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Trial record 1 of 1 for:    NCT04085601
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A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH

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: NCT04085601
Recruitment Status : Completed
First Posted : September 11, 2019
Last Update Posted : December 1, 2021
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE September 9, 2019
First Posted Date  ICMJE September 11, 2019
Last Update Posted Date December 1, 2021
Actual Study Start Date  ICMJE August 15, 2019
Actual Primary Completion Date June 23, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 9, 2019)
  • Hemoglobin stabilization defined as avoidance of a > 1 g/dl decrease in hemoglobin levels in the absence of transfusion. [ Time Frame: Baseline through Week 26 ]
  • Reduction in lactate dehydrogenase (LDH) level [ Time Frame: Baseline to Week 26 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Official Title  ICMJE A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Brief Summary Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Paroxysmal Nocturnal Hemoglobinuria
Intervention  ICMJE Drug: APL-2
Complement (C3) Inhibitor
Study Arms  ICMJE
  • No Intervention: Standard of Care (SOC) excluding complement inhibitors
  • Experimental: 1,080mg APL-2 administered subcutaneously twice weekly
    Intervention: Drug: APL-2
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 26, 2020)
53
Original Estimated Enrollment  ICMJE
 (submitted: September 9, 2019)
54
Actual Study Completion Date  ICMJE June 23, 2021
Actual Primary Completion Date June 23, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Be at least 18 years old (inclusive).
  • Have LDH ≥1.5 x ULN at the screening visit.
  • Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone >10%).
  • Have Hb less than the lower limit of normal (LLN) at the screening visit.
  • Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
  • Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
  • Have a platelet count of >50,000/mm3 at the screening visit.
  • Have an absolute neutrophil count >500/mm3 at the screening visit.

Exclusion Criteria:

  • Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
  • Hereditary complement deficiency.
  • History of bone marrow transplantation.
  • Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:

    • Erythropoietin or immunosuppressants for at least 8 weeks
    • Systemic corticosteroids for at least 4 weeks
    • Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
    • Iron supplements, vitamin B12, or folic acid for at least 4 weeks
    • Low-molecular-weight heparin for at least 4 weeks
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Colombia,   Hong Kong,   Malaysia,   Mexico,   Peru,   Philippines,   Poland,   Serbia,   Singapore,   Thailand
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04085601
Other Study ID Numbers  ICMJE APL2-308
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Apellis Pharmaceuticals, Inc.
Study Sponsor  ICMJE Apellis Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Apellis Pharmaceuticals, Inc.
Verification Date November 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP