IPTACOPAN for Atypical hemolytic uremic syndrome

Inclusion criteria

• {"criterion_text":"- Signed informed consent must be obtained prior to participation in the open label extension study\n- Willing and able to comply with the study Schedule of Activities (Section 1.3 of the protocol)\n- Participants who have completed the full study treatment period of any prior \"Novartis sponsored\" iptacopan Phase 3 clinical trial in aHUS (e.g. CLNP023F12301, CLNP023F12302) are still on iptacopan study treatment and derive benefit from it as per Investigator's judgement\n- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e. any boosters required should be administered according to local guidelines)"}

Exclusion criteria

• {"criterion_text":"- Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the drugs listed in Section 6.8.2\n- Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the Investigator could put the participant at risk\n- Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae\n- History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes\n- Pregnant or nursing (lactating) women\n- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping of investigational drug."}

Primary endpoints

• {"endpoint_text":"- Safety evaluations including adverse events (AE) /serious adverse events (SAE), safety laboratory parameters, vital signs and electrocardiograms (ECGs) through study duration.","definition_or_measurement_approach":"Safety assessed via reporting and monitoring of adverse events (AE/SAE), safety laboratory parameters, vital signs and ECGs throughout the study duration (as stated)."}

Secondary endpoints

• {"endpoint_text":"- Absence of TMA manifestation without use of anti-C5 antibody throughout the study. TMA manifestation defined by the coexistence of min. two of the three criteria at the same visit attributable to aHUS: ● thrombocytopenia (platelet count decrease of ≥ 25% compared to baseline and < LLN), ● microangiopathic hemolytic anemia (hemoglobin ≤ LLN for age and gender and LDH ≥ 1.5 x ULN), ● worsening kidney function (serum creatinine increase of >25% compared to baseline levels)","definition_or_measurement_approach":"Absence of TMA defined as not meeting at least two of the listed criteria at the same visit; measured by platelet count, hemoglobin, LDH and serum creatinine compared to baseline and laboratory reference limits."}
• {"endpoint_text":"- Complete TMA response status without the use of anti-C5 antibody therapy through study duration. Complete TMA Response is defined as: hematological normalization in platelet count (platelet count ≥150 x 109 /L) and LDH (below ULN), and 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)","definition_or_measurement_approach":"Complete TMA Response measured by platelet count, LDH and serum creatinine change from baseline (thresholds as defined)."}
• {"endpoint_text":"- Observed value and change from baseline in eGFR and CKD stage (1-5) based on eGFR categories through study duration","definition_or_measurement_approach":"Renal function assessed by eGFR and CKD stage; reporting observed values and change from baseline across study visits."}
• {"endpoint_text":"- Dialysis requirement status through study duration","definition_or_measurement_approach":"Assessment of dialysis requirement status (need for renal replacement therapy) throughout the study."}
• {"endpoint_text":"- TMA related events during the study defined as any of the following: ● Irreversible (>3 months) reduction in eGFR rate by ≥20%, not attributable to another cause ● An episode of acute kidney injury (AKI) attributed to a TMA that requires renal replacement therapy ● A non-renal manifestation of a TMA that requires hospitalization or causes irreversible organ damage or death","definition_or_measurement_approach":"TMA-related events recorded per the specified event definitions (irreversible eGFR reduction, AKI requiring RRT, non-renal TMA manifestations requiring hospitalization or causing irreversible damage or death)."}

Methods

• Patient recruitment and retention, provide study related materials i.e posters, flyers (third party: Publicis Healthcare Communications Group Limited) as specified in sponsor third party duties.

Czechia

Earliest CTIS Part Ii Submission Date

24-09-2024

Latest Decision Or Authorization Date

13-02-2026

Processing Time Days

507

Number Of Sites

1

Number Of Participants

6

Primary sponsor

Full Name

Novartis Pharma AG

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Contract research organisations

Name

Parexel International (IRL) Limited

Name

IQVIA Limited

Responsibilities

management of drug supply logistics, dispensing

Name

Q Squared Solutions Limited

Responsibilities

sample and samples kit management, shipment & storage

Third parties

• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Patient recruitment and retention, provide study related materials i.e posters, flyers","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"management of drug supply logistics, dispensing","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"sample and samples kit management, shipment & storage","organisation_type":"Non-Pharmaceutical company"}