Atypical Hemolytic Uremic Syndrome Clinical Trial
Official title:
Multicentric, Prospective Open-label Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical hemolytic syndrome (aHUS) is a severe renal disease affecting children and adults. It is characterized by the occlusion of intrarenal vessels due to the presence of platelet/fibrin thrombi, and leads to end-stage renal disease in up to 2/3 of patients. The discovery of complement alternative pathway as a major risk factor for aHUS has led to the design of a disease-specific treatment, the anti-C5 monoclonal antibody, eculizumab. Complement inhibition using eculizumab has clearly improved the renal outcome of aHUS patients with a dramatic decrease in the risk of end-stage renal disease. However, the optimal duration of eculizumab therapy is still debated. The present study aims to assess the feasibility and safety of the discontinuation of eculizumab treatment in children and adults with aHUS.
A visit (physical examination; blood pressure measurement) will be performed every month for
3 months, and every 3 months for 21 months.
Blood (serum creatinine, platelet count, hemoglobin, LDH, haptoglobin) and urine
(proteinuria/creatininuria ratio and microscopic hematuria) tests will be performed every 2
weeks from inclusion to M6 and subsequently every month starting M7 Urine dipstick (for
albuminuria and microscopic hematuria) will be performed by the patients at home at least
twice a week.
Markers of complement activation and biomarkers of endothelial cells activation and immune
cells activation will be assessed at baseline, M1, M3, M6, M9, M12, M18 and M24.
;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT03605511 -
TTP and aHUS in Complicated Pregnancies
|
||
| Withdrawn |
NCT03303313 -
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
|
Phase 2 | |
| Recruiting |
NCT04861259 -
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
|
Phase 3 | |
| Recruiting |
NCT06065852 -
National Registry of Rare Kidney Diseases
|
||
| Terminated |
NCT02614898 -
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
|
||
| Recruiting |
NCT04958265 -
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
|
Phase 3 | |
| Completed |
NCT00844545 -
Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS
|
Phase 2 | |
| Completed |
NCT00844844 -
Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS
|
Phase 2 | |
| Not yet recruiting |
NCT05795140 -
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
|
Phase 3 | |
| Terminated |
NCT01522170 -
aHUS Observational Long Term Follow-Up
|
N/A | |
| Terminated |
NCT02464891 -
Complement Inhibition in aHUS Dialysis Patients
|
Phase 2 | |
| Withdrawn |
NCT02626663 -
The Role of Microparticles as a Biomarker
|
||
| Recruiting |
NCT01793168 -
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
|
||
| Completed |
NCT00838513 -
Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS)
|
Phase 2 | |
| Completed |
NCT00844428 -
Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS
|
Phase 2 | |
| Withdrawn |
NCT03999840 -
Eculizumab to Cemdisiran Switch in aHUS
|
Phase 2 | |
| Recruiting |
NCT04889430 -
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
|
Phase 3 | |
| Recruiting |
NCT05935215 -
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
|
Phase 3 | |
| Recruiting |
NCT03205995 -
Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome
|
Phase 3 | |
| Recruiting |
NCT05996731 -
Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases
|
N/A |