This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab.

Phase 1

Conditions: Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1Level: LLTClassification code 10055629Term: Paroxysmal nocturnal hemoglobinuriaSystem Organ Class: 100000004857
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2017-002370-39-IT

Lead Sponsor: ALEXION PHARMACEUTICALS INCORPORATED

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 105

Inclusion Criteria

Age
1. Patients must be at least 18 years of age at the time of signing the
informed consent.

Patient and Disease Characteristics
2. Treated with eculizumab according to the labeled dosing
recommendation for PNH (900 mg every 14 days ± 2 days) for at least 6
months prior to study entry with no missed doses within 2 months prior
to study entry and no more than 2 doses outside of the visit window.
3. Lactate dehydrogenase levels = 1.5 × ULN (upper limit of normal),
according to central laboratory, at Screening. Sample must be obtained
within 24 hours of or immediately prior to a scheduled eculizumab dose
administration (ie, at trough eculizumab level).
4. Documented diagnosis of PNH confirmed by high-sensitivity flow
cytometry evaluation (Borowitz, Craig et al. 2010).
5. Vaccinated against meningococcal infections within 3 years prior to,
or at the time of, initiating study drug to reduce the risk of
meningococcal infection (N meningitidis).
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 95
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

Medical Conditions
1. More than 1 LDH value > 2 × ULN within the 6 months prior to study
entry.
2. Major adverse vascular event (MAVE) in the 6 months prior to study
entry.
3. Platelet count < 30,000/mm3 (30 × 109/L) at Screening.
4. Absolute neutrophil count < 500/µL (0.5 × 109/L) at Screening.
5. History of bone marrow transplantation.
6. History of N meningitidis infection.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate PK noninferiority of ravulizumab SC versus ravulizumab IV in adult patients with PNH.;Secondary Objective: - To characterize PK of ravulizumab SC<br>- To characterize PD of ravulizumab SC<br>- To characterize immunogenicity of ravulizumab SC<br>- To evaluate HRQoL and treatment satisfaction on ravulizumab SC<br>- To evaluate safety of ravulizumab SC and ravulizumab OBDS<br>- To evaluate efficacy of ravulizumab SC<br>- To assess performance of ravulizumab OBDS;Primary end point(s): Day 71 serum ravulizumab Ctrough;Timepoint(s) of evaluation of this end point: Day 71

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - PK Endpoint: Ctrough over time<br>- PD Endpoint: Free serum C5 concentrations over time<br>- Immunogenicity<br>- HRQoL and Treatment Satisfaction Endpoints<br>- Safety Endpoints<br>- Efficacy Endpoints;Timepoint(s) of evaluation of this end point: Throughout the study

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