Phase IIa study of APL-2 in patients with 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
- EUCTR2017-005140-16-BG
- Lead Sponsor
- Apellis Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 20
1. At least 18 years old (inclusive)
2. Diagnosed with PNH (WBC clone >10%)
3. Lactose dehydrogenase =2 times the upper limit of normal
4. 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 (see Protocol Section 8.4.4)
5. Last transfusion within 12 months prior to screening
6. Platelet count of >30,000/mm3 at the screening visit
7. Absolute neutrophil count >500/ mm3 at the screening visit
8. 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
9. Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study
10. 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
11. 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 16
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4
1. Prior eculizumab (Soliris®) treatment
2. Active bacterial infection
3. Hereditary complement deficiency
4. History of bone marrow transplantation
5. Concurrent SAA, defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or antithymocyte globulin
6. Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
7. Evidence of QTcF prolongation defined as >450 ms for males and >470 ms for females at screening
8. Breast-feeding women
9. History of meningococcal disease
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