REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.

NCT04162470 | Terminated Phase 3 Results FDA Drug

• 2 other identifiers: R3918-PNH-18682019-000130-20
• Study Type: interventional
• Target: 24
• Locations: 6 countries
• Sites: 14

Brief 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). 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

Keywords

PNH

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Baseline up to Week 26

Secondary Outcomes (11)

• Percentage of Participants Who Had Breakthrough Hemolysis Through Week 26 and 78

  At Week 26 and 78

• Overall Rate of Transfusion With Red Blood Cell (RBCs) Through Week 26

  Baseline up to Week 26

• Percentage of Participants Who Are Transfusion-free (With RBCs) Through Week 26 and 78

  At Week 26 and 78

• Percentage of Participants Who Achieved Adequate Control of Intravascular Hemolysis Through Week 78

  Baseline up to Week 78

• Percentage of Participants Who Achieved Normalization of Intravascular Hemolysis Through Week 26 and Week 78

  Baseline, Week 26 and 78

Study Arms (1)

REGN3918

EXPERIMENTAL

Participants who have completed 1 of the 2 parent studies (R3918-PNH-1852 \[NCT03946748\] or R3918-PNH-1853)

Drug: REGN3918

Interventions

REGN3918 DRUG

Subcutaneous (SC) every week (QW) over the treatment period

Also known as: Pozelimab

REGN3918

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (14)

Regeneron Study Site

Shatin, Hong Kong

Location

Regeneron Study Site

Budapest, 1907, Hungary

Location

Regeneron Study Site

Miri, Sarawak, 98000, Malaysia

Location

Regeneron Study Site

Sibu, Sarawak, 96000, Malaysia

Location

Regeneron Study Site

Kuala Terengganu, Terengganu, 20400, Malaysia

Location

Regeneron Study Site

Busan, 49241, South Korea

Location

Regeneron Study Site

Seoul, 03080, South Korea

Location

Regeneron Study Site

Seoul, 03722, South Korea

Location

Regeneron Study Site

Seoul, 05030, South Korea

Location

Regeneron Study Site

Seoul, 06351, South Korea

Location

Regeneron Study Site

Seoul, 07985, South Korea

Location

Regeneron Study Site

Taipei, 10002, Taiwan

Location

Regeneron Study Site

Taoyuan, 333, Taiwan

Location

Regeneron Study Site

Leeds, LS97TF, United Kingdom

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Results Point of Contact

• Title: Clinical Trials Administrator
• Organization: Regeneron Pharmaceuticals, Inc

clinicaltrials@regeneron.com

844-734-6643

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 11, 2019
• First Posted: November 14, 2019
• Study Start: December 3, 2019
• Primary Completion: April 7, 2022
• Study Completion: April 7, 2022
• Last Updated: June 12, 2023
• Results First Posted: June 12, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.
• Access Criteria: Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency \[EMA\], Pharmaceuticals and Medical Devices Agency \[PMDA\], etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).

Locations

MY (3); HK (1); HU (1); KR (6); TW (2); GB (1)