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PNH patients get extended access to iptacopan in safety follow-up

NCT ID NCT04747613

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 07, 2026 · Updated 2 times

Summary

This study looks at the long-term safety and tolerability of iptacopan in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have already completed earlier phase 2 or 3 studies with the drug. About 208 participants will continue taking iptacopan and be monitored for side effects and how well the drug controls their disease. The goal is to provide ongoing treatment access while gathering more safety data.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

208 people

The number who actually took part.

Started

Jul 2021

Expected to finish

Oct 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion criteria: * 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 Exclusion Criteria: * 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.

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Conditions

The condition(s) this trial relates to.

anemia Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Paroxysmal nocturnal hemoglobinuria

Contacts and locations

Locations

  • Augusta University

    Augusta, Georgia, 30912, United States

  • Augusta University Georgia

    Augusta, Georgia, 30912, United States

  • City Of Hope

    Duarte, California, 91010, United States

  • City Of Hope National Med Center

    Duarte, California, 91010, United States

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • Lakes Research

    Miami Lakes, Florida, 33014, United States

  • Montefiore Medical Center

    The Bronx, New York, 10461, United States

  • Novartis Investigative Site

    Santo André, São Paulo, 09090-790, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 01323-900, Brazil

  • Novartis Investigative Site

    Beijing, 100730, China

  • Novartis Investigative Site

    Tianjin, 300020, China

  • Novartis Investigative Site

    Tianjin, 300052, China

  • Novartis Investigative Site

    Ostrava, Poruba, 708 52, Czechia

  • Novartis Investigative Site

    Brno, 625 00, Czechia

  • Novartis Investigative Site

    Lille, 59037, France

  • Novartis Investigative Site

    Nantes, 44093, France

  • Novartis Investigative Site

    Nice, 06202, France

  • Novartis Investigative Site

    Paris, 75475, France

  • Novartis Investigative Site

    Toulouse, 31059, France

  • Novartis Investigative Site

    Dresden, Saxony, 01307, Germany

  • Novartis Investigative Site

    Aachen, 52074, Germany

  • Novartis Investigative Site

    Essen, 45147, Germany

  • Novartis Investigative Site

    Hamburg, 20246, Germany

  • Novartis Investigative Site

    Riesa, 01589, Germany

  • Novartis Investigative Site

    Ulm, 89081, Germany

  • Novartis Investigative Site

    Ascoli Piceno, AP, 63100, Italy

  • Novartis Investigative Site

    Avellino, AV, 83100, Italy

  • Novartis Investigative Site

    Florence, FI, 50134, Italy

  • Novartis Investigative Site

    Milan, MI, 20122, Italy

  • Novartis Investigative Site

    Roma, RM, 00161, Italy

  • Novartis Investigative Site

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    Bassano del Grappa, VI, 36061, Italy

  • Novartis Investigative Site

    Nagoya, Aichi-ken, 453-8511, Japan

  • Novartis Investigative Site

    Isehara, Kanagawa, 259-1193, Japan

  • Novartis Investigative Site

    Suwa, Nagano, 392-8510, Japan

  • Novartis Investigative Site

    Suita, Osaka, 5650871, Japan

  • Novartis Investigative Site

    Shinjuku Ku, Tokyo, 160-0023, Japan

  • Novartis Investigative Site

    Fukushima, 9601295, Japan

  • Novartis Investigative Site

    Ishikawa, 9208641, Japan

  • Novartis Investigative Site

    Kyoto, 6068507, Japan

  • Novartis Investigative Site

    Niigata, 9518520, Japan

  • Novartis Investigative Site

    Vilnius, LT-08406, Lithuania

  • Novartis Investigative Site

    Kota Kinabalu, Sabah, 88586, Malaysia

  • Novartis Investigative Site

    Kuching, Sarawak, 93586, Malaysia

  • Novartis Investigative Site

    Nijmegen, 6525 GA, Netherlands

  • Novartis Investigative Site

    Singapore, 119074, Singapore

  • Novartis Investigative Site

    Seoul, Seoul, 03080, South Korea

  • Novartis Investigative Site

    Seoul, Seoul, 06351, South Korea

  • Novartis Investigative Site

    Santiago Compostela, A Coruna, 15706, Spain

  • Novartis Investigative Site

    Donostia / San Sebastian, Gipuzkoa, 20014, Spain

  • Novartis Investigative Site

    Barcelona, 08036, Spain

  • Novartis Investigative Site

    Hualien City, 970, Taiwan

  • Novartis Investigative Site

    Taipei, 10002, Taiwan

  • Novartis Investigative Site

    Istanbul, Fatih, 34093, Turkey (Türkiye)

  • Novartis Investigative Site

    Leeds, West Yorkshire, LS9 7TF, United Kingdom

  • Novartis Investigative Site

    London, SE5 9RS, United Kingdom

  • Prisma Health Upstate

    Greenville, South Carolina, 29615, United States

  • USC Norris Cancer Center

    Los Angeles, California, 90033, United States

  • Univ Cali Irvine ALS Neuromuscular

    Orange, California, 92868, United States

  • Univ of California Irvine (Chao Family Comprehensive Cancer Center)

    Orange, California, 92868, United States

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