Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT04162470
REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.
A Phase 3 study testing REGN3918 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Regeneron Pharmaceuticals at 14 sites in 6 countries.
Trial summary
The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- REGN3918
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 14 sitesin 6 countries
- Study dates
- 3 Dec 2019to 7 Apr 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with PNH who have completed, without discontinuation, study treatment in one of the parent studies in which they participated (either R3918-PNH-1852 [NCT03946748] or R3918-PNH-1853)
Who cannot join
- Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient (for example, repetitive non-compliance with dosing by the patient)
- Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study
- NOTE: Other protocol defined exclusion criteria apply
Trial locations and contacts
14 sites in 6 countries.
14 sites in 6 countries
South Korea6 sites
Regeneron Study Site
Busan, 49241
Regeneron Study Site
Seoul, 03080
Regeneron Study Site
Seoul, 03722
Regeneron Study Site
Seoul, 05030
Regeneron Study Site
Seoul, 06351
Regeneron Study Site
Seoul, 07985
Malaysia3 sites
Regeneron Study Site
Kuala Terengganu, Terengganu, 20400
Regeneron Study Site
Miri, Sarawak, 98000
Regeneron Study Site
Sibu, Sarawak, 96000
Taiwan2 sites
Regeneron Study Site
Taipei, 10002
Regeneron Study Site
Taoyuan City, 333
About this study
Official title: An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria
The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
The secondary objectives of the study are:
- To evaluate the long-term effect of REGN3918 on intravascular hemolysis
- To assess the concentrations of total REGN3918 in serum
- To evaluate the occurrence of the immunogenicity of REGN3918
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- REGN3918
- Keywords
- PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
REGN3918Drug
Also called Pozelimab
Subcutaneous (SC) every week (QW) over the treatment period
| Study group | Role | What they receive |
|---|---|---|
| REGN3918 | Experimental | Participants who have completed 1 of the 2 parent studies (R3918-PNH-1852 [NCT03946748] or R3918-PNH-1853) Receives: Drug: REGN3918 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
An adverse event (AE) was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs was defined as AEs that developed or worsened during the on-treatment period. SAE was defined as any untoward medical occurrence that resulted in any of following outcomes: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. TEAEs included both Serious TEAEs and non-serious TEAEs.
Measured: Baseline up to Week 104
Percentage of Participants Who Achieved Lactate Dehydrogenase (LDH) Less Than or Equal to (≤) 1.5* ULN From Baseline to Week 26
Percentage of participants who achieved LDH ≤1.5* Upper limit of normal (ULN) over Week 26, defined as LDH ≤1.5*ULN from baseline up to Week 26 were reported. A participant was considered to have met the criteria for adequate control of intravascular hemolysis if all of their LDH readings from the baseline through Week 26 inclusive or through the analysis end date, whichever is earlier, had values ≤ 1.5*ULN.
Measured: Baseline up to Week 26
Secondary outcomes (11)
Percentage of Participants Who Had Breakthrough Hemolysis Through Week 26 and 78
A participant was considered to have breakthrough hemolysis if he/she had any LDH measurement greater than or equal to (≥) 2*ULN, concomitant with associated signs or symptoms at any time subsequent to an initial achievement of disease control (i.e., LDH ≤ 1.5* ULN).
Measured: At Week 26 and 78
Overall Rate of Transfusion With Red Blood Cell (RBCs) Through Week 26
The overall rate of transfusion for a participant was calculated based on the duration of treatment exposure of the participant.
Measured: Baseline up to Week 26
Percentage of Participants Who Are Transfusion-free (With RBCs) Through Week 26 and 78
Transfusion free was defined as not having received an RBC transfusion during the first 26 and 78 weeks. A transfusion was counted only if it was per-protocol, that is, if it follows the predefined transfusion algorithm: RBC transfusion due to a post-baseline hemoglobin level less than (<) 9 gram per deciliter (g/dL) (with anemia symptoms) or a post-baseline hemoglobin level < 7 g/dL (without anemia symptoms).
Measured: At Week 26 and 78
Percentage of Participants Who Achieved Adequate Control of Intravascular Hemolysis Through Week 78
A participant was considered to have met the criteria for adequate control of intravascular hemolysis if all of his/her LDH readings from the baseline through Week 78 inclusive or through the analysis end date, whichever is earlier, had values <=1.5* ULN. and must not have discontinued study treatment early.
Measured: Baseline up to Week 78
Percentage of Participants Who Achieved Normalization of Intravascular Hemolysis Through Week 26 and Week 78
A participant was considered to have met normalization of intravascular hemolysis if all of their LDH readings from the baseline through Week 26 or 78 inclusive, or through the analysis end date, whichever is earlier, had values ≤ 1.0*ULN.
Measured: Baseline, Week 26 and 78
Changes From Baseline in LDH Levels at Week 26, 78, and 104
Change from baseline in LDH levels at Week 26, 78, and 104 was reported. Reported baseline is from R3918-PNH-1852 study.
Measured: Baseline, Week 26, 78, and 104
Percent Change From Baseline in LDH Levels at Week 26, 78, and 104
Percent change from baseline in LDH levels at Week 26, 78, and 104 was reported. Reported baseline is from R3918-PNH-1852 study.
Measured: Baseline, Week 26, 78, and 104
Change From Baseline in Red Blood Cell (RBC) Hemoglobin Levels at Week 26, 78, and 104
Change from baseline in RBC hemoglobin levels at Week 26, 78, and 104 was reported.
Measured: Baseline, Week 26, 78, and 104
Change From Baseline in Free Hemoglobin Levels at Week 26, 78 and 104
Change from baseline in free hemoglobin levels at Week 26, 78 and 104 was reported.
Measured: Baseline, Week 26, 78 and 104
Serum Concentrations of Total REGN3918
Serum Concentrations of total REGN3918 was reported.
Measured: Pre-dose (Day 1), End of infusion at Week 13, 26, 39, 52, 65, 78, 91 and 104
Number of Participants With Treatment-emergent Anti-Drug Antibodies (ADA) to REGN3918
Number of Participants with treatment-emergent ADA response to REGN3918 was reported.
The ADA analysis set (AAS) includes all treated participants who received any amount of study drug (active [SAF]) and had at least 1 non missing anti pozelimab antibody result following the first dose of study drug. The AAS is based on the actual treatment received (as treated).
Measured: Baseline up to Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2019
- Study start3 Dec 2019
- Primary completion7 Apr 2022
- Study completion7 Apr 2022
Why it stopped: Business Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04162470
- Sponsor's protocol ID
- R3918-PNH-1868
- Other IDs
- 2019-000130-20, 2019
- Status last verified
- May 2023
- First submitted
- 11 Nov 2019
- Last updated
- 12 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT04162470
- Acronym / sponsor ID
- R3918-PNH-1868
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Business Decision
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 3 Dec 2019 (actual)
- Primary completion
- 7 Apr 2022 (actual)
- Study completion
- 7 Apr 2022 (actual)
- First posted
- 14 Nov 2019 (actual)
- Results
- Posted 12 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
The secondary objectives of the study are:
- To evaluate the long-term effect of REGN3918 on intravascular hemolysis
- To assess the concentrations of total REGN3918 in serum
- To evaluate the occurrence of the immunogenicity of REGN3918
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| REGN3918 | Experimental | Drug: REGN3918Participants who have completed 1 of the 2 parent studies (R3918-PNH-1852 [NCT03946748] or R3918-PNH-1853) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Patients with PNH who have completed, without discontinuation, study treatment in one of the parent studies in which they participated (either R3918-PNH-1852 [NCT03946748] or R3918-PNH-1853)
Key exclusion criteria (3 listed)
- Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient (for example, repetitive non-compliance with dosing by the patient)
- Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study
- NOTE: Other protocol defined exclusion criteria apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| REGN3918Also: PozelimabSubcutaneous (SC) every week (QW) over the treatment period | Drug | REGN3918 |
Primary outcome
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: Baseline up to Week 104
An adverse event (AE) was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs was defined as AEs that developed or worsened during the on-treatment period. SAE was defined as any untoward medical occurrence that resulted in any of following outcomes: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. TEAEs included both Serious TEAEs and non-serious TEAEs.
Percentage of Participants Who Achieved Lactate Dehydrogenase (LDH) Less Than or Equal to (≤) 1.5* ULN From Baseline to Week 26
Time frame: Baseline up to Week 26
Percentage of participants who achieved LDH ≤1.5* Upper limit of normal (ULN) over Week 26, defined as LDH ≤1.5*ULN from baseline up to Week 26 were reported. A participant was considered to have met the criteria for adequate control of intravascular hemolysis if all of their LDH readings from the baseline through Week 26 inclusive or through the analysis end date, whichever is earlier, had values ≤ 1.5*ULN.
11 secondary outcomes
Percentage of Participants Who Had Breakthrough Hemolysis Through Week 26 and 78
Time frame: At Week 26 and 78
Overall Rate of Transfusion With Red Blood Cell (RBCs) Through Week 26
Time frame: Baseline up to Week 26
Percentage of Participants Who Are Transfusion-free (With RBCs) Through Week 26 and 78
Time frame: At Week 26 and 78
Percentage of Participants Who Achieved Adequate Control of Intravascular Hemolysis Through Week 78
Time frame: Baseline up to Week 78
Percentage of Participants Who Achieved Normalization of Intravascular Hemolysis Through Week 26 and Week 78
Time frame: Baseline, Week 26 and 78
Changes From Baseline in LDH Levels at Week 26, 78, and 104
Time frame: Baseline, Week 26, 78, and 104
Percent Change From Baseline in LDH Levels at Week 26, 78, and 104
Time frame: Baseline, Week 26, 78, and 104
Change From Baseline in Red Blood Cell (RBC) Hemoglobin Levels at Week 26, 78, and 104
Time frame: Baseline, Week 26, 78, and 104
Change From Baseline in Free Hemoglobin Levels at Week 26, 78 and 104
Time frame: Baseline, Week 26, 78 and 104
Serum Concentrations of Total REGN3918
Time frame: Pre-dose (Day 1), End of infusion at Week 13, 26, 39, 52, 65, 78, 91 and 104
Number of Participants With Treatment-emergent Anti-Drug Antibodies (ADA) to REGN3918
Time frame: Baseline up to Week 104
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Management study director
Regeneron Pharmaceuticals
14 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing REGN3918 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Regeneron Pharmaceuticals at 14 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 12 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in South Korea, Malaysia, Taiwan, Hong Kong, Hungary and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs (time frame: Baseline up to Week 104). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jun 2023.