAEL-101 + Plasma Cell Dyscrasia Treatment
- serious affected
- 132
- serious at risk
- 187
- other affected
- 179
- other at risk
- 187
- frequency threshold
- 5
- collection period
- Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, safety analysis for the study was conducted 18-49 months after the study randomization.)
- event definition
- Serious and non-serious adverse events based upon the Safety Set: all randomized participants who received at least 1 dose of study intervention. All-cause mortality based upon the Intent-to-treat Set: all participants who were randomized. All deaths, regardless of causality or whether they were reported as a reason for study discontinuation, are reported in the All-Cause Mortality section. Only deaths indicated as leading to study discontinuation are reported in the Participant Flow section.
- 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
- NCT04512235
- harm key
- EG001
- source section id
- NCT04512235-ae
- event group key
- EG001
- event group label
- Placebo + Plasma Cell Dyscrasia Treatment
- serious affected
- 68
- serious at risk
- 94
- other affected
- 91
- other at risk
- 94
- frequency threshold
- 5
- collection period
- Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, safety analysis for the study was conducted 18-49 months after the study randomization.)
- event definition
- Serious and non-serious adverse events based upon the Safety Set: all randomized participants who received at least 1 dose of study intervention. All-cause mortality based upon the Intent-to-treat Set: all participants who were randomized. All deaths, regardless of causality or whether they were reported as a reason for study discontinuation, are reported in the All-Cause Mortality section. Only deaths indicated as leading to study discontinuation are reported in the Participant Flow section.
- 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
- release id
- clinical-indications-draft-1
- disease state id
- ds-al-amyloidosis
- nct id
- NCT04512235
- qol key
- qol-0002
- source section id
- NCT04512235-om-0002
- source outcome index
- 2
- instrument
- Unnamed quality-of-life measure
- instrument basis
- matched 'quality of life' in the study's own wording
- scale title
- Change From Baseline in the Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)
- measure definition
- The KCCQ-OS is a 23-item self-administered questionnaire that quantifies physical function, symptoms, social function, self-efficacy and knowledge and quality of life. It uses an ordinal, adjectival (Likert) scale. Participants provide their level of agreement or disagreement with an agree-disagree scale for a series of statements. The questionnaire captures how the participants feel physically. The overall score was calculated as the sum from all 23 items and ranges from 0 to 100, where higher scores reflected better health status (fewer symptoms, fewer social or physical limitations, and better quality of life).
- time frame
- Baseline, Week 50
- analysis role
- secondary
- reporting status
- POSTED
- higher is better
- 1
- direction quote
- higher scores reflected better
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'. Here, 'Overall Number of Participants Analyzed' and 'Number Analyzed' signifies those participants evaluable for this outcome measure.
- 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
- NCT04512235
- qol key
- qol-0007
- source section id
- NCT04512235-om-0007
- source outcome index
- 7
- instrument
- SF-36 / SF-12
- instrument basis
- matched 'sf-36' in the study's own wording
- scale title
- Change From Baseline in the Short Form-36 (SF-36) Version 2 (v2) Physical Component Score (PCS)
- measure definition
- The SF-36v2 is a self-administered questionnaire containing 36 items that measure health on functional status, well-being, and overall evaluation of health in 8 domains, including vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health, using scaled, ordinal responses (for example, All of the time, Most of the time, A good bit of the time). The SF-36v2 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale, with higher scores indicating an increased quality of life. The 8 multi-item scales are aggregated and summed to give the final PCS. The final score ranges from 0-100, with higher scores indicating an increased quality of life.
- time frame
- Baseline, Week 50
- analysis role
- secondary
- reporting status
- POSTED
- higher is better
- Source null
- direction quote
- Source null
- population note
- Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported for Intent-to-Treat Set. Here, 'Overall Number of Participants Analyzed' signifies those participants evaluable for this outcome measure.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- setting source links
Preserved source evidence · Not medical advice