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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 : March 26, 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 : 12 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 : April 2019
Estimated Study Completion Date : April 2019


Arm Intervention/treatment
Experimental: Cohort 1 wAIHA (n=6) Drug: APL-2
Complement Inhibitor

Experimental: Cohort 2 CAD (n=6) Drug: APL-2
Complement 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. Clinical symptoms 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 Neisseria meningitides 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 -14, OR willing to receive vaccinations against Neisseria meningitides at least two weeks prior to dosing on Day 1 with a booster after Day 56 (for both vaccinations) and Pneumococcal and Hib vaccines 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. Active aggressive lymphoma requiring therapy or an active non-lymphatic malignant disease other than basal cell carcinoma or CIS of the cervix.
  5. Presence or suspicion of active bacterial or viral infection, in the opinion of the Investigator, at screening or severe recurrent bacterial infections.
  6. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period.
  7. Pregnant, breast-feeding, or intending to conceive during the course of the study, including the Post-Treatment Phase.
  8. 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.
  9. 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
  10. QTcF > 470 ms
  11. PR > 280 ms
  12. 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: Federico Grossi 617-977-5701 clinicaltrials@apellis.com

Locations
United States, California
Apellis Clinical Site Recruiting
Whittier, California, United States, 70603
United States, Florida
Apellis Clinical Site Recruiting
Miami Lakes, Florida, United States, 33014
United States, Minnesota
Apellis Clinical Site Recruiting
Rochester, Minnesota, United States, 55905
United States, North Carolina
Apellis Clinical Site Recruiting
Greenville, North Carolina, United States, 27834
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: March 26, 2018
Last Verified: March 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