A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 1

• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH); MedDRA version: 21.1Level: PTClassification code 10034042Term: Paroxysmal nocturnal haemoglobinuriaSystem Organ Class: 10038359 - Renal and urinary disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2017-002820-26-NL
• Lead Sponsor: Alexion Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-01-10
• Last Update Posted: 26 April 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 13

Inclusion Criteria

1. Patients from birth up to < 18 years of age and weighing = 5 kg at the time of consent.
2. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry
3. For patients not currently treated with eculizumab, presence of 1 or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin <10 g/dL), history of a major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction; or history of pRBC transfusion due to PNH.
4. LDH level = 1.5 × ULN for patients not being treated with eculizumab at screening and LDH level = 1.5 × ULN for patients being treated with eculizumab.
5. Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae
Are the trial subjects under 18? yes
Number of subjects for this age range: 13
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Platelet count < 30,000/mm (30x109/L) at screening.
2. Absolute neutrophil count < 500 µL (0.5 x109/L) at screening
3. History of Bone Marrow transplantation
4. History of N. meningitidis infection
5. History of unexplained, recurrent infection

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified