Study of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria (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
- EUCTR2018-003956-19-FI
- Lead Sponsor
- Ra Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 40
To be eligible for this study, subjects must meet ALL the following inclusion criteria:
1. Male or female =18 years and <85 years
2. Able to provide informed consent, including signing and dating the informed consent form (ICF).
3. Diagnosis of PNH by flow cytometry.
4. Subjects must not have received treatment with eculizumab or another complement inhibitor within 6 months prior to Screening.
5. Subjects must have a lactate dehydrogenase (LDH) level =2 times the upper limit of normal (xULN) during Screening.
6. Subjects must be transfusion-dependent, defined as receiving at least one qualifying transfusion (for Hb =9g/dL with symptoms of anemia; or for Hb =7g/dL irrespective of symptoms) in the 6 months prior to Screening.
7. Female subject of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test within 24 hours prior to the first dose of study drug.
8. Sexually active female subjects of childbearing potential (i.e., women who are not postmenopausal or who have not had a hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) and all male subjects (who have not been surgically sterilized by vasectomy) must agree to use effective contraception during the study.
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 32
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8
Subjects who meet ANY of the following exclusion criteria will be excluded from the study:
1. Platelet count <50,000/µL or absolute neutrophil count (ANC) <500 cells/µL at Screening.
2. Calculated glomerular filtration rate of <30 mL/min/1.73m2 based on the Modification of Diet in Renal Disease (MDRD) equation at Screening.
3. Elevation of liver function tests: alanine aminotransferase (ALT) >2xULN or Direct Bilirubin and Alkaline Phosphatase both >2xULN.
4. Elevation of amylase or lipase >2xULN.
5. History of meningococcal disease.
6. Current or recent systemic infection within 2 weeks prior to Screening or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Screening.
7. Pregnant, planning to become pregnant, or nursing female subjects.
8. History of bone marrow transplantation, or expected to undergo bone marrow transplantation during the 6-month treatment period.
9. Recent surgery requiring general anesthesia within 2 weeks prior to Screening or expected to have surgery requiring general anesthesia during the 6-month Treatment Period.
10. Active malignancy (except curatively resected squamous or basal cell carcinoma of the skin) requiring surgery, chemotherapy, or radiation within the prior 12 months (subjects with a history of malignancy who have undergone curative resections or otherwise not requiring a treatment for at least 12 months prior to Screening with no detectable recurrence are allowed).
11. History of any significant medical or psychiatric disorder that in the opinion of the investigator would make the subject unsuitable for participation in the study.
12. Treatment with any investigational medicinal product or investigational device within 30 days prior to Screening.
13. Participation in another concurrent clinical trial involving an investigational, therapeutic intervention (participation in observational studies and or registry studies is permitted).
14. Unable or unwilling to comply with the requirements of the study.
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