Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
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ClinicalTrials.gov Identifier: NCT02949128 |
Recruitment Status :
Active, not recruiting
First Posted : October 31, 2016
Results First Posted : February 10, 2020
Last Update Posted : May 5, 2020
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Sponsor:
Alexion Pharmaceuticals
Information provided by (Responsible Party):
Alexion Pharmaceuticals
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Tracking Information | |||||
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First Submitted Date ICMJE | October 25, 2016 | ||||
First Posted Date ICMJE | October 31, 2016 | ||||
Results First Submitted Date ICMJE | December 11, 2019 | ||||
Results First Posted Date ICMJE | February 10, 2020 | ||||
Last Update Posted Date | May 5, 2020 | ||||
Actual Study Start Date ICMJE | March 18, 2017 | ||||
Actual Primary Completion Date | November 16, 2018 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Proportion Of Participants With Complete Thrombotic Microangiopathy (TMA) Response [ Time Frame: Week 26 ] Complete TMA response during the 26-week Initial Evaluation Period is a composite outcome measure that required normalization of hematological parameters (platelet count and lactate dehydrogenase [LDH]) and improvement in kidney function (≥25% reduction in serum creatinine from baseline); for participants on dialysis, baseline was established at least 6 days after the end of dialysis. Participants had to meet these criteria for 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between. To be considered a responder during the 26-week Initial Evaluation Period, the latest time point a participant could first meet the response criteria was 28 days before the Week 26 (Day 183) assessment. Formal statistical comparison analyses were not planned for this study. The proportion was based on the responders among treated participants. The 95% confidence interval (CI) was based on the asymptotic Gaussian approximation method with a continuity correction.
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Original Primary Outcome Measures ICMJE |
Complete TMA response [ Time Frame: 26 weeks ] | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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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 Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS) | ||||
Official Title ICMJE | Single Arm Study of ALXN1210 in Complement Inhibitor Treatment-naïve Adult and Adolescent Patients With Atypical Hemolytic Uremic Syndrome (aHUS) | ||||
Brief Summary | The purpose of the study is to assess the safety and efficacy of ravulizumab to control disease activity in adolescent and adult participants with aHUS who had not previously used a complement inhibitor. | ||||
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 |
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Condition ICMJE | Atypical Hemolytic Uremic Syndrome (aHUS) | ||||
Intervention ICMJE | Biological: Ravulizumab
Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight: ≥ 40 to < 60 kilograms (kg), 2400 milligrams (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.
Other Names:
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Study Arms ICMJE | Experimental: Ravulizumab
Participants were administered weight-based doses of ravulizumab every 8 weeks for 26 weeks in the Initial Evaluation Period. After the Initial Evaluation Period, participants could enter an Extension Period and receive ravulizumab until the product registration or approval (in accordance with country-specific regulations) or for up to 4.5 years, whichever occurs first. Intervention: Biological: Ravulizumab
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Publications * | Gäckler A, Schönermarck U, Dobronravov V, La Manna G, Denker A, Liu P, Vinogradova M, Yoon SS, Praga M. Efficacy and safety of the long-acting C5 inhibitor ravulizumab in patients with atypical hemolytic uremic syndrome triggered by pregnancy: a subgroup analysis. BMC Nephrol. 2021 Jan 6;22(1):5. doi: 10.1186/s12882-020-02190-0. Erratum in: BMC Nephrol. 2021 Feb 2;22(1):49. | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Actual Enrollment ICMJE |
58 | ||||
Original Estimated Enrollment ICMJE |
55 | ||||
Estimated Study Completion Date ICMJE | July 2023 | ||||
Actual Primary Completion Date | November 16, 2018 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 12 Years and older (Child, Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Australia, Austria, Belgium, Canada, France, Germany, Italy, Japan, Korea, Republic of, Russian Federation, Spain, Taiwan, United Kingdom, United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT02949128 | ||||
Other Study ID Numbers ICMJE | ALXN1210-aHUS-311 2016-002027-29 ( EudraCT Number ) |
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Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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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 |