Recruiting
Phase 3

Iptacopan

Sponsor:

Novartis Pharmaceuticals

Code:

NCT06934967

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility Criteria

Sex: All

Age: 2 - 18

Healthy Volunteers: Not accepted

Interventions

LNP023

Study Details

Brief summary:

The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to < 18 years of age.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study ID

NCT06934967

Start date

Oct 28, 2025

Status verified date

Aug, 2026

Completion date

Nov 13, 2031

Anticipated

Primary completion date

Oct 14, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 18

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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.
  • Patients 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.
  • Patients who are anti-C5 treatment naive: mean hemoglobin level < 10 g/dL confirmed by central laboratory assessment during screening.
  • Patients 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.
  • 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.
  • Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan.

Exclusion Criteria:

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

Other protocol-defined inclusion/exclusion criteria may apply.

Study Design

Enrollment

12 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: LNP023-Cohort 1 (12 < 18 years old)

Participants (12 to < 18 years old) will take iptacopan at the dose of 200 mg twice per day (in the morning and in the evening).

experimental: LNP023 -Cohort 2 (2 to < 12 years old)

Participants (2 to < 12 years old) will be dosed based on weight at the Day 1 visit, initially. The study medication dose will be reassessed and re-adjusted as needed based on their weight at Week 12, 26, and 38.

Interventions

LNP023

Cohort 1-administered orally a dosing scheme of 200 mg twice-daily (two 100 mg capsules). Cohort 2- administered orally a dosing scheme based on weight at the Day 1, Week 12, 26 and 38.

Primary outcome measure

  • Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 26 weeks ]
  • PK parameter (Cmax) [ Time Frame: Week 2 ]
  • PK parameter (AUClast) [ Time Frame: Week 2 ]
  • PK parameter (AUCtau) [ Time Frame: Week 2 ]
  • PK parameter (Ctrough) [ Time Frame: Weeks 2, 4, 12 and 26 ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111

Locations

Childrens Healthcare of Atlanta

Recruiting

Atlanta, Georgia, United States, 30342

Contacts

Robert Uddo

robert.uddo@choa.org

Principal Investigator:

Satheesh Chonat

Cancer Institute of New Jersey

Recruiting

New Brunswick, New Jersey, United States, 08901

Contacts

Serban Morojanu

+1 732 235 7577moroiasa@cinj.rutgers.edu

Principal Investigator:

Richard DRACHTMAN

Childrens Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104-4399

Contacts

Peter Nicholas

+1 267 425 2136nicholasp@email.chop.edu

Principal Investigator:

Timothy Olson

St Jude Childrens Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

Michelle Brignac

michelle.brignac@stjude.org

Principal Investigator:

Marcin Wlodarski

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Aug 7, 2026

Last verified

Aug, 2026

Keywords

  • Iptacopan,
  • PNH,
  • hemoglobin,
  • anemia

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-08-07.