Trial · NCT04973137
A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis
NCT04973137, Phase 3, Terminated, Prothena Biosciences Ltd.; results first posted 2025-12-16
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis. Trials, record NCT04973137. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04973137
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis
- NCT number
- NCT04973137
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 208 participants, actual
- Lead sponsor
- Prothena Biosciences Ltd.
- Conditions
- Light Chain (AL) Amyloidosis
- Interventions
- Birtamimab, Placebo, Standard of Care Chemotherapy
- First posted
- 2021-07-22
- Last updated
- 2025-12-16
- Results first posted
- 2025-12-16
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time to All-cause Mortality for the Double Blind Phase | Time from the first dose of study drug until the pre-defined number of events (all-cause mortality) have been reached, assessed up to 39 months. | Comparison of time to all-cause mortality for birtamimab and placebo control. |
| Secondary | 6MWT Distance at Month 9 | Month 9 | Change from baseline to Month 9 in the 6-Minute Walk Test (6MWT) distance calculated as the value at 9 months minus the value at baseline. |
| Secondary | Physical Component Summary Score of the Short Form-36, Version 2 at Month 9 | Month 9 | Change from baseline to Month 9 in the Short Form-36, version 2 (SF-36) calculated as the value at 9 months minus the value at baseline. SF-36 v2 is a 36-item self-administered quality-of-life questionnaire that measures health on functional status, well-being, and overall evaluation of health. The Physical Component Summary score ranges from 0 to 100 with higher scores indicating higher health-related quality of life. The Physical Component Summary is derived primarily from questions regarding physical functioning, physical problems, bodily pain, and general health questions. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Time to All-cause Mortality for the Double Blind Phase | Time from the first dose of study drug until the pre-defined number of events (all-cause mortality) have been reached, assessed up to 39 months. | ITT Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | 6MWT Distance at Month 9 | Month 9 | ITT Population | Meters | MEDIAN |
| SECONDARY | Physical Component Summary Score of the Short Form-36, Version 2 at Month 9 | Month 9 | ITT Population | Score | MEDIAN |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Time to All-cause Mortality for the Double Blind Phase | Birtamimab (24 mg/kg) + Standard of Care | Event (Death) Occurred | 32 | Participants | |
| Time to All-cause Mortality for the Double Blind Phase | Placebo + Standard of Care | Event (Death) Occurred | 17 | Participants | |
| Time to All-cause Mortality for the Double Blind Phase | Birtamimab (24 mg/kg) + Standard of Care | Number of Subjects Censored | 107 | Participants | |
| Time to All-cause Mortality for the Double Blind Phase | Placebo + Standard of Care | Number of Subjects Censored | 51 | Participants | |
| 6MWT Distance at Month 9 | Birtamimab (24 mg/kg) + Standard of Care | 4.5 | Inter-Quartile Range -52.00 to 50.50 | Meters | |
| 6MWT Distance at Month 9 | Placebo + Standard of Care | 7.75 | Inter-Quartile Range -33.00 to 36.00 | Meters | |
| Physical Component Summary Score of the Short Form-36, Version 2 at Month 9 | Birtamimab (24 mg/kg) + Standard of Care | 1.710 | Inter-Quartile Range -2.695 to 6.920 | Score | |
| Physical Component Summary Score of the Short Form-36, Version 2 at Month 9 | Placebo + Standard of Care | 4.205 | Inter-Quartile Range -0.430 to 10.320 | Score |
Source ClinicalTrials.gov
Participant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double Blind Phase | STARTED | Birtamimab (24 mg/kg) + Standard of Care | 139 | |
| Double Blind Phase | STARTED | Placebo + Standard of Care | 68 | |
| Double Blind Phase | COMPLETED | Birtamimab (24 mg/kg) + Standard of Care | 97 | |
| Double Blind Phase | COMPLETED | Placebo + Standard of Care | 49 | |
| Double Blind Phase | NOT COMPLETED | Birtamimab (24 mg/kg) + Standard of Care | 42 | |
| Double Blind Phase | NOT COMPLETED | Placebo + Standard of Care | 19 | |
| Open Label Extension Phase | STARTED | Birtamimab (24 mg/kg) + Standard of Care | 92 | |
| Open Label Extension Phase | STARTED | Placebo + Standard of Care | 44 | |
| Open Label Extension Phase | COMPLETED | Birtamimab (24 mg/kg) + Standard of Care | 0 | |
| Open Label Extension Phase | COMPLETED | Placebo + Standard of Care | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events672
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 | Birtamimab (24 mg/kg) + Standard of Care (Double-Blind Phase) | 34 | 139 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo + Standard of Care (Double-Blind Phase) | 16 | 68 | |
| other | Anaemia | Blood and lymphatic system disorders | Birtamimab (24 mg/kg) + Standard of Care (Open Label Extension Phase) | 3 | 136 | |
| other | Neutropenia | Blood and lymphatic system disorders | Birtamimab (24 mg/kg) + Standard of Care (Double-Blind Phase) | 11 | 139 | |
| other | Neutropenia | Blood and lymphatic system disorders | Placebo + Standard of Care (Double-Blind Phase) | 6 | 68 | |
| other | Neutropenia | Blood and lymphatic system disorders | Birtamimab (24 mg/kg) + Standard of Care (Open Label Extension Phase) | 0 | 136 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Birtamimab (24 mg/kg) + Standard of Care (Double-Blind Phase) | 11 | 139 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Placebo + Standard of Care (Double-Blind Phase) | 5 | 68 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Birtamimab (24 mg/kg) + Standard of Care (Open Label Extension Phase) | 0 | 136 | |
| other | Cardiac failure | Cardiac disorders | Birtamimab (24 mg/kg) + Standard of Care (Double-Blind Phase) | 19 | 139 |
1 to 10 of 672Source ClinicalTrials.gov
See all 672 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Birtamimab (24 mg/kg) + Standard of Care (Double-Blind Phase) | 32 | 139 |
| Total, all-cause mortality | Placebo + Standard of Care (Double-Blind Phase) | 17 | 68 |
| Total, all-cause mortality | Birtamimab (24 mg/kg) + Standard of Care (Open Label Extension Phase) | 3 | 136 |
| Total, other adverse events | Birtamimab (24 mg/kg) + Standard of Care (Double-Blind Phase) | 136 | 139 |
| Total, other adverse events | Placebo + Standard of Care (Double-Blind Phase) | 66 | 68 |
| Total, other adverse events | Birtamimab (24 mg/kg) + Standard of Care (Open Label Extension Phase) | 53 | 136 |
| Total, serious adverse events | Birtamimab (24 mg/kg) + Standard of Care (Double-Blind Phase) | 83 | 139 |
| Total, serious adverse events | Placebo + Standard of Care (Double-Blind Phase) | 39 | 68 |
| Total, serious adverse events | Birtamimab (24 mg/kg) + Standard of Care (Open Label Extension Phase) | 17 | 136 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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.
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.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04973137
GET https://conductscience.org/api/trials/v1/NCT04973137/events?page=2