Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 3NCT06934967
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
A Phase 3 study testing LNP023 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Novartis Pharmaceuticals at 15 sites in 6 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- LNP023
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 15 sitesin 6 countries
- Study dates
- 28 Oct 2025to 13 Nov 2031
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male and female participants 2 to < 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes/monocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg.
- Patients being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator.
- Patients who are anti-C5 treatment naive: mean hemoglobin level < 10 g/dL confirmed by central laboratory assessment during screening.
- Patients who are anti-C5 treatment naive: lactate dehydrogenase (LDH) > 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab.
- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated.
- Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan.
Who cannot join
- History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes.
- Known or suspected hereditary complement deficiency at screening.
- History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1).
- Patients with laboratory evidence of bone marrow failure (reticulocytes < 100 x 10 to the ninth/L; platelets < 30 × 10 to the ninth/L; neutrophils < 0.5 × 10 to the ninth/L).
- Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration.
- Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration.
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
15 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
15 sites in 6 countries
Brazil6 sites, 6 recruiting
- Recruiting
Novartis Investigative Site
Brasília, Federal District, 70684-831
- Recruiting
Novartis Investigative Site
Natal, Rio Grande do Norte, 59012 300
- Recruiting
Novartis Investigative Site
Porto Alegre, Rio Grande do Sul, 90035-003
- Recruiting
Novartis Investigative Site
Santo André, São Paulo, 09090-401
- Recruiting
Novartis Investigative Site
São Paulo, São Paulo, 01323001
- Recruiting
Novartis Investigative Site
São Paulo, São Paulo, 04038-002
United States4 sites, 4 recruiting
- Recruiting
Childrens Healthcare of Atlanta
Atlanta, Georgia, 30342
Robert Uddo, robert.uddo@choa.org
Satheesh Chonat
- Recruiting
St Jude Childrens Research Hospital
Memphis, Tennessee, 38105
Michelle Brignac, michelle.brignac@stjude.org
Marcin Wlodarski
- Recruiting
Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
Serban Morojanu, +1 732 235 7577, moroiasa@cinj.rutgers.edu
Richard DRACHTMAN
- Recruiting
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399
Peter Nicholas, +1 267 425 2136, nicholasp@email.chop.edu
Timothy Olson
Germany2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Berlin, 13353
- Recruiting
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
Colombia1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Cali, Valle del Cauca Department, 760012
Italy1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Genova, GE, 16147
Netherlands1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Utrecht, 3584 CX
About this study
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
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.
Read the detailed description
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).
- Being tested
- LNP023
- Keywords
- Iptacopan,, PNH,, hemoglobin,, anemia
- Medical subject headings
- Hemoglobinuria, Paroxysmal, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LNP023Drug
Also called Iptacopan
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 group | Role | What they receive |
|---|---|---|
| LNP023-Cohort 1 (12 < 18 years old) | Experimental | 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). Receives: Drug: LNP023 |
| LNP023 -Cohort 2 (2 to < 12 years old) | Experimental | 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. Receives: Drug: LNP023 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 26 weeks
PK parameter (Cmax)
Cmax is defined as the maximum (peak) observed concentration following a dose.
Measured: 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).
Measured: Week 2
PK parameter (AUCtau)
AUCtau describes the area under the curve limited to the end of a dosing interval.
Measured: 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.
Measured: Weeks 2, 4, 12 and 26
Secondary outcomes (6)
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).
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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).
Measured: Baseline, Week 26, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2025
- Study start28 Oct 2025
- Primary completion14 Oct 2031 (estimated)
- Study completion13 Nov 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06934967
- Sponsor's protocol ID
- CLNP023I12201
- Other IDs
- 2024-515926-10
- Status last verified
- Aug 2026
- First submitted
- 31 Mar 2025
- Last updated
- 7 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06934967
- Acronym / sponsor ID
- CLNP023I12201
- Keywords
- Iptacopan,, PNH,, hemoglobin,, anemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 28 Oct 2025 (actual)
- Primary completion
- 14 Oct 2031 (estimated)
- Study completion
- 13 Nov 2031 (estimated)
- First posted
- 18 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LNP023-Cohort 1 (12 < 18 years old) | Experimental | Drug: LNP023Participants (12 to < 18 years old) will take iptacopan at the dose of 200 mg twice per day (in the morning and in the evening). |
| LNP023 -Cohort 2 (2 to < 12 years old) | Experimental | Drug: LNP023Participants (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. |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Male and female participants 2 to < 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes/monocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg.
- Patients being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator.
- Patients who are anti-C5 treatment naive: mean hemoglobin level < 10 g/dL confirmed by central laboratory assessment during screening.
- Patients who are anti-C5 treatment naive: lactate dehydrogenase (LDH) > 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab.
- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated.
Key exclusion criteria (7 listed)
- History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes.
- Known or suspected hereditary complement deficiency at screening.
- History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1).
- Patients with laboratory evidence of bone marrow failure (reticulocytes < 100 x 10 to the ninth/L; platelets < 30 × 10 to the ninth/L; neutrophils < 0.5 × 10 to the ninth/L).
- Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LNP023Also: IptacopanCohort 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. | Drug | LNP023 -Cohort 2 (2 to < 12 years old), LNP023-Cohort 1 (12 < 18 years old) |
Primary outcome
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 26 weeks
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.
PK parameter (Cmax)
Time frame: Week 2
Cmax is defined as the maximum (peak) observed concentration following a dose.
PK parameter (AUClast)
Time frame: Week 2
AUClast is the area under the plasma concentration-time curve from time zero to the time of last quantifiable concentration (tlast).
PK parameter (AUCtau)
Time frame: Week 2
AUCtau describes the area under the curve limited to the end of a dosing interval.
PK parameter (Ctrough)
Time frame: Weeks 2, 4, 12 and 26
Ctrough is the observed plasma concentration that is just prior to the beginning of, or at the end of a dosing interval.
6 secondary outcomes
Change in hemoglobin (Hb) from baseline ≥1 g/dL (in the absence of RBC transfusions from Day 14).
Time frame: Baseline, Week 26, Week 52
Change in Hb from baseline ≥2 g/dL (in the absence of RBC transfusions from Day 14).
Time frame: Baseline, Week 26, Week 52
Normal Hb in the absence of red blood cell (RBC) transfusions from Day 14.
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
Time frame: Week 26 and Week 52
Change from baseline in hemoglobin
Time frame: Baseline, Week 26, Week 52
Change from baseline in lactate dehydrogenase (LDH)
Time frame: Baseline, Week 26, Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
15 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing LNP023 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Novartis Pharmaceuticals at 15 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Brazil, United States, Germany, Colombia, Italy and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: 26 weeks). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2026.