Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With wAIHA or CAD
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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 :
Estimated Study Completion Date :
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Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
At least 18 years of age.
Weight < 125 Kg.
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).
Hemoglobin <11 g/dL.
Clinical symptoms of hemolysis with abnormal values by any of the hemolytic markers:
Increased absolute reticulocyte count (above ULN)
Reduced haptoglobin (below LLN)
Increased lactase dehydrogenase (LDH) (above ULN)
Increased indirect bilirubin (above ULN)
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.
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.
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.
Willing and able to give informed consent.
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).
Prior treatment with rituximab within 90 days.
Deficiency of iron, folic acid and vitamin B12 prior to treatment phase
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.
Active aggressive lymphoma requiring therapy or an active non-lymphatic malignant disease other than basal cell carcinoma or CIS of the cervix.
Presence or suspicion of active bacterial or viral infection, in the opinion of the Investigator, at screening or severe recurrent bacterial infections.
Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period.
Pregnant, breast-feeding, or intending to conceive during the course of the study, including the Post-Treatment Phase.
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.
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
QTcF > 470 ms
PR > 280 ms
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