Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04162470
Recruitment Status : Enrolling by invitation
First Posted : November 14, 2019
Last Update Posted : April 15, 2020
Sponsor:
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

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

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: REGN3918 Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 147 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Actual Study Start Date : December 3, 2019
Estimated Primary Completion Date : August 28, 2023
Estimated Study Completion Date : August 28, 2023


Arm Intervention/treatment
Experimental: REGN3918
Participants who have completed 1 of the 2 parent studies (R3918-PNH-1852 [NCT03946748] or R3918-PNH-1853)
Drug: REGN3918
Subcutaneous (SC) every week (QW) over the treatment period




Primary Outcome Measures :
  1. Incidence and severity of treatment-emergent adverse events (TEAEs) [ Time Frame: Up to week 104 ]
    TEAEs include adverse events (AEs), serious adverse events (SAEs), AEs of special interest (AESIs), laboratory data, vital signs, and electrocardiograms (ECGs)

  2. Proportion of patients achieving lactate dehydrogenase (LDH) less than or equal to 1.5ULN [ Time Frame: Up to week 26 ]

Secondary Outcome Measures :
  1. Proportion of patients with breakthrough hemolysis [ Time Frame: Up to week 26, 78, 104 ]
  2. Rate/number of units of transfusion [ Time Frame: Up to week 26, 78, 104 ]
  3. Proportions of patients who are transfusion-free (with red blood cell [RBCs]) [ Time Frame: Up to week 26, 78, 104 ]
  4. Proportions of patients achieving adequate control of their intravascular hemolysis [ Time Frame: Up to week 78, 104 ]
  5. Proportions of patients achieving normalization of their intravascular hemolysis [ Time Frame: Up to week 26, 78, 104 ]
  6. Changes in LDH from baseline of the Open-label extension (OLE) study [ Time Frame: Up to week 26, 78, 104 ]
  7. Percent changes in LDH from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
  8. Changes in RBC hemoglobin levels from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
  9. Changes in free hemoglobin levels from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
  10. Concentrations of REGN3918 in serum [ Time Frame: Up to week 104 ]
  11. Incidence of treatment-emergent anti-drug antibodies (ADA) to REGN3918 [ Time Frame: Up to week 104 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04162470


Locations
Layout table for location information
Korea, Republic of
Regeneron Study Site
Seoul, Korea, Republic of, 03080
Regeneron Study Site
Seoul, Korea, Republic of, 03722
Regeneron Study Site
Seoul, Korea, Republic of, 05030
Regeneron Study Site
Seoul, Korea, Republic of, 06351
Sponsors and Collaborators
Regeneron Pharmaceuticals
Investigators
Layout table for investigator information
Study Director: Clinical Trial Management Regeneron Pharmaceuticals
Layout table for additonal information
Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04162470    
Other Study ID Numbers: R3918-PNH-1868
2019-000130-20 ( EudraCT Number )
First Posted: November 14, 2019    Key Record Dates
Last Update Posted: April 15, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (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).
URL: https://vivli.org/

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Regeneron Pharmaceuticals:
PNH
Additional relevant MeSH terms:
Layout table for MeSH terms
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases