Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
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| ClinicalTrials.gov Identifier: NCT03131219 |
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Recruitment Status :
Recruiting
First Posted : April 27, 2017
Last Update Posted : April 12, 2018
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Sponsor:
Alexion Pharmaceuticals
Information provided by (Responsible Party):
Alexion Pharmaceuticals
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Atypical Hemolytic Uremic Syndrome (aHUS) | Biological: ALXN1210 | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 16 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 : | December 2018 |
| Estimated Study Completion Date : | December 2020 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Atypical hemolytic-uremic syndrome
| 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. |
Primary Outcome Measures :
- 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 :
- 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
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| Ages Eligible for Study: | up to 18 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Patients from birth up to < 18 years of age and weighing ≥ 5 kg at the time of consent.
- Evidence of thrombotic microangiopathy (TMA), including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
- Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae
- 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:
- ADAMTS13 deficiency (Activity < 5%)
- Shiga toxin-related hemolytic uremic syndrome (STEC-HUS)
- Positive direct Coombs test
- Pregnancy or breastfeeding
- Identified drug exposure-related hemolytic uremic syndrome (HUS)
- Bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT) within last 6 months prior to start of Screening
- HUS related to vitamin B12 deficiency
- Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
- Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage kidney disease (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): NCT03131219
Contacts
| Contact: Alexion Pharmaceuticals | 475-230-ALXN (2596) | 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 | |
| Belgium | |
| Clinical Trial Site | Recruiting |
| Bruxelles, Belgium | |
| Germany | |
| Clinical Trial Site | Recruiting |
| Heidelberg, Germany | |
| Japan | |
| Clinical Trial Site | Recruiting |
| Fuchū, 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 | |
Sponsors and Collaborators
Alexion Pharmaceuticals
| 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: | April 12, 2018 |
| Last Verified: | April 2018 |
| Studies a U.S. FDA-regulated Drug Product: | Yes | |
| Studies a U.S. FDA-regulated Device Product: | No | |
Additional relevant MeSH terms:
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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 |