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This multicenter, observational cohort study uses retrospective collection of past medical history and prospective follow-up to capture longitudinal data on the management and clinical outcomes of patients with atypical hemolytic uremic syndrome (aHUS) treated with ravulizumab as part of routine clinical practice under Poland's National Drug Program (NDP).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Prospective cohort | Group of patients naive to complement inhibitors |
| |
| Retrospective cohort | Group of patients who transitioned from other complement inhibitors to ravulizumab. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Drug | Ultomiris |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve) | In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS | Up to 24 months |
| Proportion of patients attaining/maintaining. Complete TMA Response during observation (switched) | In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS | Up to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Complete TMA Response | Time from initiation of ravulizumab to TMA Response: continuous variable (days; specify when complete response criteria are met) | Up to 24 months |
| Proportion of dialysis-free patients |
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Inclusion Criteria:
Exclusion Criteria:
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Consecutive patients naïve to complement inhibitors (prospective cohort) with a body weight of 10 kg or above (no age restrictions) diagnosed with atypical haemolytic uremic syndrome and meeting all ravulizumab treatment eligibility criteria listed in NDP - no previous exposition to CIs including clinical trials, EAP etc.
Patients who have been switched to ravulizumab from other CI (retrospective cohort) with a body weight of 10 kg or above (no age restrictions) diagnosed with atypical haemolytic uremic syndrome and meeting all ravulizumab treatment eligibility criteria listed in NDP.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| AstraZeneca Clinical Study Information Center Study Information Center | Contact | +118772409479 | information.center@astrazeneca.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Not yet recruiting | Gdansk | Poland | |||
| Research Site |
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
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AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
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| ID | Term |
|---|---|
| D065766 | Atypical Hemolytic Uremic Syndrome |
| ID | Term |
|---|---|
| D006463 | Hemolytic-Uremic Syndrome |
| D014511 | Uremia |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
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| ID | Term |
|---|---|
| C000629409 | ravulizumab |
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In order to achieve the secondary objectives, the following variables will be estimated
| Up to 24 months |
| Complete TMA response | In order to achieve the secondary objectives, the following variables will be estimated:
| Up to 24 months |
| Proportion of patients with lab results normalization during observation | Laboratory Parameters:
| Up to 24 months |
| Change from baseline in CKD stage, as evaluated by the physician over time | In order to achieve the secondary objectives, the following variables will be estimated: CKD stage 1 (rather theoretical at baseline, possible following successful treatment): eGFR ≥90 ml/min./1.73m2 CKD stage 2 (rather theoretical at baseline, possible following successful treatment): eGFR 60 - 90 ml/min./1.73m2 CKD stage 3a: eGFR 45 - 59 ml/min./1.73m2 CKD stage 3b: eGFR 30 - 44 ml/min./1.73m2 CKD stage 4: eGFR 15 - 29 ml/min./1.73m2 CKD stage 5: eGFR< 15 ml/min./1.73m2 CKD stage 5D: need for dialysis independent from eGFR value | Up to 24 months |
| Change from baseline in proteinuria status over time | In order to achieve the secondary objectives, the following variables will be estimated: At least one of the following numbers describing the highest proteinuria and/or albuminuria:
Urine protein reduction to ≤ 500 mg/g (500 mg/24 hours) Urine protein reduction by ≥50% from baseline Time to urine protein reduction to ≤ 500 mg/g (500 mg/24 hours) Time to urine protein reduction by ≥50% from baseline | Up to 24 months |
| Change from baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue; adults) and Pediatric Functional Assessment of Chronic Illness Therapy - Fatigue (Peds FACIT-F; pediatric patients) score overtime (naïve) | FACIT-Fatigue - scoring ranges from 0 (highest fatigue) to 52 (lowest fatigue), where higher scores indicate lower levels of fatigue. Peds FACIT-F - scoring ranges from 0 (highest fatigue) to 52 (lowest fatigue), where higher scores indicate lower levels of fatigue. | Up to 24 months |
| Change form baseline in EQ- 5D-5L (adults) and EQ-5D-Y- 5L (paediatric patients) score overtime (naïve) | EQ-5D-5L/EQ-5D-Y-5L consists of 2 pages: EQ-5D descriptive system and EQ visual analogue scale (EQ VAS). EQ-5D/EQ-5D-Y comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. EQ VAS - scale rating from 0 (worst imaginable health state) to 100 (best imaginable health state) | Up to 24 months |
| Not yet recruiting |
| Katowice |
| Poland |
| Research Site | Recruiting | Katowice | Poland |
| Research Site | Not yet recruiting | Krakow | Poland |
| Research Site | Not yet recruiting | Lodz | Poland |
| Research Site | Not yet recruiting | Poznan | Poland |
| Research Site | Recruiting | Poznan | Poland |
| Research Site | Not yet recruiting | Warsaw | Poland |
| Research Site | Recruiting | Warsaw | Poland |
| Research Site | Not yet recruiting | Wroclaw | Poland |
| 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 |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |