Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT04747613
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
A Phase 3 study testing Iptacopan in people with Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 60 sites in 17 countries.
Trial summary
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Iptacopan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 208 participants
- Where
- 60 sitesin 17 countries
- Study dates
- 27 Jul 2021to 18 Oct 2027
- 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 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension.
- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
- Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months
Who cannot join
- Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
- History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- History of hematopoietic stem cell transplantation
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
60 sites in 17 countries.
60 sites in 17 countries
United States11 sites
Augusta University Georgia
Augusta, Georgia, 30912
Augusta University
Augusta, Georgia, 30912
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
City Of Hope National Med Center
Duarte, California, 91010
City Of Hope
Duarte, California, 91010
Prisma Health Upstate
Greenville, South Carolina, 29615
USC Norris Cancer Center
Los Angeles, California, 90033
Lakes Research
Miami Lakes, Florida, 33014
Univ Cali Irvine ALS Neuromuscular
Orange, California, 92868
Univ of California Irvine (Chao Family Comprehensive Cancer Center)
Orange, California, 92868
Montefiore Medical Center
The Bronx, New York, 10461
Japan9 sites
Novartis Investigative Site
Fukushima, 9601295
Novartis Investigative Site
Isehara, Kanagawa, 259-1193
Novartis Investigative Site
Ishikawa, 9208641
Novartis Investigative Site
Kyoto, 6068507
Novartis Investigative Site
Nagoya, Aichi-ken, 453-8511
Novartis Investigative Site
Niigata, 9518520
Novartis Investigative Site
Shinjuku Ku, Tokyo, 160-0023
Novartis Investigative Site
Suita, Osaka, 5650871
Novartis Investigative Site
Suwa, Nagano, 392-8510
Italy7 sites
Novartis Investigative Site
Ascoli Piceno, AP, 63100
Novartis Investigative Site
Avellino, AV, 83100
Novartis Investigative Site
Bassano del Grappa, VI, 36061
Novartis Investigative Site
Florence, FI, 50134
Novartis Investigative Site
Milan, MI, 20122
Novartis Investigative Site
Roma, RM, 00161
Novartis Investigative Site
Torino, TO, 10126
Germany6 sites
Novartis Investigative Site
Aachen, 52074
Novartis Investigative Site
Dresden, Saxony, 01307
Novartis Investigative Site
Essen, 45147
Novartis Investigative Site
Hamburg, 20246
Novartis Investigative Site
Riesa, 01589
Novartis Investigative Site
Ulm, 89081
France5 sites
Novartis Investigative Site
Lille, 59037
Novartis Investigative Site
Nantes, 44093
Novartis Investigative Site
Nice, 06202
Novartis Investigative Site
Paris, 75475
Novartis Investigative Site
Toulouse, 31059
China3 sites
Novartis Investigative Site
Beijing, 100730
Novartis Investigative Site
Tianjin, 300020
Novartis Investigative Site
Tianjin, 300052
Spain3 sites
Novartis Investigative Site
Barcelona, 08036
Novartis Investigative Site
Donostia / San Sebastian, Gipuzkoa, 20014
Novartis Investigative Site
Santiago Compostela, A Coruna, 15706
Brazil2 sites
Novartis Investigative Site
Santo André, São Paulo, 09090-790
Novartis Investigative Site
São Paulo, São Paulo, 01323-900
Czechia2 sites
Novartis Investigative Site
Brno, 625 00
Novartis Investigative Site
Ostrava, Poruba, 708 52
Malaysia2 sites
Novartis Investigative Site
Kota Kinabalu, Sabah, 88586
Novartis Investigative Site
Kuching, Sarawak, 93586
South Korea2 sites
Novartis Investigative Site
Seoul, Seoul, 03080
Novartis Investigative Site
Seoul, Seoul, 06351
Taiwan2 sites
Novartis Investigative Site
Hualien City, 970
Novartis Investigative Site
Taipei, 10002
United Kingdom2 sites
Novartis Investigative Site
Leeds, West Yorkshire, LS9 7TF
Novartis Investigative Site
London, SE5 9RS
Turkey (Türkiye)1 site
Novartis Investigative Site
Istanbul, Fatih, 34093
About this study
Official title: An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan
Read the detailed description
The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Iptacopan
- Keywords
- Iptacopan, Paroxysmal Nocturnal Hemoglobinuria (PNH), Hemoglobin, Anemia, LNP023
- 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.
IptacopanDrug
Also called LNP023
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
| Study group | Role | What they receive |
|---|---|---|
| Iptacopan | Experimental | Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy Receives: Drug: Iptacopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with adverse events
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
Measured: Up to 74 months
Secondary outcomes (4)
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
Measured: Up to 74 months
Proportion of participants who remain free from transfusions
Proportion of participants who remain free from transfusions evaluated over yearly intervals
Measured: Up to 74 months
Rate of breakthrough hemolysis (BTH)
Rate of breakthrough hemolysis (BTH)
Measured: Up to 74 months
Proportion of participants with Major Adverse Vascular Events MAVEs
MAVEs (incl. thrombosis) evaluated over yearly intervals
Measured: Up to 74 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2021
- Study start27 Jul 2021
- Primary completion18 Oct 2027 (estimated)
- Study completion18 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 208 participants (actual)
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
- NCT04747613
- Sponsor's protocol ID
- CLNP023C12001B
- Other IDs
- 2023-509843-28-00, 2023
- Status last verified
- Aug 2026
- First submitted
- 5 Feb 2021
- Last updated
- 7 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- 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
Publications
DeCastro C, Sheinberg P, Han B, Vallow S, Bermann G, Dhalke M, Kumar R, Dickie G, Galipeau N, Lamoureux R, Rupinski K, Lowe C, Nieves A, de Fontbrune FS, de Latour RP. Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan. Patient. 2025 Nov;18(6):699-712. doi: 10.1007/s40271-025-00755-5. Epub 2025 Jul 22.
PubMed 40694294
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, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan
- ClinicalTrials.gov ID
- NCT04747613
- Acronym / sponsor ID
- CLNP023C12001B
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Iptacopan, Paroxysmal Nocturnal Hemoglobinuria (PNH), Hemoglobin, Anemia, LNP023
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 208 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 27 Jul 2021 (actual)
- Primary completion
- 18 Oct 2027 (estimated)
- Study completion
- 18 Oct 2027 (estimated)
- First posted
- 10 Feb 2021 (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.
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan
Detailed description
The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Iptacopan | Experimental | Drug: IptacopanParticipants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension.
- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
- Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months
Key exclusion criteria (4 listed)
- Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
- History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- History of hematopoietic stem cell transplantation
- Other protocol-defined inclusion/exclusion criteria may apply.
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 |
|---|---|---|
| IptacopanAlso: LNP023Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral | Drug | Iptacopan |
Primary outcome
Proportion of participants with adverse events
Time frame: Up to 74 months
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
4 secondary outcomes
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Time frame: Up to 74 months
Proportion of participants who remain free from transfusions
Time frame: Up to 74 months
Rate of breakthrough hemolysis (BTH)
Time frame: Up to 74 months
Proportion of participants with Major Adverse Vascular Events MAVEs
Time frame: Up to 74 months
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.
- Derived from this study: PubMed 40694294DeCastro C, Sheinberg P, Han B, Vallow S, Bermann G, Dhalke M, Kumar R, Dickie G, Galipeau N, Lamoureux R, Rupinski K, Lowe C, Nieves A, de Fontbrune FS, de Latour RP. Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan. Patient. 2025 Nov;18(6):699-712. doi: 10.1007/s40271-025-00755-5. Epub 2025 Jul 22.
- 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
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
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. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
60 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Iptacopan in people with Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 60 sites in 17 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 3 inclusion and 4 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: under way, not looking for participants, 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 60 sites in United States, Japan, Italy, Germany, France and 12 more countries. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants with adverse events (time frame: Up to 74 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04747613, which also lists 1 linked publication.
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.