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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 : Active, not recruiting
First Posted : April 27, 2017
Last Update Posted : February 20, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE April 24, 2017
First Posted Date  ICMJE April 27, 2017
Last Update Posted Date February 20, 2020
Actual Study Start Date  ICMJE August 31, 2017
Estimated Primary Completion Date July 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 6, 2017)
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
Original Primary Outcome Measures  ICMJE
 (submitted: April 24, 2017)
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
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 24, 2017)
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
Official Title  ICMJE A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Atypical Hemolytic Uremic Syndrome (aHUS)
Intervention  ICMJE Biological: ALXN1210
Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.
Study Arms  ICMJE Experimental: ALXN1210
Intervention: Biological: ALXN1210
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: February 19, 2020)
30
Original Estimated Enrollment  ICMJE
 (submitted: April 24, 2017)
16
Estimated Study Completion Date  ICMJE January 2025
Estimated Primary Completion Date July 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Germany,   Italy,   Japan,   Korea, Republic of,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03131219
Other Study ID Numbers  ICMJE ALXN1210-aHUS-312
2016‐002499‐29 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alexion Pharmaceuticals
Study Sponsor  ICMJE Alexion Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date February 2020

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