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Multi-center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (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: NCT03531255
Recruitment Status : Enrolling by invitation
First Posted : May 21, 2018
Last Update Posted : September 7, 2018
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE May 9, 2018
First Posted Date  ICMJE May 21, 2018
Last Update Posted Date September 7, 2018
Actual Study Start Date  ICMJE August 27, 2018
Estimated Primary Completion Date December 20, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 9, 2018)
Incidence and severity of hematologic treatment-emergent adverse events [ Time Frame: 48 Weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03531255 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 9, 2018)
Mean change from baseline to 48 weeks in hemoglobin levels (g/dL) [ Time Frame: 48 Weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Multi-center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official Title  ICMJE A Phase III, Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Brief Summary This is an Open Label, Non-Randomized, Multi-Center Extension Study for ongoing and planned Phase IB, Phase II, and Phase III studies with APL-2 in subjects with PNH.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE PNH
Intervention  ICMJE Drug: APL-2
Complement (C3) Inhibitor
Study Arms  ICMJE
  • Experimental: APL-2 1,080mg twice weekly
    1,080mg APL-2 will be administered subcutaneously twice weekly.
    Intervention: Drug: APL-2
  • Experimental: APL-2 1,080mg every three days
    1,080mg APL-2 will be administered subcutaneously every three days.
    Intervention: Drug: APL-2
  • Experimental: APL-2 1,080mg twice weekly or every three days
    1,080mg APL-2 will be administered subcutaneously twice weekly or 1,080mg APL-2 will be administered subcutaneously every three days.
    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 Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: May 9, 2018)
109
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 20, 2020
Estimated Primary Completion Date December 20, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Subjects with PNH who have participated in an APL-2 clinical trial, and have experienced clinical benefit in the opinion of the Investigator.
  2. Willing and able to give written informed consent.
  3. Women of child-bearing potential (WOCBP) must have a negative pregnancy test and must agree to use protocol defined methods of contraception for the duration of the study and 60 days after their last dose of study drug.
  4. Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study and 60 days after their last dose of study drug.

Exclusion Criteria:

  1. Subjects who have withdrawn from an APL-2 clinical study
  2. Any condition that could increase the subject's risk by participating in the study
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 United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03531255
Other Study ID Numbers  ICMJE APL2-307
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
Product Manufactured in and Exported from the U.S.: 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 September 2018

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