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Single Arm Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Patients With Atypical Hemolytic Uremic Syndrome (aHUS)

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ClinicalTrials.gov Identifier: NCT02949128
Recruitment Status : Recruiting
First Posted : October 31, 2016
Last Update Posted : June 1, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

October 25, 2016
October 31, 2016
June 1, 2018
November 2016
November 2018   (Final data collection date for primary outcome measure)
Complete TMA response [ Time Frame: 26 weeks ]
Same as current
Complete list of historical versions of study NCT02949128 on ClinicalTrials.gov Archive Site
  • Dialysis requirement status [ Time Frame: 26 weeks ]
  • Time to Complete TMA Response [ Time Frame: 26 weeks ]
  • Complete TMA Response status over time [ Time Frame: 26 weeks ]
  • Observed value and change from baseline in estimated glomerular filtration rate (eGFR) [ Time Frame: 26 weeks ]
  • Change from baseline in chronic kidney disease (CKD) stage [ Time Frame: 26 weeks ]
  • Change from baseline in hematologic parameters (platelets, LDH, hemoglobin) [ Time Frame: 26 weeks ]
  • Increase in hemoglobin ≥ 20g/L from baseline [ Time Frame: 26 weeks ]
  • Change from baseline in quality of life, as measured by the EQ-5D-3L (all patients) [ Time Frame: 26 weeks ]
  • Change from baseline in quality of life, as measured by the FACIT Fatigue Version 4 questionnaire (patients ≥ 18 years of age) [ Time Frame: 26 weeks ]
  • Change from baseline in quality of life, as measured by the Pediatric FACIT Fatigue questionnaire (patients 12 to < 18 years of age) [ Time Frame: 26 weeks ]
Same as current
Not Provided
Not Provided
 
Single Arm Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
Not Provided
The purpose of the study is to assess the efficacy of ALXN1210 to control disease activity in adolescent and adult patients with aHUS who have not previously used a complement inhibitor.
Not Provided
Interventional
Phase 3
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Atypical Hemolytic Uremic Syndrome (aHUS)
Biological: ALXN1210

Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.

  • ≥ 40 to <60 kg: 2400 mg loading, then 3000 mg every 8 weeks
  • ≥ 60 to <100 kg: 2700 mg loading, then 3300 mg every 8 weeks
  • ≥100 kg: 3000 mg loading, then 3600 mg every 8 weeks
Experimental: ALXN1210
ALXN1210
Intervention: Biological: ALXN1210
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
55
Same as current
November 2020
November 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Male or female ≥ 12 years of age and weighing ≥ 40 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, or at the time of, initiating study drug. Patients who receive a meningococcal vaccine less than 2 weeks before initiating ALXN1210 treatment must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Patients who have not been vaccinated prior to initiating ALXN1210 treatment should receive prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination. Patients < 18 years of age must have been vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to national and local vaccination schedule guidelines.
  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

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 known genetic defects of cobalamin C metabolism
  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)
Sexes Eligible for Study: All
12 Years and older   (Child, Adult, Older Adult)
No
Contact: Alexion Pharmaceuticals, Email for a Complete Site List clinicaltrials@alexion.com
Australia,   Austria,   Belgium,   Canada,   France,   Germany,   Italy,   Japan,   Korea, Republic of,   Russian Federation,   Spain,   Taiwan,   United Kingdom,   United States
 
 
NCT02949128
ALXN1210-aHUS-311
2016‐002027‐29 ( EudraCT Number )
Yes
Not Provided
Not Provided
Alexion Pharmaceuticals
Alexion Pharmaceuticals
Not Provided
Not Provided
Alexion Pharmaceuticals
May 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP