Actively Recruiting
Study to Assess Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria Aged 2 to Under 18 Years
Led by Novartis Pharmaceuticals · Updated on 2026-04-16
12
Participants Needed
13
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the pharmacokinetics, safety, and tolerability of iptacopan in children with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. This open-label, single-arm, phase 3 trial includes pediatric patients aged 2 to under 18 years. The study aims to understand how iptacopan acts in the body and its safety when used in young PNH patients. Participants are grouped into three cohorts based on age: adolescents 12 to under 18 years, children 6 to under 12 years, and younger children 2 to under 6 years. Adolescents will take 200 mg of iptacopan twice daily. Younger participants will receive doses based on their weight, with dose adjustments at weeks 12, 26, and 38 if needed. The study includes an up to 8-week screening period, a 26-week treatment period, and a 26-week extension treatment period. During the study, participants will have regular assessments including blood tests to measure drug levels and monitor safety. Researchers will track adverse events and changes in hemoglobin and lactate dehydrogenase levels. The primary outcomes focus on drug concentration measures and safety over 26 weeks, with additional assessments extending to 52 weeks. Participants will be monitored throughout treatment and extension phases to evaluate the drug’s effects and tolerability.
CONDITIONS
Brief Title
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female patients aged 2 to under 18 years with confirmed PNH diagnosis by high-sensitivity flow cytometry
- Minimum body weight of 35 kg for patients in the adolescent cohort (12 to under 18 years)
- Patients treated with anti-C5 therapy on a stable dose and interval for at least 6 months prior to enrollment
- Anti-C5 treatment naive patients with mean hemoglobin level under 10 g/dL confirmed during screening
- Anti-C5 treatment naive patients with lactate dehydrogenase (LDH) greater than 1.5 times the upper limit of normal documented by two lab measurements 2 to 6 weeks apart
- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae prior to study start; booster vaccination or vaccination if not previously given, at least 2 weeks before treatment
- Vaccination against Haemophilus influenzae recommended at least 2 weeks before iptacopan administration
You will not qualify if you...
- History of hypersensitivity to iptacopan or similar drugs
- Known or suspected hereditary complement deficiency at screening
- History of hematopoietic stem cell transplantation or planned transplantation within 52 weeks of enrollment
- Laboratory evidence of bone marrow failure (reticulocytes less than 100 x 10^9/L; platelets less than 30 x 10^9/L; neutrophils less than 0.5 x 10^9/L)
- Active bacterial, viral (including COVID-19), or fungal infection within 14 days before study drug administration
- Fever of 38°C (100.4°F) or higher within 7 days before study drug administration
- Other protocol-defined inclusion or exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 8 weeks
Participants are screened for eligibility to participate in the trial.
Screening and enrollment visit
Duration - 26 weeks
Participants take iptacopan orally with dosing based on age and weight to assess pharmacokinetics, safety, and tolerability.
Visits at Day 1, Weeks 2, 4, 12, and 26 for assessments and dose adjustments
Duration - 26 weeks
Participants continue iptacopan treatment in an extension period to further assess safety and efficacy.
Visits at Weeks 38 and 52 for dose reassessment and follow-up
Trial Site Locations
Total: 13 locations
1
Cancer Institute of New Jersey
New Brunswick, New Jersey, United States, 08901
Actively Recruiting
2
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104 4399
Actively Recruiting
3
St Jude Childrens Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
4
Novartis Investigative Site
Brasília, Federal District, Brazil, 70684-831
Actively Recruiting
5
Novartis Investigative Site
Natal, Rio Grande do Norte, Brazil, 59012 300
Actively Recruiting
6
Novartis Investigative Site
Porto Alegre, Rio Grande do Sul, Brazil, 90035-003
Actively Recruiting
7
Novartis Investigative Site
Santo André, São Paulo, Brazil, 09090-401
Actively Recruiting
8
Novartis Investigative Site
São Paulo, São Paulo, Brazil, 01323001
Actively Recruiting
9
Novartis Investigative Site
São Paulo, São Paulo, Brazil, 04038-002
Actively Recruiting
10
Novartis Investigative Site
Cali, Valle del Cauca Department, Colombia, 760012
Actively Recruiting
11
Novartis Investigative Site
Berlin, Germany, 13353
Actively Recruiting
12
Novartis Investigative Site
Genova, GE, Italy, 16147
Actively Recruiting
13
Novartis Investigative Site
Utrecht, Netherlands, 3584 CX
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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