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NCT06934967
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
Phase 3 trial testing LNP023 in Paroxysmal Nocturnal Hemoglobinuria (PNH) in 12 participants. Currently enrolling.
19 November 2031
Quick facts
| Lead sponsor | Novartis Pharmaceuticals |
|---|---|
| Phase | Phase 3 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | sequential |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 12 |
| Start date | 28 October 2025 |
| Primary completion | 19 November 2031 |
| Estimated completion | 19 December 2031 |
| Sites | 13 locations across Italy, Colombia, Netherlands, Germany, United States, Brazil |
Drugs / interventions tested
- LNP023 — full drug profile →
Conditions studied
- Paroxysmal Nocturnal Hemoglobinuria (PNH) — all drugs for Paroxysmal Nocturnal Hemoglobinuria (PNH) →
Sponsor
Novartis Pharmaceuticals — full company profile →
Who can join
Adults 2 to 18, any sex, with Paroxysmal Nocturnal Hemoglobinuria (PNH). Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
The varieties of therapeutic experience: navigating treatment options for patients with PNH.
Bienz M, Patriquin CJ. · · 2025 · PMID 41348037 · DOI 10.1182/hematology.2025000701
Verify or expand the search:
- PubMed search for NCT06934967
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of LNP023
Trials testing the same drug.
- NCT06931691 — A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China · recruiting
- NCT04557462 — A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in P · Phase 3 · recruiting
- NCT04558918 — Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antib · Phase 3 · completed
- NCT04578834 — Study of Efficacy and Safety of LNP023 in Primary IgA Nephropathy Patients · Phase 3 · completed
- NCT04154787 — Efficacy and Safety of LNP023 Compared With Rituximab in Subjects With Idiopathic Membranous Nephropathy · Phase 2 · terminated
Other recruiting trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Currently open trials in the same condition.
- NCT07187401 — A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in · Phase 1, PHASE2 · recruiting
- NCT07229235 — REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria · recruiting
- NCT06312644 — Study of Ultomiris® (Ravulizumab) Safety in Pregnancy · recruiting
- NCT06933914 — Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria · Phase 2, PHASE3 · recruiting
- NCT05876312 — Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient · Phase 1, PHASE2 · active not recruiting
Other Novartis Pharmaceuticals trials
Trials by the same sponsor.
- NCT07498335 — Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Atrasentan in Pediatric Patients With Primary · Phase 3 · not yet recruiting
- NCT07489573 — Study of Efficacy and Safety of Secukinumab in Chinese Adult Patients With Moderate to Severe Hidradenitis Suppurativa · Phase 4 · not yet recruiting
- NCT07484269 — PULSE Registry: for Patients Receiving Lutetium (177Lu) Vipivotide Tetraxetan · not yet recruiting
- NCT07416162 — A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy · not yet recruiting
- NCT07387926 — Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+ · Phase 1, PHASE2 · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06934967 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
- Last refreshed: 16 April 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06934967.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing