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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-502965-34-00 | Other Identifier | EU CT |
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This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.
The extension study Baseline/Day 1 visit is equivalent to the End of Treatment visit of the parent study. The study will begin on Day 1 followed by on-site visits every 4 months during the study treatment period. A Safety Follow Up tele-visit must be conducted 7 days after last study treatment to collect information on Adverse Events.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Iptacopan 200 mg | Experimental | Open label , single arm |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Iptcaopan 200 mg | Drug | Open label, participant specific kits, hard gelatin capsules to be taken twice a day |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with adverse events and serious adverse events | Number of participants with adverse events and serious adverse events, including abnormal safety laboratory parameters, vital signs and ECGs | Throughout the study duration, up to 4 years |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with absence of TMA manifestation without the use of anti-C5 antibody | Thrombotic microangiopathy (TMA) manifestation is defined by the coexistence of at least two of the following three criteria at the same visit attributable to aHUS:
|
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Novartis Pharmaceuticals | Contact | +41613241111 | novartis.email@novartis.com | |
| Novartis Pharmaceuticals | Contact | +81337978748 |
| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Active, not recruiting | São Paulo | São Paulo | 05403 000 | Brazil | |
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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| ID | Term |
|---|---|
| D065766 | Atypical Hemolytic Uremic Syndrome |
| D057049 | Thrombotic Microangiopathies |
| D015432 | Glomerulonephritis, Membranoproliferative |
| ID | Term |
|---|---|
| D006463 | Hemolytic-Uremic Syndrome |
| D014511 | Uremia |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
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| Throughout the study duration, up to 4 years |
| Number of participants with complete TMA response status without the use of anti-C5 antibody therapy | Complete thrombotic microangiopathy (TMA) Response is defined as (1) hematological normalization in platelet count (platelet count ≥150 x 109/L) and LDH (below ULN), and (2) improvement in kidney function (≥ 25% serum creatinine reduction from baseline or ≥ 25% serum creatinine reduction compared to serum creatinine values prior to initiation of anti-C5 antibody therapy) | Throughout the study duration, up to 4 years |
| Estimated glomerular filtration rate (eGFR) | Estimated glomerular filtration rate (eGFR) based on eGFR categories will be collected. Serum creatinine as measured in mg/dL as part of the clinical chemistry panel through the central laboratory will be used to calculate the eGFR applying the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula. | Throughout study duration, up to 4 years |
| Chronic kidney disease (CKD) stage | Chronic kidney disease (CKD) stage (1-5) based on eGFR categories will be provided:
| Throughout study duration, up to 4 years |
| Number of participants by dialysis requirement status | Dialysis requirement status will be provided | Throughout the study duration, up to 4 years |
| Number of participants with Thrombotic Microangiopathy (TMA) related adverse events | TMA related events during the study is defined as any one of the following:
| Throughout study duration, up to 4 years |
| Novartis Investigative Site |
| Recruiting |
| Rio de Janeiro |
| 22270-060 |
| Brazil |
| Novartis Investigative Site | Recruiting | Beijing | 100034 | China |
| Novartis Investigative Site | Recruiting | Ostrava | Poruba | 708 52 | Czechia |
| Novartis Investigative Site | Active, not recruiting | Nagpur | Maharashtra | 440015 | India |
| Novartis Investigative Site | Recruiting | Pune | Maharashtra | 411011 | India |
| Novartis Investigative Site | Recruiting | Iruma-gun | Saitama | 3500495 | Japan |
| Novartis Investigative Site | Recruiting | Ankara | Yenimahalle | 06500 | Turkey (Türkiye) |
| Novartis Investigative Site | Recruiting | Mersin | Yenisehir | 33110 | Turkey (Türkiye) |
| D052776 |
| Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D005921 | Glomerulonephritis |
| D009393 | Nephritis |
| D007154 | Immune System Diseases |