Clinical trial NCT04162470: REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.
Official title: An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: PNH
Lead sponsor: Regeneron Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2019-11-14
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: Key Inclusion Criteria:

• 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:

* 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

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
timeFrame: Baseline up to Week 104
measure: Percentage of Participants Who Achieved Lactate Dehydrogenase (LDH) Less Than or Equal to (≤) 1.5* ULN From Baseline to Week 26
timeFrame: Baseline up to Week 26
Secondary outcomes:
measure: Percentage of Participants Who Had Breakthrough Hemolysis Through Week 26 and 78
timeFrame: At Week 26 and 78
measure: Overall Rate of Transfusion With Red Blood Cell (RBCs) Through Week 26
timeFrame: Baseline up to Week 26
measure: Percentage of Participants Who Are Transfusion-free (With RBCs) Through Week 26 and 78
timeFrame: At Week 26 and 78
measure: Percentage of Participants Who Achieved Adequate Control of Intravascular Hemolysis Through Week 78
timeFrame: Baseline up to Week 78
measure: Percentage of Participants Who Achieved Normalization of Intravascular Hemolysis Through Week 26 and Week 78
timeFrame: Baseline, Week 26 and 78
measure: Changes From Baseline in LDH Levels at Week 26, 78, and 104
timeFrame: Baseline, Week 26, 78, and 104
measure: Percent Change From Baseline in LDH Levels at Week 26, 78, and 104
timeFrame: Baseline, Week 26, 78, and 104
measure: Change From Baseline in Red Blood Cell (RBC) Hemoglobin Levels at Week 26, 78, and 104
timeFrame: Baseline, Week 26, 78, and 104
measure: Change From Baseline in Free Hemoglobin Levels at Week 26, 78 and 104
timeFrame: Baseline, Week 26, 78 and 104
measure: Serum Concentrations of Total REGN3918
timeFrame: Pre-dose (Day 1), End of infusion at Week 13, 26, 39, 52, 65, 78, 91 and 104
measure: Number of Participants With Treatment-emergent Anti-Drug Antibodies (ADA) to REGN3918
timeFrame: Baseline up to Week 104
Other outcomes:
Locations:
city: Sha Tin
country: Hong Kong
facility: Regeneron Study Site
city: Budapest
country: Hungary
facility: Regeneron Study Site
city: Busan
country: Korea, Republic of
facility: Regeneron Study Site
city: Seoul
country: Korea, Republic of
facility: Regeneron Study Site
city: Seoul
country: Korea, Republic of
facility: Regeneron Study Site
city: Seoul
country: Korea, Republic of
facility: Regeneron Study Site
city: Seoul
country: Korea, Republic of
facility: Regeneron Study Site
city: Seoul
country: Korea, Republic of
facility: Regeneron Study Site
city: Miri
country: Malaysia
facility: Regeneron Study Site
state: Sarawak
city: Sibu
country: Malaysia
facility: Regeneron Study Site
state: Sarawak
city: Kuala Terengganu
country: Malaysia
facility: Regeneron Study Site
state: Terengganu
city: Taipei City
country: Taiwan
facility: Regeneron Study Site
city: Taoyuan City
country: Taiwan
facility: Regeneron Study Site
city: Leeds
country: United Kingdom
facility: Regeneron Study Site
Study officials:
affiliation: Regeneron Pharmaceuticals
name: Clinical Trial Management
role: STUDY_DIRECTOR
References: