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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 : December 14, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Description
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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.

Condition or disease Intervention/treatment Phase
Atypical Hemolytic Uremic Syndrome (aHUS) Biological: ALXN1210 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 23 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
Actual Study Start Date : August 31, 2017
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : September 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: ALXN1210 Biological: ALXN1210
Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.


Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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

  2. 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

  3. Complete TMA Response status over time [ Time Frame: 26 weeks ]
    Proportion of patients achieving complete TMA response at each time point.

  4. Observed value and change from baseline in estimated glomerular filtration rate (eGFR) [ Time Frame: 26 weeks ]
    Observed value and change from baseline in eGFR

  5. 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.

  6. 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.

  7. 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

  8. 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


Eligibility Criteria
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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03131219


Contacts
Contact: Alexion Pharmaceuticals 1-855-585-8266 clinicaltrials@alexion.com

Locations
United States, Colorado
Clinical Trial Site Recruiting
Aurora, Colorado, United States, 80045
United States, Florida
Clinical Trial Site Recruiting
Hollywood, Florida, United States, 33021
United States, Georgia
Clinical Trial Site Recruiting
Atlanta, Georgia, United States, 30322
United States, Michigan
Clinical Trial Site Recruiting
Detroit, Michigan, United States, 48201
United States, Nebraska
Clinical Trial Site Recruiting
Omaha, Nebraska, United States, 68114-4113
United States, North Carolina
Clinical Trial Site Recruiting
Charlotte, North Carolina, United States, 28203
Belgium
Clinical Trial Site Recruiting
Bruxelles, Belgium
Germany
Clinical Trial Site Recruiting
Heidelberg, Germany
Japan
Clinical Trial Site Recruiting
Fuchū, Japan
Clinical Trial Site Recruiting
Ōbu, Japan
Korea, Republic of
Clinical Trial Site Recruiting
Jeju, Korea, Republic of
Clinical Trial Site Recruiting
Seoul, Korea, Republic of
Clinical Trial Site Recruiting
Yangsan, Korea, Republic of
Spain
Clinical Trial Site Recruiting
Barcelona, Spain
Clinical Trial Site Recruiting
Esplugues De Llobregat, Spain
Clinical Trial Site Recruiting
Valencia, Spain
Sponsors and Collaborators
Alexion Pharmaceuticals
More Information
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03131219     History of Changes
Other Study ID Numbers: ALXN1210-aHUS-312
2016‐002499‐29 ( EudraCT Number )
First Posted: April 27, 2017    Key Record Dates
Last Update Posted: December 14, 2018
Last Verified: December 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Syndrome
Hemolysis
Azotemia
Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
Disease
Pathologic Processes
Uremia
 Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders