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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 : Active, not recruiting
First Posted : October 31, 2016
Last Update Posted : July 27, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
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.

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 56 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Actual Study Start Date : November 2016
Estimated Primary Completion Date : November 2018
Estimated Study Completion Date : November 2020


Arm Intervention/treatment
Experimental: ALXN1210
ALXN1210
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




Primary Outcome Measures :
  1. Complete TMA response [ Time Frame: 26 weeks ]

Secondary Outcome Measures :
  1. Dialysis requirement status [ Time Frame: 26 weeks ]
  2. Time to Complete TMA Response [ Time Frame: 26 weeks ]
  3. Complete TMA Response status over time [ Time Frame: 26 weeks ]
  4. Observed value and change from baseline in estimated glomerular filtration rate (eGFR) [ Time Frame: 26 weeks ]
  5. Change from baseline in chronic kidney disease (CKD) stage [ Time Frame: 26 weeks ]
  6. Change from baseline in hematologic parameters (platelets, LDH, hemoglobin) [ Time Frame: 26 weeks ]
  7. Increase in hemoglobin ≥ 20g/L from baseline [ Time Frame: 26 weeks ]
  8. Change from baseline in quality of life, as measured by the EQ-5D-3L (all patients) [ Time Frame: 26 weeks ]
  9. 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 ]
  10. 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 ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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)

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): NCT02949128


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Sponsors and Collaborators
Alexion Pharmaceuticals

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02949128     History of Changes
Other Study ID Numbers: ALXN1210-aHUS-311
2016‐002027‐29 ( EudraCT Number )
First Posted: October 31, 2016    Key Record Dates
Last Update Posted: July 27, 2018
Last Verified: July 2018

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