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

Part of paid clinical trials in New Brunswick, New Jersey.

Sponsor
Novartis Pharmaceuticals
Study ID
NCT06934967
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility Criteria

Sex
ALL
Age
2 Years - 18 Years
Healthy Volunteers
Not accepted

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.

Study Details

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.

Key Dates

Start date
Oct 28, 2025
Status verified
Apr 2026
Primary completion
Nov 19, 2031
Completion
Dec 19, 2031

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

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.

Primary Outcome Measure

Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 26 weeks ]

Central Contacts

  • Novartis Pharmaceuticals
    1-888-669-6682
    [email protected]
  • Novartis Pharmaceuticals
    +41613241111

Locations (3)

FacilityCityStateZIPSite coordinators
Cancer Institute of New JerseyNew BrunswickNew Jersey08901
Serban Morojanu
[email protected]
+1 732-235-7577
Richard DRACHTMAN (PRINCIPAL_INVESTIGATOR)
Childrens Hospital of PhiladelphiaPhiladelphiaPennsylvania19104 4399
Ariel Rivera
[email protected]
+1 267 425 2136
Timothy Olson (PRINCIPAL_INVESTIGATOR)
St Jude Childrens Research HospitalMemphisTennessee38105
Marcin Wlodarski (PRINCIPAL_INVESTIGATOR)

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By research site
Cancer Institute of New Jersey· New Brunswick, NJChildrens Hospital of Philadelphia· Philadelphia, PASt Jude Childrens Research Hospital· Memphis, TN

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