Phase 2
N=24
Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)
Warm Autoimmune Hemolytic Anemia · Cold Agglutinin Disease
Bottom Line
View on ClinicalTrials.gov: NCT03226678 ↗Enrolled (actual)
24
Serious AEs
47.4%
Results posted
Dec 2024
Primary outcome: Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity — 5; 5; 7; 6 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- APL-2 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Apellis Pharmaceuticals, Inc.
- Primary completion
- Sep 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity |
5; 5; 7; 6; 5; 9 | — |
| SECONDARY Mean Change From Baseline in Hemoglobin at Weeks 48 and 132 |
0.38; 1.93; 1.62; 2.63; 0.18; -0.20 | — |
| SECONDARY Number of Subjects Who Received Red Blood Cell (RBC) Transfusions |
2; 2; 2; 1; 0; 3 | — |
| SECONDARY Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Weeks 48 and 132 |
-145.050; -130.187; -73.515; -84.064; 11.485; 81.585 | — |
| SECONDARY Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Weeks 48 and 132 |
-51.8; -169.7; -475.8; -77.8; 1.78; 35.18 | — |
| SECONDARY Mean Change From Baseline in Haptoglobin at Weeks 48 and 132 |
0.715; 0.747; 0.528; 0.172; -0.270; -0.335 | — |
| SECONDARY Mean Change From Baseline in Indirect Bilirubin at Weeks 48 and 132 |
-10.723; -9.697; -15.879; -17.981; 0.195; 4.095 | — |
| SECONDARY Maximum Observed Trough Serum Concentration (Ctrough,Max) of Pegcetacoplan |
629.6; 679.7; 661.6; 807.8; 813.6; 795.6 | — |
| SECONDARY Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Weeks 48 and 132 |
-3.8; 10.7; 0.5; 9.0; -1.0; 2.8 | — |
| SECONDARY Mean Change From Baseline in Linear Analog Scale Assessment (LASA) Score at Weeks 48 and 132 |
0.0; 1.3; 0.8; 1.0; 0.0; 0.5 | — |
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).
Eligibility Criteria
Inclusion Criteria
- At least 18 years of age.
- Weight 2x upper limit of normal. Please note elevated indirect bilirubin due to hemolysis is not an exclusion criteria.
- Elevated bilirubin not due to active hemolysis. Any elevation of bilirubin >ULN will require Sponsor review and approval for subject enrollment into the trial.
- 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.
- 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
Data sourced from ClinicalTrials.gov (NCT03226678). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.