A Research Study to Gather Scientific Information About the Efficacy and Safety of the Investigational Drug APL-2 In Treating Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated with Anemia, In a Randomly Assigned Comparison with the Current Standard of Care Treatment Approved for PNH
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)MedDRA version: 20.1Level: LLTClassification code 10055629Term: Paroxysmal nocturnal hemoglobinuriaSystem Organ Class: 100000004857Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2018-004220-11-PL
- Lead Sponsor
- Apellis Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 54
At Visit 1 screening, subjects must fulfill all of the following inclusion criteria to be eligible for participation in the study:
1. Be at least 18 years old (inclusive).
2. Have LDH =1.5 x ULN at the screening visit.
3. Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone >10%).
4. Have Hb less than the lower limit of normal (LLN) at the screening visit.
5. Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject’s dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
6. Body mass index (BMI) = 35 kg/m2 at the screening visit.
7. Have a platelet count of >50,000/mm3 at the screening visit.
8. Have an absolute neutrophil count >500/mm3 at the screening visit.
9. 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 and for 90 days after their last dose of study drug.
10. Males must agree to use protocol-defined methods of contraception and agree to refrain from donating sperm for the duration of the study and for 90 days after their last dose of study drug.
11. Have vaccination against Streptococcus pneumoniae, Neisseria meningitides (types A, C, W, Y, and B), and Haemophilus influenzae (type B) either within 2 years prior to Day 1 dosing, or agree to receive vaccination 14 days after starting treatment with APL-2 (along with prophylactic antibiotic therapy for at least the 14 days between APL-2 treatment initiation and vaccination and for 14 days post vaccination). Vaccination is mandatory, unless documented evidence exists that subjects are non-responders to vaccination (as evidenced by titers or display titer levels within acceptable local limits).
12. Be willing and able to give informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 27
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 27
Subjects will be excluded from the study if there is evidence of any of the following criteria at screening:
1. Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
2. Hereditary complement deficiency.
3. History of bone marrow transplantation.
4. Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:
• Erythropoietin or immunosuppressants for at least 8 weeks
• Systemic corticosteroids for at least 4 weeks
• Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
• Iron supplements, vitamin B12, or folic acid for at least 4 weeks
• Low-molecular-weight heparin for at least 4 weeks
5. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
6. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5 half-lives, whichever is longer.
7. Planning to become pregnant or currently a breast-feeding woman.
8. History of meningococcal disease.
9. Any comorbidity or condition (such as malignancy) that, in the opinion of the Investigator, could put the subject at increased risk or potentially confound study data.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method