A Phase 2 interventional study of CCX168 in Atypical Hemolytic Uremic Syndrome, sponsored by Mario Negri Institute for Pharmacological Research. Terminated at 1 site in Italy. Open to participants aged 18 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2017-11-14.
Sponsored by Mario Negri Institute for Pharmacological Research · Phase 2, Interventional, and Treatment
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Among 16 studies in Atypical Hemolytic Uremic Syndrome with a site in Italy — 5 open to participants now, 6 under way, 2 of them phase 3. Atypical Hemolytic Uremic Syndrome · Italy.
Among the 16 studies in Atypical Hemolytic Uremic Syndrome with a site in Italy, this one is the 11th largest by enrollment of the 13 interventional ones that give a count, is the only one that started in 2015, is one of 7 in phase 2, and was the 8th registered.
45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.
Browse Atypical Hemolytic Uremic Syndrome studies →5 of the 16 studies in Atypical Hemolytic Uremic Syndrome with a site in Italy are open now. Recruiting first, the newest first:
Alexion Pharmaceuticals, Inc. · Sites in Italy, United States, Australia and 4 more countries
Women only
Novartis Pharmaceuticals · Sites in Italy, China, France and 5 more countries
Ages 18–100
Mario Negri Institute for Pharmacological Research · Sites in Italy
Healthy volunteers
Mario Negri Institute for Pharmacological Research · Sites in Italy
Healthy volunteers
Alexion Pharmaceuticals, Inc. · Sites in Italy, United States, Australia and 14 more countries
A study can be open while its sites in Italy have not opened yet or have stopped. Only those recruiting at a site in Italy →
Of the 12 Atypical Hemolytic Uremic Syndrome studies recruiting now, 10 (83%) set no upper age limit and none (0%) stop at 75 or younger; 6 take children under 18; 2 take healthy volunteers.
This study: 18 Years and older · all sexes · healthy volunteers too.
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16 studies; the count open now beside each. This study's row is marked.
First in 2009; this study’s year is marked. 1 started in 2015.
This study's enrollment of 6 is below the median of 34 across 26 interventional studies indexed under Atypical Hemolytic Uremic Syndrome.
Median enrollment 44 across the 13 interventional studies, 94 across the 3 observational. Of the 9 that completed or were terminated, 7 (78%) have results posted.
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12 of 16 have an industry lead sponsor. The 5 most frequent:
Mario Negri Institute for Pharmacological Research is the lead sponsor of 160 studies on the registry; 37 are open to participants now.
Mario Negri Institute for Pharmacological Research has 4 studies filed under Atypical Hemolytic Uremic Syndrome — fewer than ten, so there is no comparison to draw.
1 of the studies in Atypical Hemolytic Uremic Syndrome with a site in Italy that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05805202Mario Negri Institute for Pharmacological ResearchFunctional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized Therapy | 2026-12-31 |
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Among the 16 studies in Atypical Hemolytic Uremic Syndrome with a site in Italy, from their registry updates:
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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.
Inherited defects that determine uncontrolled activation of the alternative complement pathway have been well documented in atypical Hemolytic Uremic Syndrome (aHUS) patients. Research in recent years has identified more than 120 different mutations, accounting for around 40%-60% of cases, in the genes encoding complement factor H (CFH), membrane cofactor protein (MCP), complement factor I (CFI), C3, complement factor B (CFB), and thrombomodulin (THBD). A therapeutic approach could be the administration of molecules that pharmacologically target complement activation, which is the primary common pathogenic mechanism in all genetic forms of aHUS. Eculizumab has been successfully used as prophylaxis of aHUS recurrences in subjects with plasma dependent or plasma resistant disease or in renal transplant recipients at high risk of recurrence due to CFH, CFI, or C3 complement gene mutations. However the drug must be administered chronically and drug spacing or discontinuance was associated with disease recurrence in the graft. The C5aR receptor antagonist CCX168 could present an appealing alternative to eculizumab for post-transplant prophylaxis of aHUS recurrences since it is orally administrable with lower cost of goods. In addition, CCX168 is theoretically associated with lower risk of infections than eculizumab since the former does not target C5b and leaves the terminal complement pathway intact.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Study 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.
Drug: CCX168
Ex vivo thrombogenesis.
Time frame: Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement component 3 serum levels.
Time frame: Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement component 4 serum levels.
Time frame: Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement component 5 serum levels.
Time frame: Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement Factor H.
Time frame: Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Complement component 5a.
Time frame: Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Soluble thrombomodulin.
Time frame: Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Fibrin split products..
Time frame: Changes from baseline at day 2,14 (during CCX168 treatment), 16 and 21 (after treatment withdrawal).
Ex vivo C5b-9 deposition on microvascular endothelial cells
Time frame: At baseline.
Changes in pre-dialysis and intradialytic blood pressure.
Time frame: The participants will be followed for the duration of the study up to 21 days.
Changes in heart rate.
Time frame: The participants will be followed for the duration of the study up to 21 days.
Safety and tolerability parameters including serious and non serious events
Time frame: The participants will be followed for the duration of the study up to 21 days.
Patient health-related quality of life as measured by administration of EQ-5D-5L questionnaire.
Time frame: Changes from baseline at 14 and 21 day.
Characterization of CCX168 pharmacokinetic profile after oral administration by determining by maximum plasma concentration, time of maximum plasma concentration and area under the plasma concentration-time curve from time 0 to hour 6
Time frame: Changes from Baseline at 4,9,11 and 15 day.
Ran from Jun 2015 to primary completion in Jul 2017, 25 months.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Nov 2017. You cannot join it, but the record below documents what was studied.
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Atypical Hemolytic Uremic Syndrome →
Mario Negri Institute for Pharmacological Research →