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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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02264639
First Posted: October 15, 2014
Last Update Posted: July 7, 2017
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.
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.
  Purpose
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.

Condition Intervention Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: APL-2 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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).

Resource links provided by NLM:


Further study details as provided by Apellis Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability. [ Time Frame: Five months ]

Secondary Outcome Measures:
  • PK Parameters [ Time Frame: Five months ]
    • Cmax
    • Maximum Concentration (Cmax)
    • Time to Cmax (Tmax)


Estimated Enrollment: 15
Study Start Date: November 2014
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort 1
First Dose 25mg, Repeated Dose 5 mg/day
Drug: APL-2
Experimental: Cohort 2
First Dose 50 mg, Repeated Dose 30 mg/day
Drug: APL-2
Experimental: Cohort 3
Repeated Dose 180 mg/day
Drug: APL-2
Experimental: Cohort 4
Repeated Dose 270 mg/day
Drug: APL-2
Experimental: Cohort 4 (Intra-subject)
optional intra-subject repeated dose 360mg/day
Drug: APL-2

  Eligibility

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.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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): NCT02264639


Locations
United States, California
Jane Anne Nohl Division of Hematology Keck-USC School of Medicine
Los Angeles, California, United States
United States, Florida
Lakes Research
Miami Lakes, Florida, United States
United States, Kentucky
University of Lousiville
Louisville, Kentucky, United States, 40202
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States
Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.
Investigators
Study Director: Federico Grossi, MD, PhD Apellis Pharmaceuticals, Inc.
  More Information

Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02264639     History of Changes
Other Study ID Numbers: APL-CP0514
First Submitted: October 8, 2014
First Posted: October 15, 2014
Last Update Posted: July 7, 2017
Last Verified: July 2017

Additional relevant 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