Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)

Phase 1

• Conditions: Atypical Hemolytic Uremic Syndrome (aHUS); MedDRA version: 20.0Level: LLTClassification code 10019515Term: Hemolytic uremic syndromeSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-002499-29-IT
• Lead Sponsor: Alexion Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-01-30
• Last Update Posted: 30 November 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

1. Patients from birth up to < 18 years of age and weighing = 5 kg at the time of consent.
2. Evidence of thrombotic microangiopathy (TMA), including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
3. Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae
4. Female patients of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ALXN1210
Are the trial subjects under 18? yes
Number of subjects for this age range: 16
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. ADAMTS13 deficiency (Activity < 5%)
2. Shiga toxin-related hemolytic uremic syndrome (STEC-HUS)
3. Positive direct Coombs test
4. Pregnancy or breastfeeding
5. Identified drug exposure-related hemolytic uremic syndrome (HUS)
6. Bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT) within last 6 months prior to start of Screening
7. HUS related to vitamin B12 deficiency
8. Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
9. Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for ESKD)

Study & Design

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