Ended Therapeutic confirmatory (Phase III) Atypical hemolytic uremic syndrome

Efficacy and safety of iptacopan (LNP023) in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor therapy

EU CTIS ID: 2023-508840-22-00

What this study is testing

To assess the proportion of participants treated with iptacopan achieving complete thrombotic microangiopathy (TMA) response during 26 weeks of study treatment.

  • Therapeutic confirmatory (Phase III)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • Male and female patients ≥ 18 years of age with evidence of active thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and kidney injury, based on the following laboratory findings: • Platelet count <150x109/L during the Screening Period, and • LDH ≥1.5 x upper limit of normal (ULN) during the Screening Period and hemoglobin ≤ lower limit of normal (LLN) for age and gender during the Screening Period, and • Serum creatinine ≥ULN during the Screening Period with acute worsening of kidney function. (Patients requiring dialysis for acute kidney injury are eligible).
  • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of study treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local regulations, at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study treatment and for at least 2 weeks after vaccination.
  • If not received previously, vaccination against Haemophilus influenzae infection should be given if available and according to local regulations. The vaccine should be given at least 2 weeks prior to first study drug administration.
  • Among patients with a kidney transplant, (a) known history of aHUS prior to current kidney transplantation, or (b) If no history of aHUS prior to the current transplantation is available, patients may be eligible without changes of immunosuppressive regimen if investigator excludes other causes of TMA not attributable to aHUS, especially transplant rejection. If immunosuppressive regimens (e.g., calcineurin inhibitor [CNI] or mammalian target of rapamycin inhibitor [mTORi] need to be modified after transplantation, evidence that TMA has persisted for at least 4 days after modification needs to be available.

You likely can't join if

  • Previous or ongoing treatment with complement inhibitors, including anti-C5 antibody.
  • Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome.
  • Chronic hemo- or peritoneal dialysis.
  • Any adenoviral COVID-19 vaccine within 28 days prior to screening visit.
  • A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) deficiency, and/or Shiga toxin-related hemolytic uremic syndrome (STX-HUS), and/or positive direct Coombs test.
  • Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS.
See the full eligibility criteria
Who can join
  • Male and female patients ≥ 18 years of age with evidence of active thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and kidney injury, based on the following laboratory findings: • Platelet count <150x109/L during the Screening Period, and • LDH ≥1.5 x upper limit of normal (ULN) during the Screening Period and hemoglobin ≤ lower limit of normal (LLN) for age and gender during the Screening Period, and • Serum creatinine ≥ULN during the Screening Period with acute worsening of kidney function. (Patients requiring dialysis for acute kidney injury are eligible).
  • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of study treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local regulations, at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study treatment and for at least 2 weeks after vaccination.
  • If not received previously, vaccination against Haemophilus influenzae infection should be given if available and according to local regulations. The vaccine should be given at least 2 weeks prior to first study drug administration.
  • Among patients with a kidney transplant, (a) known history of aHUS prior to current kidney transplantation, or (b) If no history of aHUS prior to the current transplantation is available, patients may be eligible without changes of immunosuppressive regimen if investigator excludes other causes of TMA not attributable to aHUS, especially transplant rejection. If immunosuppressive regimens (e.g., calcineurin inhibitor [CNI] or mammalian target of rapamycin inhibitor [mTORi] need to be modified after transplantation, evidence that TMA has persisted for at least 4 days after modification needs to be available.
What rules you out
  • Previous or ongoing treatment with complement inhibitors, including anti-C5 antibody.
  • Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome.
  • Chronic hemo- or peritoneal dialysis.
  • Any adenoviral COVID-19 vaccine within 28 days prior to screening visit.
  • A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) deficiency, and/or Shiga toxin-related hemolytic uremic syndrome (STX-HUS), and/or positive direct Coombs test.
  • Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS.
  • Started receiving PE/PI 14 days or longer before the start of screening visit for the current TMA.
  • Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation.
  • In patients with a kidney transplant, acute kidney dysfunction consistent with the diagnosis of transplantation failure due to acute/chronic active T-Cell mediated rejection (TCMR) and/or active/chronic active antibody-mediated rejection (ABMR) according to Banff 2017 criteria.
  • Among patients with native kidney, history or presence of any kidney disease other than aHUS, such as: • Known kidney biopsy finding suggestive of underlying disease other than aHUS • Kidney ultrasound finding demonstrating small kidneys suggestive of chronic kidney failure • Known family history and/or genetic diagnosis of non-complement mediated genetic kidney disease (e.g., focal segmental glomerulosclerosis) • Laboratory tests indicative of a kidney disease other than aHUS (eg, Anti-glomerular Basement Membrane (anti-GBM) antibodies, Anti-Neutrophil Cytoplasmic Antibodies (ANCA), Anti-Phospholipase A2 Receptor (anti-PLA2R) antibodies, anti-double stranded DNA (anti-dsDNA) antibodies) • Liver disease or liver injury at screening • Patients with sepsis or active severe systemic bacterial, viral (including COVID-19) or fungal infection.
  • Presence of systemic infections (bacterial, viral, fungal or parasitic) that, in the opinion of the Investigator, confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease.
  • Active infection, or history of recurrent invasive infections, caused by encapsulated bacteria (i.e., meningococcus, pneumococcus), or H. influenzae.

The study team makes the final eligibility decision.

Where it's taking place

  • Brazil
  • Taiwan
  • United Kingdom
  • United States
  • Korea, Republic of
  • China
  • India
  • Japan

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Brazil; Taiwan; United Kingdom; United States; Korea, Republic of; China and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.