Study Stopped
The study had accomplished its goal with the 6 patients who have been enrolled.
Complement Inhibition in aHUS Dialysis Patients
ACCESS
An Open-label Phase 2 Study to Assess the Effect of C5aR Antagonist Therapy by CCX168 Oral Administration on ex Vivo Thrombus Formation and Disease Activity in ESRD Patients With Atypical Hemolytic Uremic Syndrome
2 other identifiers
interventional
6
1 country
1
Brief Summary
This study evaluates the effect of CCX168, a C5aR Antagonist, Oral Administration on Ex Vivo Thrombus Formation and Disease Activity in ten patients with diagnosis of Atypical Hemolytic Uremic Syndrome with or without genetic abnormalities in the complement system or thrombomodulin, on stable chronic extracorporeal or peritoneal dialysis therapy since at least 6 months.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2015
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 26, 2015
CompletedStudy Start
First participant enrolled
June 4, 2015
CompletedFirst Posted
Study publicly available on registry
June 8, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 13, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
July 13, 2017
CompletedNovember 14, 2017
November 1, 2017
2.1 years
May 26, 2015
November 13, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Ex vivo thrombogenesis.
Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Secondary Outcomes (13)
Complement component 3 serum levels.
Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement component 4 serum levels.
Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement component 5 serum levels.
Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement Factor H.
Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement component 5a.
Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
- +8 more secondary outcomes
Study Arms (1)
CCX168
EXPERIMENTALStudy medication will be administered as hard gelatin capsules containing 10 mg CCX168. Patients will take 30 mg CCX168, given as 3 x 10 mg capsule, twice daily for 15 days.
Interventions
Eligibility Criteria
You may qualify if:
- Age \>18 years;
- Diagnosis of aHUS with or without identified genetic abnormalities in the complement system or thrombomodulin;
- Stable chronic extracorporeal or peritoneal dialysis therapy since at least 6 months;
- Written informed consent.
You may not qualify if:
- Women of childbearing potential or women who are breastfeeding;
- Shiga toxin-associated HUS or secondary forms of thrombotic microangiopathy;
- ADAMTS13 activity \<10 % or circulating anti ADAMTS13 autoantibodies consistent with the diagnosis of thrombotic thrombocytopenic purpura;
- Need for specific intervention with plasma therapy and/or complement inhibitors as deemed clinically appropriate;
- Plasma therapy or treatment with complement inhibitors or antiplatelet and antithrombotic agents over the last two weeks;
- Liver function impairment (serum liver enzymes or bilirubin levels \>3 x upper limit of normal);
- Neutrophil count \< 2000/μL or lymphocyte count \< 1000/μL;
- Infection requiring antibiotic treatment within the previous 4 weeks prior to screening;
- Participated in any clinical study of an investigational product within 30 days prior to screening or within 5 half-lives after taking the last dose;
- History or presence of any medical condition or disease which, in the opinion of the Investigator may place the subject at unacceptable risk for study participation;
- Inability to understand the potential risks and benefits of the study;
- Legal incapacity.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Mario Negri Institute for Pharmacological Researchlead
- Amgencollaborator
Study Sites (1)
A.O. Papa Giovanni XXIII - U.O. Nefrologia e Dialisi/IRCCS IRFMN - Centro di Ricerche Cliniche per le Malattie Rare Aldo e Cele Daccò
Bergamo, Italy
Related Publications (1)
Aiello S, Gastoldi S, Galbusera M, Ruggenenti P, Portalupi V, Rota S, Rubis N, Liguori L, Conti S, Tironi M, Gamba S, Santarsiero D, Benigni A, Remuzzi G, Noris M. C5a and C5aR1 are key drivers of microvascular platelet aggregation in clinical entities spanning from aHUS to COVID-19. Blood Adv. 2022 Jan 8;6(3):866-881. doi: 10.1182/bloodadvances.2021005246.
PMID: 34852172DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Giuseppe Remuzzi, MD
IRCCS - Mario Negri Institute for Pharmacological Research/A.O. Papa Giovanni XXIII- BG
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 26, 2015
First Posted
June 8, 2015
Study Start
June 4, 2015
Primary Completion
July 13, 2017
Study Completion
July 13, 2017
Last Updated
November 14, 2017
Record last verified: 2017-11