Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients

NCT06934967 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: CLNP023I122012024-515926-10
• Study Type: interventional
• Target: 12
• Locations: 6 countries
• Sites: 13

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)

Keywords

Iptacopan,; PNH,; hemoglobin,; anemia

Outcome Measures

Primary Outcomes (5)

• Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Incidence and severity of AEs and SAEs by treatment group, including changes in vital signs, electrocardiograms (ECGs) and laboratory results qualifying and reported as AEs.

  26 weeks

• PK parameter (Cmax)

  Cmax is defined as the maximum (peak) observed concentration following a dose.

  Week 2

• PK parameter (AUClast)

  AUClast is the area under the plasma concentration-time curve from time zero to the time of last quantifiable concentration (tlast).

  Week 2

• PK parameter (AUCtau)

  AUCtau describes the area under the curve limited to the end of a dosing interval.

  Week 2

• PK parameter (Ctrough)

  Ctrough is the observed plasma concentration that is just prior to the beginning of, or at the end of a dosing interval.

  Weeks 2, 4, 12 and 26

Secondary Outcomes (6)

• Change in hemoglobin (Hb) from baseline ≥1 g/dL (in the absence of RBC transfusions from Day 14).

  Baseline, Week 26, Week 52

• Change in Hb from baseline ≥2 g/dL (in the absence of RBC transfusions from Day 14).

  Baseline, Week 26, Week 52

• Normal Hb in the absence of red blood cell (RBC) transfusions from Day 14.

  Week 26 and Week 52

• Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 26 and Week 52

  Week 26 and Week 52

• Change from baseline in hemoglobin

  Baseline, Week 26, Week 52

Study Arms (2)

LNP023-Cohort 1 (12 < 18 years old)

EXPERIMENTAL

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).

Drug: LNP023

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

EXPERIMENTAL

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.

Drug: LNP023

Interventions

LNP023 DRUG

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.

Also known as: Iptacopan

LNP023 -Cohort 2 (2 to < 12 years old); LNP023-Cohort 1 (12 < 18 years old)

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (13)

Cancer Institute of New Jersey

New Brunswick, New Jersey, 08901, United States

RECRUITING

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104 4399, United States

RECRUITING

St Jude Childrens Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

Novartis Investigative Site

Brasília, Federal District, 70684-831, Brazil

RECRUITING

Novartis Investigative Site

Natal, Rio Grande do Norte, 59012 300, Brazil

RECRUITING

Novartis Investigative Site

Porto Alegre, Rio Grande do Sul, 90035-003, Brazil

RECRUITING

Novartis Investigative Site

Santo André, São Paulo, 09090-401, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 01323001, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 04038-002, Brazil

RECRUITING

Novartis Investigative Site

Cali, Valle del Cauca Department, 760012, Colombia

RECRUITING

Novartis Investigative Site

Berlin, 13353, Germany

RECRUITING

Novartis Investigative Site

Genova, GE, 16147, Italy

RECRUITING

Novartis Investigative Site

Utrecht, 3584 CX, Netherlands

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal; Anemia

Interventions

iptacopan

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

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

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2025
• First Posted: April 18, 2025
• Study Start: October 28, 2025
• Primary Completion (Estimated): November 19, 2031
• Study Completion (Estimated): December 19, 2031
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

IT (1); NL (1); US (3); BR (6); DE (1); CO (1)