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Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With wAIHA or CAD

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ClinicalTrials.gov Identifier: NCT03226678
Recruitment Status : Recruiting
First Posted : July 24, 2017
Last Update Posted : October 9, 2018
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Brief Summary:
This study is to assess the safety, tolerability, preliminary efficacy, and pharmacokinetics of APL-2 in subjects with warm Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD).

Condition or disease Intervention/treatment Phase
Warm Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD) Drug: APL-2 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label, Prospective, Study to Assess the Safety, Tolerability, Efficacy and Pharmacokinetics of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)
Actual Study Start Date : August 31, 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: Cohort 1 wAIHA (n=12) Drug: APL-2
Complement (C3) Inhibitor

Experimental: Cohort 2 CAD (n=12) Drug: APL-2
Complement (C3) Inhibitor




Primary Outcome Measures :
  1. Total Number of AEs [ Time Frame: 12 months from baseline ]
    The primary safety endpoints of the study are the incidence and severity of treatment emergent adverse events (TEAEs) following administration of multiple doses of subcutaneous (SC) APL-2.

  2. Change from baseline in hemoglobin [ Time Frame: 6 months from baseline ]
    Efficacy endpoint assessment.

  3. Cmax [ Time Frame: 12 months from baseline ]
    APL-2 serum concentrations and pharmacokinetic (PK) parameter (Cmax)

  4. AUC [ Time Frame: 12 months from baseline ]
    APL-2 serum concentrations and pharmacokinetic (PK) parameter (AUC)



Information from the National Library of Medicine

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

Inclusion Criteria:

  1. At least 18 years of age.
  2. Weight < 125 Kg.
  3. Subjects must have a primary diagnosis of wAIHA or CAD defined by the presence of hemolytic anemia and positive DAT for wAIHA (IgG) or CAD (C3).
  4. Hemoglobin <11 g/dL.
  5. Signs of hemolysis with abnormal values by any of the hemolytic markers:

    1. Increased absolute reticulocyte count (above ULN)
    2. Reduced haptoglobin (below LLN)
    3. Increased lactase dehydrogenase (LDH) (above ULN)
    4. Increased indirect bilirubin (above ULN)
  6. Women of child-bearing potential (WOCBP) (defined as any female who has experienced menarche and who is NOT permanently sterile or postmenopausal. Postmenopausal is defined as 12 consecutive months with no menses without an alternative medical cause) must have a negative pregnancy test at screening 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.
  7. 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.
  8. Able to provide documentary evidence of the following vaccinations within 2 years prior to screening:

    1. Neisseria meningitides types A, C, W, Y and type B (administered as two separate vaccinations)
    2. Streptococcus pneumoniae (Pneumococcal conjugate vaccine and Pneumococcal polysaccharide vaccine 23 [PCV13 and/or PPSV23, respectively])
    3. Haemophilus influenzae Type B (Hib) vaccine

    Subjects that do not have documentary evidence must be willing to receive any missing vaccinations as outlined below:

    1. Neisseria meningitides types A, C, W, Y and type B must be administered prior to dosing on Day 1. A booster is administered after at least 8 weeks (Day 56; for both vaccinations).
    2. Streptococcus pneumoniae PCV13 must be administered prior to dosing on Day 1 (see Section 12.2 for details). A PPSV23 booster is administered after at least 8 weeks (Day 56)
    3. Haemophilus influenze Type B (Hib) must be administered prior to dosing on Day 1.
  9. Willing and able to give informed consent.
  10. Specific for wAIHA: Relapsed from, did not respond or relapsed, or did not tolerate, at least one prior wAIHA treatment regimen (such as prednisone, rituximab).

Exclusion Criteria:

  1. Prior treatment with rituximab within 90 days.
  2. Deficiency of iron, folic acid and vitamin B12 prior to treatment phase
  3. Abnormal liver function as indicated by a direct bilirubin above normal level, and/or an AST or ALT level > 2x upper limit of normal. Please note elevated indirect bilirubin due to hemolysis is not an exclusion criteria.
  4. Elevated bilirubin not due to active hemolysis. Any elevation of bilirubin >ULN will require Sponsor review and approval for subject enrollment into the trial.
  5. Active aggressive lymphoma requiring therapy or an active non-lymphatic malignant disease other than basal cell carcinoma or carcinoma in situ (CIS) of the cervix.
  6. Presence or suspicion of active bacterial or viral infection, in the opinion of the Investigator, at screening or severe recurrent bacterial infections.
  7. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period.
  8. Pregnant, breast-feeding, or intending to conceive during the course of the study, including the Post-Treatment Phase.
  9. Inability to cooperate or any condition that, in the opinion of the investigator, could increase the subject's risk by participating in the study or confound the outcome of the study.
  10. Myocardial infarction, CABG, coronary or cerebral artery stenting and /or angioplasty, stroke, cardiac surgery, or hospitalization for congestive heart failure within 3 months or > Class 2 Angina Pectoris or NYHA Heart Failure Class >2
  11. QTcF > 470 ms
  12. PR > 280 ms
  13. Mobitz II 2nd degree AV Block, 2:1 AV Block, High Grade AV Block, or Complete Heart Block unless the patient has an implanted pacemaker or implantable cardiac defibrillator (ICD) with backup pacing capabilities

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): NCT03226678


Contacts
Contact: Alice Shen 617-977-5721 clinicaltrials@apellis.com

Locations
United States, California
Apellis Clinical Site Recruiting
Whittier, California, United States, 70603
American Institute of Research Recruiting
Whittier, California, United States, 90603
United States, Florida
Apellis Clinical Site Recruiting
Miami Lakes, Florida, United States, 33014
Mid Florida Hematology Oncology Recruiting
Orange City, Florida, United States, 32763
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
United States, Iowa
University of Iowa Hospitals Recruiting
Iowa City, Iowa, United States, 52242
United States, Maryland
University of Maryland Recruiting
Baltimore, Maryland, United States, 21201
United States, Michigan
Cancer and Hematology centers of Western Michigan Recruiting
Grand Rapids, Michigan, United States, 49503
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
United States, New Jersey
New Jersey Oncology Hematology Associates Recruiting
Brick, New Jersey, United States, 08724
United States, New York
New York Cancer and Blood Specialists Recruiting
Bronx, New York, United States, 10469
Weill Cornell Not yet recruiting
New York, New York, United States, 10065
New York Cancer and Blood Specialists Not yet recruiting
Setauket, New York, United States, 11733
United States, North Carolina
Apellis Clinical Site Recruiting
Greenville, North Carolina, United States, 27834
East Carolina University Recruiting
Greenville, North Carolina, United States, 27834
United States, Tennessee
University of Tennessee Medical Center Recruiting
Knoxville, Tennessee, United States, 37920
University of Tennessee Health Sciences Center Recruiting
Memphis, Tennessee, United States, 38119
Baptist Cancer Center Not yet recruiting
Memphis, Tennessee, United States, 38120
United States, Washington
Northwest Medical Specialties Recruiting
Tacoma, Washington, United States, 98405
Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.

Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03226678     History of Changes
Other Study ID Numbers: APL2-CP-AIHA-208
First Posted: July 24, 2017    Key Record Dates
Last Update Posted: October 9, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Anemia
Hemolysis
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hematologic Diseases
Pathologic Processes
Autoimmune Diseases
Immune System Diseases