Trial Outcomes & Findings for Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (NCT NCT05131204)
NCT ID: NCT05131204
Last Updated: 2025-04-08
Results Overview
TERMINATED
PHASE3
3 participants
From baseline to week 36
2025-04-08
Participant Flow
A total of 140 participants were expected to be enrolled, however, due to feasibility: five participants were screened and 3 were randomized and treated. There were 2 screen failures.
Participant milestones
| Measure |
Pozelimab and Cemdisiran
Randomized 1:1
|
Anti-C5 Standard-of-care
Randomized 1:1
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
1
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
Pozelimab and Cemdisiran
Randomized 1:1
|
Anti-C5 Standard-of-care
Randomized 1:1
|
|---|---|---|
|
Overall Study
Sponsor request
|
2
|
1
|
Baseline Characteristics
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
Baseline characteristics by cohort
| Measure |
Pozelimab and Cemdisiran
n=2 Participants
Randomized 1:1
|
Anti-C5 Standard-of-care
n=1 Participants
Randomized 1:1
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
White
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: From baseline to week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Participants who do not receive a red blood cell (RBC) transfusion as per protocol algorithm based on post baseline hemoglobin values
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Participants who do not receive an RBC transfusion as per protocol algorithm based on post baseline hemoglobin values
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4 (day 29) through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4 (day 29) through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 8 (day 57) through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Percentage of participants with adequate control of LDH as defined in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Percentage of participants with adequate control of LDH as defined in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 8 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Percentage of participants with adequate control of LDH as defined in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Percentage of participants with adequate control of LDH as defined in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 8 (day 57) through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Percentage of participants with normalization of LDH as defined in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
Percentage of participants with normalization of LDH as defined in the protocol
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From baseline to week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
The FACIT-Fatigue is a 13 item, self-administered clinical outcome assessment (COA) assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-Fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From baseline to week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From baseline to week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..
EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Per protocol algorithm
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Per protocol algorithm
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Day 1 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Per protocol algorithm
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 4 through week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Per protocol algorithm
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From baseline to week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Per protocol algorithm
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to week 29Treatment period and safety follow up period
Outcome measures
| Measure |
Pozelimab and Cemdisiran
n=2 Participants
Randomized 1:1
|
Anti-C5 Standard-of-care
n=1 Participants
Randomized 1:1
|
|---|---|---|
|
Incidence of Treatment Emergent Serious Adverse Events (SAEs)
|
0 Events
|
0 Events
|
SECONDARY outcome
Timeframe: Up to week 29Treatment period and safety follow up period
Outcome measures
| Measure |
Pozelimab and Cemdisiran
n=2 Participants
Randomized 1:1
|
Anti-C5 Standard-of-care
n=1 Participants
Randomized 1:1
|
|---|---|---|
|
Incidence of Treatment-emergent Adverse Events (TEAEs) of Special Interest
|
1 Events
|
0 Events
|
SECONDARY outcome
Timeframe: Up to week 29Treatment period and safety follow up period
Outcome measures
| Measure |
Pozelimab and Cemdisiran
n=2 Participants
Randomized 1:1
|
Anti-C5 Standard-of-care
n=1 Participants
Randomized 1:1
|
|---|---|---|
|
Incidence of TEAEs Leading to Treatment Discontinuation
|
0 Events
|
0 Events
|
SECONDARY outcome
Timeframe: From baseline to week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From baseline to week 36Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Through week 62Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Treatment period and safety follow up period
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Through week 62Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Treatment period and safety follow up period
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Through week 32Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Treatment period
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Through week 40Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Treatment period
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Through week 44Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Treatment period
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Through week 62Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Treatment period and safety follow up period
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Through week 62Population: Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.
Treatment period and safety follow up period
Outcome measures
Outcome data not reported
Adverse Events
Pozelimab and Cemdisiran
Anti-C5 Standard-of-care
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Pozelimab and Cemdisiran
n=2 participants at risk
Randomized 1:1
|
Anti-C5 Standard-of-care
n=1 participants at risk
Randomized 1:1
|
|---|---|---|
|
General disorders
Injection site rash
|
50.0%
1/2 • Number of events 1 • From first dose to week 29
|
0.00%
0/1 • From first dose to week 29
|
|
Investigations
Blood glucose increased
|
50.0%
1/2 • Number of events 1 • From first dose to week 29
|
0.00%
0/1 • From first dose to week 29
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/2 • From first dose to week 29
|
100.0%
1/1 • Number of events 1 • From first dose to week 29
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.00%
0/2 • From first dose to week 29
|
100.0%
1/1 • Number of events 1 • From first dose to week 29
|
Additional Information
Clinical Trials Administrator
Regeneron Pharmaceuticals, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator has the right to independently publish study results from the investigator's site after a multi-center publication, or a defined period after the completion of the study by all sites. The investigator must provide the sponsor a copy of any such publication derived from the study for review and comment in advance of any submission, and delay publication, if requested, to allow the Sponsor to preserve its proprietary rights.
- Publication restrictions are in place
Restriction type: OTHER