Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome

Sponsor
University Hospital Goettingen
Study ID
NCT02378805
Status
Recruiting
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Conditions

  • Alport Syndrome
  • Familial Benign Hematuria
  • Hereditary Kidney Disease
  • Pediatric Kidney Disease
  • Thin Basement Membrane Disease

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Accepted

Interventions

  • ACE-inhibitor — DRUG
    observational study
  • Angiotensin-receptor blocker (ARB) — DRUG
    observational study
  • HMG-Coenzyme inhibitor (statin) — DRUG
    observational study
  • Spironolactone or Finerenone — DRUG
    observational study
  • Paricalcitol — DRUG
    observational study!
  • SGLT2 inhibitor — DRUG
    observational study

Study Details

The hereditary type IV collagen disease Alport syndrome leads to kidney failure early in life. Currently there are no specific medications approved for treatment, however, several therapies have been evaluated preclinically and could improve outcome. For that reason, this non-interventional, observational study investigates, if medications (1) delay disease progression; (2) delay time to kidney failure; (3) improve life-expectancy compared to untreated patients (relatives). This observational study started in 2006 as an European registry. Since 2019, this registry has been expanded to "Alport XXL" via the International Alport Alliance as a global effort across all continents. From 2020 on to present, "Alport XXL" has a special focus on the outcomes of early therapy in young patients on ACE-inhibitors vs. Angiotensin-receptor blockers vs. their combination.

Key Dates

Start date
Jul 31, 1995
Status verified
Mar 2025
Primary completion
Mar 1, 2036
Completion
Mar 1, 2036

Study Design

Enrollment
800 participants (estimated)

Arms

  • Arm: no-T: untreated patients
    untreated patients, typically uncles or grandfathers of present patients. No Intervention (means no therapy until CKD stage V, on renal replacement therapy)
  • Arm: T-III: late therapy in patients
    patients treated with medications with low eGFR (below 60 ml/min) (starts at patients with CKD stages III and IV).
  • Arm: T-II: early therapy in patients
    therapy starts in patients with albuminuria \>300mg/gCreatinine and eGFR higher than 60 ml/min.
  • Arm: T-I: very early therapy in patients
    therapy starts in patients with microhematuria only (usually at birth) or microalbuminuria (30-300 mg albumin per gCreatinine).
  • Arm: no therapy in heterozygous patients
    heterozygous patients without therapy
  • Arm: therapy in heterozygous patients
    heterozygous patients with therapy (which also can be divided into subgroups stage T-0, I, II, III)

Primary Outcome Measure

age at onset of end stage kidney failure (need for renal replacement therapy) [ Time Frame: until end of observation in 2037 ]

Central Contacts

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