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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02949128




Registration number
NCT02949128
Ethics application status
Date submitted
25/10/2016
Date registered
31/10/2016
Date last updated
20/02/2024

Titles & IDs
Public title
Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Scientific title
Single Arm Study of ALXN1210 in Complement Inhibitor Treatment-naïve Adult and Adolescent Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
Secondary ID [1] 0 0
2016-002027-29
Secondary ID [2] 0 0
ALXN1210-aHUS-311
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Atypical Hemolytic Uremic Syndrome (aHUS) 0 0
Condition category
Condition code
Blood 0 0 0 0
Other blood disorders
Renal and Urogenital 0 0 0 0
Kidney disease
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Blood 0 0 0 0
Anaemia
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Ravulizumab

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.


Treatment: Other: 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.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage Of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26
Timepoint [1] 0 0
Week 26
Secondary outcome [1] 0 0
Time To Complete TMA Response
Timepoint [1] 0 0
Baseline through Week 114
Secondary outcome [2] 0 0
Participants Who Do Not Require Dialysis at Weeks 26 and 52
Timepoint [2] 0 0
Week 26 and Week 52
Secondary outcome [3] 0 0
Proportion Of Participants With Complete TMA Response At Week 52
Timepoint [3] 0 0
Week 52
Secondary outcome [4] 0 0
Change From Baseline In Estimated Glomerular Filtration Rate (eGFR) At Weeks 26 and 52
Timepoint [4] 0 0
Baseline, Week 26 and Week 52
Secondary outcome [5] 0 0
Participants With Change From Baseline In CKD Stage At Weeks 26 and 52
Timepoint [5] 0 0
Baseline, Week 26, and Week 52
Secondary outcome [6] 0 0
Change From Baseline In Platelet Count At Weeks 26 and 52
Timepoint [6] 0 0
Baseline, Week 26 and Week 52
Secondary outcome [7] 0 0
Change From Baseline In LDH At Weeks 26 and 52
Timepoint [7] 0 0
Baseline, Week 26 and Week 52
Secondary outcome [8] 0 0
Change From Baseline In Hemoglobin At Weeks 26 and 52
Timepoint [8] 0 0
Baseline, Week 26 and Week 52
Secondary outcome [9] 0 0
Percentage Of Complement Inhibitor Treatment-naïve Participants With An Increase From Baseline In Hemoglobin =20 g/L Through Week 26 and Week 52
Timepoint [9] 0 0
Baseline through Week 26 and through Week 52
Secondary outcome [10] 0 0
Change From Baseline In Quality Of Life As Measured By The EuroQol 5-Dimension 3-Level (EQ-5D-3L) At Weeks 26 and 52
Timepoint [10] 0 0
Baseline, Week 26 and Week 52
Secondary outcome [11] 0 0
Change From Baseline In Quality Of Life As Measured By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Version 4 Questionnaire At Weeks 26 and 52
Timepoint [11] 0 0
Baseline, Week 26 and Week 52

Eligibility
Key inclusion criteria
1. Male or female = 12 years of age and weighing = 40 kg at the time of consent.
2. Evidence of thrombotic microangiopathy, 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. Participants who received a meningococcal vaccine less than 2 weeks before initiating ravulizumab treatment must have received treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Participants who had not been vaccinated prior to initiating ravulizumab treatment should have received prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination. Participants < 18 years of age must have been vaccinated against haemophilus influenzae type b and streptococcus pneumoniae according to national and local vaccination schedule guidelines.
4. Female participants of childbearing potential and male participants with female partners of childbearing potential had to use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 deficiency (activity < 5%).
2. Shiga toxin-related hemolytic uremic syndrome.
3. Positive direct Coombs test.
4. Pregnancy or breastfeeding.
5. Identified drug exposure-related hemolytic uremic syndrome (HUS).
6. Bone marrow transplant/hematopoietic stem cell transplant 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, 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).

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
11/01/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
24/01/2023
Sample size
Target
Accrual to date
Final
58
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Clayton
Recruitment hospital [2] 0 0
Clinical Trial Site - Geelong
Recruitment hospital [3] 0 0
Clinical Trial Site - Parkville
Recruitment postcode(s) [1] 0 0
- Clayton
Recruitment postcode(s) [2] 0 0
- Geelong
Recruitment postcode(s) [3] 0 0
- Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Indiana
Country [2] 0 0
United States of America
State/province [2] 0 0
North Carolina
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
Austria
State/province [4] 0 0
Vienna
Country [5] 0 0
Belgium
State/province [5] 0 0
Brussels
Country [6] 0 0
Canada
State/province [6] 0 0
London
Country [7] 0 0
France
State/province [7] 0 0
Bordeaux
Country [8] 0 0
France
State/province [8] 0 0
Clermont-Ferrand
Country [9] 0 0
France
State/province [9] 0 0
Lille
Country [10] 0 0
France
State/province [10] 0 0
Montpellier
Country [11] 0 0
France
State/province [11] 0 0
Nice
Country [12] 0 0
France
State/province [12] 0 0
Paris
Country [13] 0 0
Germany
State/province [13] 0 0
Aachen
Country [14] 0 0
Germany
State/province [14] 0 0
Essen
Country [15] 0 0
Germany
State/province [15] 0 0
Hanover
Country [16] 0 0
Germany
State/province [16] 0 0
Muenchen
Country [17] 0 0
Germany
State/province [17] 0 0
Tuebingen
Country [18] 0 0
Italy
State/province [18] 0 0
Bologna
Country [19] 0 0
Italy
State/province [19] 0 0
Firenze
Country [20] 0 0
Japan
State/province [20] 0 0
Saitama
Country [21] 0 0
Japan
State/province [21] 0 0
Tokyo
Country [22] 0 0
Korea, Republic of
State/province [22] 0 0
Gyeonggi-do
Country [23] 0 0
Korea, Republic of
State/province [23] 0 0
Seoul
Country [24] 0 0
Russian Federation
State/province [24] 0 0
Moscow
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Saint Petersburg
Country [26] 0 0
Spain
State/province [26] 0 0
Barcelona
Country [27] 0 0
Spain
State/province [27] 0 0
Madrid
Country [28] 0 0
Spain
State/province [28] 0 0
Valencia
Country [29] 0 0
Taiwan
State/province [29] 0 0
Taichung
Country [30] 0 0
Taiwan
State/province [30] 0 0
Taipei City
Country [31] 0 0
Taiwan
State/province [31] 0 0
Taipei
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Cardiff
Country [33] 0 0
United Kingdom
State/province [33] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

TypeCitations or Other Details
Journal Barbour T, Scully M, Ariceta G, Cataland S, Garlo ... [More Details]

Results are available at https://clinicaltrials.gov/study/NCT02949128