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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-10-28

Study Completion Date

2031-12-19

Brief Summary

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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.

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Detailed Description

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This is a multicenter, open-label, single arm study comprised of an up to a 8-week Screening Period, and a 26-week Treatment Period followed by a 26-week Extension Treatment Period.

This study will enroll a minimum of 12 pediatric patients 2 to \< 18 years of age in a staggered manner into 3 cohorts: Cohort 1 (adolescents 12 to \< 18 years of age, approximately 6 patients), Cohort 2a (6 to \< 12 years of age, approximately 4 patients), and Cohort 2b (2 to \< 6 years of age, approximately 2 patients).

Conditions

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Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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).

Group Type EXPERIMENTAL

LNP023

Intervention Type 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.

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.

Group Type EXPERIMENTAL

LNP023

Intervention Type 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.

Interventions

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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.

Intervention Type DRUG

Other Intervention Names

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Iptacopan

Eligibility Criteria

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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.

Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No