A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With PNH

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03593200
• Date of registration: 28/06/2018
• Prospective Registration: Yes
• Primary sponsor: Apellis Pharmaceuticals, Inc.
• Public title: A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With PNH
• Scientific title: Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).
• Date of first enrolment: August 16, 2018
• Target sample size: 4
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03593200
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Bulgaria, Greece, Poland, Romania, Serbia

Contacts

• Name: Federico Grossi, MD, PhD
• Affiliation: Study Director

Key inclusion & exclusion criteria

Inclusion Criteria:

- At least 18 years old (inclusive)

- Diagnosed with PNH (white blood cell (WBC) clone >10%)

- Lactose dehydrogenase (LD) =2 times the upper limit of normal

- Screening Ferritin = normal and Total Iron Binding Capacity (TIBC) < LLN based on
central lab reference ranges. If a subject is receiving iron supplements at screening,
the investigator must ensure that his/her dose has been stable for 8 weeks prior to
enrolment and must be maintained throughout the study

- Last transfusion within 12 months prior to screening

- Platelet count of >30,000/mm3 at the screening visit

- Absolute neutrophil count >500/ mm3 at the screening visit

- 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

- Males must agree to use protocol defined methods of contraception and agree to refrain
from donating sperm for the duration of the study

- Vaccination against Neisseria meningitides types A, C, W, Y and B, Streptococcus
pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day
1 dosing, or within 14 days after starting treatment with APL-2. Unless documented
evidence exists that subjects are non-responders to vaccination as evidenced by titers
or display titer levels within acceptable local limits

- Willing and able to give informed consent

Exclusion Criteria:

- Prior eculizumab (Soliris®) treatment

- Active bacterial infection

- Hereditary complement deficiency

- History of bone marrow transplantation

- Concurrent severe aplastic anemia (SAA), defined as currently receiving
immunosuppressive therapy for SAA including but not limited to cyclosporin A,
tacrolimus, mycophenolate mofetil or anti-thymocyte globulin

- Participation in any other investigational drug trial or exposure to another
investigational agent, device or procedure within 30 days

- Evidence of QTcF prolongation defined as >450 ms for males and >470 ms for females at
screeningBreast-feeding women

- History of meningococcal disease



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

PNH

Intervention(s)

Drug: APL-2

Primary Outcome(s)

1.Number of treatment emergent adverse events (TEAEs) following administration of multiple doses of SC APL-2. Severity of treatment emergent adverse events (TEAEs) following administration of multiple doses of SC APL-2. [Time Frame: Change from baseline up to week 36]

3. Haptoglobin (mg/dL) & Hemoglobin (g/dl) [Time Frame: Change from baseline up to week 32]

2. LDH (U/L) [Time Frame: Change from baseline up to week 32]

Secondary ID(s)

APL2-202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors