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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. Identifier: NCT02264639
Recruitment Status : Active, not recruiting
First Posted : October 15, 2014
Last Update Posted : July 2, 2018
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: APL-2 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
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).
Study Start Date : November 2014
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018

Arm Intervention/treatment
Experimental: Cohort 1
First Dose 25mg, Repeated Dose 5 mg/day
Drug: APL-2
Complement (C3) Inhibitor

Experimental: Cohort 2
First Dose 50 mg, Repeated Dose 30 mg/day
Drug: APL-2
Complement (C3) Inhibitor

Experimental: Cohort 3
Repeated Dose 180 mg/day
Drug: APL-2
Complement (C3) Inhibitor

Experimental: Cohort 4
Repeated Dose 270 mg/day
Drug: APL-2
Complement (C3) Inhibitor

Primary Outcome Measures :
  1. Number of Participants with Adverse Events as a Measure of Safety and Tolerability. [ Time Frame: Baseline to 24 months ]

Secondary Outcome Measures :
  1. Peak Plasma Concentration (Cmax) [ Time Frame: Baseline to 24 months ]
  2. Minimum Plasma Concentration (Cmin) [ Time Frame: Baseline to 24 months ]
  3. Time to peak plasma concentration (Tmax) [ Time Frame: Baseline to 24 months ]

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.

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

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.

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 identifier (NCT number): NCT02264639

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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.
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Study Director: Federico Grossi, MD, PhD Apellis Pharmaceuticals, Inc.

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Responsible Party: Apellis Pharmaceuticals, Inc. Identifier: NCT02264639     History of Changes
Other Study ID Numbers: APL-CP0514
First Posted: October 15, 2014    Key Record Dates
Last Update Posted: July 2, 2018
Last Verified: June 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemoglobinuria, Paroxysmal
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases