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A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects 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: NCT02264639
Recruitment Status : Active, not recruiting
First Posted : October 15, 2014
Last Update Posted : July 2, 2018
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE October 8, 2014
First Posted Date  ICMJE October 15, 2014
Last Update Posted Date July 2, 2018
Study Start Date  ICMJE November 2014
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 28, 2018)
Number of Participants with Adverse Events as a Measure of Safety and Tolerability. [ Time Frame: Baseline to 24 months ]
Original Primary Outcome Measures  ICMJE
 (submitted: October 10, 2014)
Number of Participants with Adverse Events as a Measure of Safety and Tolerability. [ Time Frame: Four months ]
Change History Complete list of historical versions of study NCT02264639 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 28, 2018)
  • Peak Plasma Concentration (Cmax) [ Time Frame: Baseline to 24 months ]
  • Minimum Plasma Concentration (Cmin) [ Time Frame: Baseline to 24 months ]
  • Time to peak plasma concentration (Tmax) [ Time Frame: Baseline to 24 months ]
Original Secondary Outcome Measures  ICMJE
 (submitted: October 10, 2014)
PK Parameters [ Time Frame: Four months ]
  • Cmax
  • Maximum Concentration (Cmax)
  • Time to Cmax (Tmax)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects With PNH
Official Title  ICMJE An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).
Brief Summary This study will be the initial exploration of APL-2 in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of APL-2 will guide decisions to further develop the drug.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Paroxysmal Nocturnal Hemoglobinuria (PNH)
Intervention  ICMJE Drug: APL-2
Complement (C3) Inhibitor
Study Arms  ICMJE
  • Experimental: Cohort 1
    First Dose 25mg, Repeated Dose 5 mg/day
    Intervention: Drug: APL-2
  • Experimental: Cohort 2
    First Dose 50 mg, Repeated Dose 30 mg/day
    Intervention: Drug: APL-2
  • Experimental: Cohort 3
    Repeated Dose 180 mg/day
    Intervention: Drug: APL-2
  • Experimental: Cohort 4
    Repeated Dose 270 mg/day
    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 Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: August 29, 2016)
15
Original Estimated Enrollment  ICMJE
 (submitted: October 10, 2014)
8
Estimated Study Completion Date  ICMJE December 2018
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Male or Female
  2. At least 18 years of age
  3. Weigh >55 kg
  4. Diagnosed with PNH
  5. On treatment with eculizumab (Soliris®) for at least 3 months
  6. Hb < 10 g/dL at screening OR have received at least one transfusion within 12 months prior to screening
  7. Platelet count of >30,000/mm3
  8. Absolute neutrophil count > 500/mm3
  9. Women of child-bearing potential (WOCBP) must have a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study (see below)
  10. Males with female partners of child bearing potential must agree to use protocol defined methods of contraception (see below) and agree to refrain from donating sperm for the duration of the study
  11. Willing and able to give informed consent

Exclusion Criteria:

  1. Active bacterial infection
  2. Known infection with hepatitis B, C or HIV
  3. Hereditary complement deficiency
  4. History of bone marrow transplantation
  5. Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
  6. Evidence of QTcF prolongation defined as > 450 ms for males and > 470 ms for females at screening
  7. Creatinine clearance (CrCl) < 50 mL/min (Cockcroft-Gault formula) at screening
  8. Breast-feeding women
  9. History of meningococcal disease
  10. No vaccination against N. meningitidis types A, C, W, Y and B (administered as two separate vaccinations), Pneumococcal conjugate vaccine or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23, respectively) and Haemophilus influenzae Type B (Hib) vaccination within 2 years prior to Day 1 (Visit 2) dosing.
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 NCT02264639
Other Study ID Numbers  ICMJE APL-CP0514
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
Study Director: Federico Grossi, MD, PhD Apellis Pharmaceuticals, Inc.
PRS Account Apellis Pharmaceuticals, Inc.
Verification Date June 2018

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