A Phase 3 interventional study of LNP023 in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Novartis Pharmaceuticals. Recruiting at 15 sites in 6 countries. Open to participants aged 2 Years to 18 Years. Per ClinicalTrials.gov, last updated 2026-08-07.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment
Among 16 studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals — 6 open to participants now, 9 under way, 2 of them phase 3. Hemoglobinuria, Paroxysmal · Novartis Pharmaceuticals.
Among the 16 studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals, this one is one of 4 that started in 2025, is one of 5 in phase 3, and was the 13th registered.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's planned enrollment of 12 is below the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →6 of the 16 studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals are open now, in 8 countries. Each count lists the studies.
Of the 30 Hemoglobinuria, Paroxysmal studies recruiting now, 14 (47%) set no upper age limit and 12 (40%) stop at 75 or younger; 12 take children under 18; 4 take healthy volunteers.
This study: 2 Years to 18 Years · all sexes · no healthy volunteers.
The full table on the hub →16 studies; the count open now beside each. This study's row is marked.
First in 2015; this study’s year is marked. 4 started in 2025.
Median enrollment 28 across the 8 interventional studies, 80 across the 7 observational. Of the 7 that completed or were terminated, 6 (86%) have results posted.
Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
2 of the studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04747613Phase 3Novartis PharmaceuticalsLong-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria | 2027-10-18 |
| NCT06931691Novartis PharmaceuticalsA Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China | 2027-04-21 |
Among the 16 studies in Hemoglobinuria, Paroxysmal by Novartis Pharmaceuticals, from their registry updates:
Nothing recorded among them since 2026-09-25.
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.
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).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
Drug: LNP023
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.
Drug: 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.
Also known as: Iptacopan
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Incidence and severity of AEs and SAEs by treatment group, including changes in vital signs, electrocardiograms (ECGs) and laboratory results qualifying and reported as AEs.
Time frame: 26 weeks
PK parameter (Cmax)
Cmax is defined as the maximum (peak) observed concentration following a dose.
Time frame: Week 2
PK parameter (AUClast)
AUClast is the area under the plasma concentration-time curve from time zero to the time of last quantifiable concentration (tlast).
Time frame: Week 2
PK parameter (AUCtau)
AUCtau describes the area under the curve limited to the end of a dosing interval.
Time frame: Week 2
PK parameter (Ctrough)
Ctrough is the observed plasma concentration that is just prior to the beginning of, or at the end of a dosing interval.
Time frame: Weeks 2, 4, 12 and 26
Change in hemoglobin (Hb) from baseline ≥1 g/dL (in the absence of RBC transfusions from Day 14).
To evaluate the proportion of patients who achieve an increase in hemoglobin levels from baseline of ≥ 1 g/dL at 26 weeks and at 52 weeks (in the absence of RBC transfusions from Day 14 until the end of the 26-week and 52-week treatment periods).
Time frame: Baseline, Week 26, Week 52
Change in Hb from baseline ≥2 g/dL (in the absence of RBC transfusions from Day 14).
To evaluate the proportion of patients who achieve an increase in hemoglobin levels from baseline of ≥ 2 g/dL at 26 weeks and at 52 weeks (in the absence of RBC transfusions from Day 14 until the end of the 26-week and 52-week treatment periods).
Time frame: Baseline, Week 26, Week 52
Normal Hb in the absence of red blood cell (RBC) transfusions from Day 14.
To evaluate the proportion of participants achieving hemoglobin normalization at 26 weeks and at 52 weeks (in the absence of RBC transfusions from Day 14 until the end of the 26-week and 52-week treatment periods).
Time frame: Week 26 and Week 52
Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 26 and Week 52
To evaluate transfusion avoidance as the proportion of participants who remain free from transfusions and do not meet transfusion criteria from Day 14 until the end of the 26-week treatment period and until the end of the 52-week treatment period.
Time frame: Week 26 and Week 52
Change from baseline in hemoglobin
To evaluate mean changes from baseline in hemoglobin at 26 weeks and at 52 weeks (evaluation of naive patients and prior anti-C5 treated patients, separately).
Time frame: Baseline, Week 26, Week 52
Change from baseline in lactate dehydrogenase (LDH)
To evaluate mean changes from baseline in LDH at 26 weeks and at 52 weeks (evaluation of naive patients and prior anti-C5 treated patients, separately).
Time frame: Baseline, Week 26, Week 52
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
No publications or documents are linked to this record.
Started Oct 2025 (actual). Primary completion is estimated for Oct 2031, 71 months after the start.
Of 35 industry-sponsored phase 3 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 23 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 41. This study's planned 71 months is above that.
Counting every sponsor, half of 35 studies reached it within 23 months; a quarter within 15 and three quarters within 41.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Iptacopan