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

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ClinicalTrials.gov Identifier: NCT03131219
Recruitment Status : Recruiting
First Posted : April 27, 2017
Last Update Posted : October 18, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

April 24, 2017
April 27, 2017
October 18, 2018
August 31, 2017
December 2018   (Final data collection date for primary outcome measure)
Complete TMA response [ Time Frame: 26 weeks ]
The proportion of patients who achieved complete thrombotic microangiopathy (TMA) response as assessed by normalization of hematological parameters and ≥ 25% improvement in serum creatinine between Baseline and Day 183
Complete TMA response [ Time Frame: 26 weeks ]
The proportion of patients who achieved complete thrombotic microangiopathy (TMA) response as assessed by normalization of hematological parameters between Baseline and Day 183
Complete list of historical versions of study NCT03131219 on ClinicalTrials.gov Archive Site
  • Dialysis requirement status [ Time Frame: 26 weeks ]
    The proportion of patients who do not require dialysis, among those who had received dialysis within 56 days prior to study drug treatment initiation
  • Time to Complete TMA response [ Time Frame: 26 weeks ]
    Time from start of treatment to achievement of complete TMA response, defined as normalization of hematological parameters and ≥ 25% improvement in serum creatinine from Baseline
  • Complete TMA Response status over time [ Time Frame: 26 weeks ]
    Proportion of patients achieving complete TMA response at each time point.
  • Observed value and change from baseline in estimated glomerular filtration rate (eGFR) [ Time Frame: 26 weeks ]
    Observed value and change from baseline in eGFR
  • Change from baseline in chronic kidney disease (CKD) stage [ Time Frame: 26 weeks ]
    The number and proportion of patients with improvement, worsening, and no change in CKD stage compared to baseline.
  • Change from baseline in hematologic parameters (platelets, lactate dehydrogenase (LDH), hemoglobin) [ Time Frame: 26 weeks ]
    Observed value and change from baseline in platelets, LDH, and hemoglobin.
  • Increase in hemoglobin of ≥ 20 g/L from baseline [ Time Frame: 26 weeks ]
    A proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline to Day 183
  • Change from baseline in quality of life, as measured by Pediatric Functional Assessment of Chronic Therapy (FACIT) Fatigue questionnaire (patients ≥ 5 years of age) [ Time Frame: 26 weeks ]
    Change in pediatric FACIT-Fatigue scores from baseline to Day 183
Same as current
Not Provided
Not Provided
 
Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
The purpose of the study is to assess the efficacy of ALXN1210 to control disease activity in children and adolescents with aHUS who have not previously used a complement inhibitor, as well as in complement inhibitor experienced adolescent patients.
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.
Experimental: 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
23
16
December 2020
December 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

Cohort 1:

  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

Cohort 2:

  1. Patients between 12 and <18 years of age who have been treated with eculizumab for aHUS for at least 90 days prior to Screening
  2. Patients with documented diagnosis of aHUS
  3. Patients with clinical evidence of response to eculizumab indicated by stable TMA parameters at Screening
  4. Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae
  5. Females 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. Females who plan to become pregnant during the study or are currently 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. 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 end-stage kidney disease (ESKD)
  10. For Cohort 2 patients, prior use of complement inhibitors other than eculizumab
  11. For Cohort 2 patients, any known abnormal TMA parameters within 90 days prior to Screening
Sexes Eligible for Study: All
up to 17 Years   (Child)
No
Contact: Alexion Pharmaceuticals 475-230-ALXN (2596) clinicaltrials@alexion.com
Belgium,   Germany,   Japan,   Korea, Republic of,   Spain,   United States
 
 
NCT03131219
ALXN1210-aHUS-312
2016‐002499‐29 ( EudraCT Number )
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Alexion Pharmaceuticals
Alexion Pharmaceuticals
Not Provided
Not Provided
Alexion Pharmaceuticals
October 2018

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