Iptacopan hydrochloride for Paroxysmal nocturnal haemoglobinuria

Inclusion criteria

• {"criterion_text":"- Male and female participants 2 to < 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes/monocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg.\n- Participants being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator.\n- Participants who are anti-C5 treatment naive: mean hemoglobin level < 10 g/dL confirmed by central laboratory assessment during screening.\n- Participants who are anti-C5 treatment naive: lactate dehydrogenase (LDH) > 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab.\n- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated.\n- Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan."}

Exclusion criteria

• {"criterion_text":"- History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes.\n- Known or suspected hereditary complement deficiency at screening.\n- History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1).\n- Participants with laboratory evidence of bone marrow failure (reticulocytes < 100 × 10^9/L, platelets < 30 × 10^9/L, neutrophils < 0.5 × 10^9/L).\n- Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration.\n- Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration."}

Primary endpoints

• {"endpoint_text":"- Safety evaluations, including AEs, SAEs, laboratory parameters, vital signs, and cardiovascular parameters.","definition_or_measurement_approach":"Safety evaluated by reporting of adverse events (AEs), serious adverse events (SAEs), laboratory parameters, vital signs and cardiovascular parameters as listed in the endpoint description."}
• {"endpoint_text":"- PK parameters Cmax, AUClast, AUCtau (and other PK parameters in plasma, as appropriate), and Ctrough concentrations.","definition_or_measurement_approach":"Pharmacokinetic measurements including Cmax, AUClast, AUCtau and other plasma PK parameters as appropriate, and Ctrough concentrations measured in plasma."}

Secondary endpoints

• {"endpoint_text":"- Change in hemoglobin (Hb) from baseline ≥ 1 g/dL at Week 26 (in the absence of red blood cell (RBC) transfusions from Day 14). Change in Hb from baseline ≥ 1 g/dL at Week 52 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 26 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 52 (in the absence of RBC transfusions from Day 14).","definition_or_measurement_approach":"Responder analyses based on change from baseline in hemoglobin at Weeks 26 and 52 with requirement of absence of RBC transfusions from Day 14 to the respective timepoint."}
• {"endpoint_text":"- Normal Hb at Weeks 26 in the absence of RBC transfusions from Day 14. Normal Hb at Week 52 in the absence of RBC transfusions from Day 14.","definition_or_measurement_approach":"Assessment of hemoglobin reaching normal range at Weeks 26 and 52, with absence of RBC transfusions from Day 14."}
• {"endpoint_text":"- Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 26. Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 52.","definition_or_measurement_approach":"Proportion of participants without packed RBC transfusions and who do not meet transfusion criteria from Day 14 through Weeks 26 and 52."}
• {"endpoint_text":"- Change from baseline in Hb and LDH at Week 26. Change from baseline in Hb and LDH at Week 52.","definition_or_measurement_approach":"Mean change from baseline in hemoglobin and lactate dehydrogenase (LDH) at Weeks 26 and 52; analyses include naive and prior anti-C5 treated patients separately."}
• {"endpoint_text":"- Safety evaluations (including AEs, SAEs, lab parameters, vital signs, and cardiovascular parameters).","definition_or_measurement_approach":"Safety monitoring via AEs, SAEs, laboratory parameters, vital signs and cardiovascular assessments over the 52-week treatment period."}

Methods

• Site-based recruitment through participating paediatric hematology clinics at listed hospital sites (country-specific site lists and recruitment arrangements exist: France, Germany, Italy, Spain, Netherlands).
• Country-specific recruitment materials and advertisements (K2 advertisements documents present for France, Germany, Italy, Spain, Netherlands).
• Pre-screening tool and digital recruitment support provided by Reify Health Inc. (duty: pre-screening tool).
• Third-party recruitment platforms and support (Jumo Health USA Inc. listed with duty: Recruitment and retention materials).
• Use of eCOA and electronic patient data capture (Kayentis listed with duty: eCOA).
• Home nursing / off-site healthcare professional services to support participation (Iqvia Rds Inc. duty: home nursing/off-site healthcare professionals).
• Patient travel and reimbursement support (Greenphire LLC duty: Patient travel and reimbursement).

France

Earliest CTIS Part Ii Submission Date

09-05-2025

Latest Decision Or Authorization Date

23-02-2026

Processing Time Days

290

Number Of Sites

1

Number Of Participants

1

Italy

Earliest CTIS Part Ii Submission Date

12-03-2025

Latest Decision Or Authorization Date

25-02-2026

Processing Time Days

350

Number Of Sites

1

Number Of Participants

1

Spain

Earliest CTIS Part Ii Submission Date

15-05-2025

Latest Decision Or Authorization Date

26-02-2026

Processing Time Days

287

Number Of Sites

1

Number Of Participants

1

Germany

Earliest CTIS Part Ii Submission Date

08-05-2025

Latest Decision Or Authorization Date

02-03-2026

Processing Time Days

301

Number Of Sites

3

Number Of Participants

1

Netherlands

Earliest CTIS Part Ii Submission Date

06-06-2025

Latest Decision Or Authorization Date

17-03-2026

Processing Time Days

285

Number Of Sites

1

Number Of Participants

1

Primary sponsor

Full Name

Novartis Pharma AG

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Contract research organisations

Name

IQVIA Limited / Iqvia Rds Inc.

Responsibilities

Various clinical operations support; home nursing/off-site healthcare professionals; other IW duties (sponsorDuties entries include code values and 'home nursing/off-site healthcare professionals')

Name

Parexel International (IRL) Limited

Responsibilities

Clinical trial operational support (sponsorDuties code present)

Name

ICON Clinical Research Limited

Responsibilities

Clinical trial operational support (sponsorDuties code present)

Name

Syneos Health Inc.

Responsibilities

Clinical trial operational support (sponsorDuties code present)

Name

Labcorp Central Laboratory Services LP

Responsibilities

Central laboratory services

Name

Q Squared Solutions LLC

Responsibilities

Laboratory/testing services

Third parties

• {"country":"United States","full_name":"Reify Health Inc.","duties_or_roles":"pre-screening tool","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"France","full_name":"Kayentis","duties_or_roles":"eCOA","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Q Squared Solutions LLC","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Iqvia Rds Inc.","duties_or_roles":"home nursing/off-site healthcare professionals","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Recruitment and retention materials","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"blood pressure equipments","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient travel and reimbursement","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Transperfect Translations International Inc.","duties_or_roles":"translation","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"central echocardiogram reading","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"SGS France","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Medical Equipment Supplies And Management Limited","duties_or_roles":"ancillary supplies","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}