Adults 6 Months to 18, any sex, with Thrombotic Microangiopathies. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.Primary· 24 weeks
Red blood cell transfusion independence for ≥28 days immediately prior to any scheduled clinical visit up to Week 24 or Urine protein creatinine ratio ≤ 2 mg/mg maintained over ≥ 28 days immediately prior to any scheduled clinical visit up to week 24
Transfusion independence is defined as no RBC transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.
RBC transfusion independent for ≥ 28 days
Group
Value
95% CI
Nomacopan
2
Urine protein creatinine ratio (UPCR) ≤ 2 mg/mg for ≥ 28 days
Group
Value
95% CI
Nomacopan
4
Number of Participants With a Normalised sC5b-9 Value (Where sC5b-9 is the Same Value as the Upper Limit of Normal or Less)Secondary· 24 weeks
Plasma sC5b-9 ≤ upper limit of normal (ULN)
Group
Value
95% CI
Nomacopan
10
Number of Participants With a Normalised Lactate Dehydrogenase (LDH) Value (Where LDH is the Same Value as the Upper Limit of Normal or Less)Secondary· 24 weeks
Lactate dehydrogenase (LDH) ≤ULN
Group
Value
95% CI
Nomacopan
2
Normalisation of Lab ParametersSecondary· 24 weeks
Number of participants with a normalised haptoglobin value (where haptoglobin is within the normal ranges)
Group
Value
95% CI
Nomacopan
4
Number of Participants Not Requiring a Platelet Transfusion (Transfusion Independence) for 28 Days or More.Secondary· 24 weeks
Transfusion independence is defined as no platelet transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.
Group
Value
95% CI
Nomacopan
0
Adverse events — posted to ClinicalTrials.gov
Time frame: Duration of the study (Maximum of 2 year follow-up).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Other recruiting trials for Thrombotic Microangiopathies
Currently open trials in the same condition.
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· recruiting
NCT06291025 — Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune
· NA
· recruiting
NCT05634928 — Construction of a Database for TMA
· recruiting
NCT04745195 — Complement Prospective Evaluation of Thrombotic Microangiopathy on Endothelium
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by AKARI Therapeutics
Last refreshed: 5 June 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04784455.