Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
Public ClinicalTrials.gov record NCT06934967. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT06934967
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 12 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years to 18 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 27, 2025
- Primary completion
- Oct 13, 2031
- Completion
- Nov 12, 2031
- Last update posted
- Aug 6, 2026
2025 – 2031
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Childrens Healthcare of Atlanta | Atlanta | Georgia | 30342 | Recruiting |
| Cancer Institute of New Jersey | New Brunswick | New Jersey | 08901 | Recruiting |
| Childrens Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104-4399 | Recruiting |
| St Jude Childrens Research Hospital | Memphis | Tennessee | 38105 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 11 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06934967, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 6, 2026 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06934967 live on ClinicalTrials.gov.