Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN

NCT ID: NCT05755386

Last Updated: 2025-12-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

106 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-10-02

Study Completion Date

2029-05-31

Brief Summary

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This study is designed as a multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in idiopathic immune complex mediated membranoproliferative glomerulonephritis.

Detailed Description

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The purpose of this Phase III study is to evaluate the efficacy and safety of iptacopan compared to placebo (both administered in combination with standard of care) in participants (adults and adolescents aged 12-17 years) with idiopathic IC-MPGN. The study aims to demonstrate a reduction in proteinuria and improvement in estimated glomerular filtration rate (eGFR) in participants treated with iptacopan compared to placebo. Change in patient-reported fatigue will also be evaluated. Alternative complement pathway (AP) dysregulation is believed to underlie the clinical manifestations and progression of IC-MPGN. Upon completion of study treatment, participants will have the option to discontinue iptacopan treatment and enter a 30 day safety follow-up or continue iptacopan treatment by transitioning to an open label extension study (CLNP023B12001B; NCT03955445) and continue iptacopan treatment.

Conditions

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IC-MPGN

Keywords

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LNP023 IC-MPGN iptacopan UPCR eGFR proteinuria

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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iptacopan 200mg b.i.d

iptacopan 200mg b.i.d

Group Type EXPERIMENTAL

iptacopan

Intervention Type DRUG

iptacopan 200 mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)

Placebo to iptacopan 200mg b.i.d.

Placebo to iptacopan 200mg b.i.d.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo to iptacopan 200mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)

Interventions

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Placebo

Placebo to iptacopan 200mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)

Intervention Type DRUG

iptacopan

iptacopan 200 mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Male and female patients including adults (aged at least 18 years to ≤ 60 years) and adolescents (12 -17 years in non-EU countries at screening and 16-17 years in EU countries at screening).
* Diagnosis of idiopathic IC-MPGN as confirmed by kidney biopsy within 12 months prior to screening in adults and within 3 years of screening in adolescents (a biopsy report, review and confirmation by the Investigator is required). If such a biopsy is not available in an adult participant, this must be obtained at screening (performed and assessed locally for adults only).
* Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of renin angiotensin system inhibitors (RASi), e.g an ACEi or ARB for at least 90 days (or as according to local guidelines). The doses of other drugs administered to reduce proteinuria and control the disease including mycophenolic acids (MPAs - mycophenolate mofetil or mycophenolate sodium), corticosteroids, SGLT2 inhibitors and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization
* UPCR ≥ 1.0 g/g (≥ 113 mg/mmol) sampled from the first morning void urine sample at Day -75 and Day -15
* Estimated GFR (using the chronic kidney disease \[CKD\]-EPI formula for adult participants and modified Schwartz formula for adolescents aged 12 to 17 years) or measured GFR ≥ 30 ml/min/1.73m2 at screening and Day -15.
* Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, the vaccine should be given according to local regulations at least 2 weeks prior to the first administration of study treatment. If the study treatment has to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment should be initiated in accordance with local standard of care.
* If not previously vaccinated, or if a booster is required, vaccination against Haemophilus influenzae infections should be given, if available and according to local regulations, at least 2 weeks prior to the first study treatment administration.

Exclusion Criteria

* Participants who have undergone cell or solid organ transplantation, including kidney transplantation.
* Participants diagnosed with secondary IC-MPGN including but not limited to any of the following conditions:
* Deposition of antigen-antibody immune complexes as a result of any chronic infections, including

* Hepatitis C virus (HCV) including HCV-associated mixed cryoglobulinemia, hepatitis B virus (HBV);
* Bacterial-endocarditis, infected ventriculo-atrial shunt, visceral abscesses, leprosy, meningococcal meningitis; chronic bacterial infections
* Protozoa/other infections- malaria, schistosomiasis, mycoplasma, leishmaniasis, filariasis, histroplasmosis

Renal deposition of immune complexes as a result of a systemic autoimmune disease:

* Systemic lupus erythematosus (SLE)
* Sjögren syndrome
* Rheumatoid arthritis
* Mixed connective tissue disease Deposition of monoclonal immunoglobulins because of a monoclonal gammopathy due to plasma cell or B cell disorders. Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.

Fibrillary glomerulonephritis

* Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in the eGFR within 3 months with kidney biopsy findings of glomerular crescent formation seen in at least 50% of glomeruli on the most recent biopsy.
* Kidney biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) of more than 50%.
* Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration or the presence of fever ≥ 38°C (100.4°F) within 7 days prior to study treatment administration.
* A history of recurrent invasive infections caused by encapsulated organisms, e.g., Neisseria meningitidis and Streptococcus pneumoniae.
* The use of inhibitors of complement factors (e.g., Factor B, Factor D, complement 3 (C3) inhibitors, anti-Complement 5 (C5) antibodies, C5a receptor antagonists) within 3 months or 5 half-lives prior to the Screening visit.
* The use of immunosuppressants (except MPAs), cyclophosphamide or systemic corticosteroids at a dose \>7.5 mg/day (or equivalent for a similar corticosteroid medication) within 90 days of study drug administration.
* The use of MPAs is not permitted within 90 days prior to randomization in India, as per the local health authority requirement.
* Acute post-infectious glomerulonephritis at screening, based upon the opinion of the investigator.
* Body mass index (BMI) \>38 kg/m2 at screening and randomization. Body weight \<35 kg at screening and randomization
Minimum Eligible Age

12 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Ronald Reagan UCLA Medical Center

Los Angeles, California, United States

Site Status RECRUITING

Univ Cali Irvine ALS Neuromuscular

Orange, California, United States

Site Status RECRUITING

UCSF

San Francisco, California, United States

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Olive View UCLA Medical Center

Sylmar, California, United States

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Childrens Hospital Colorado

Aurora, Colorado, United States

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Nicklaus Childrens Hospital

Miami, Florida, United States

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Massachusetts General Hospital

Boston, Massachusetts, United States

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Boston Univ School of Medicine

Boston, Massachusetts, United States

Site Status RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

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University of New Mexico

Albuquerque, New Mexico, United States

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Col Uni Med Center New York Presby

New York, New York, United States

Site Status RECRUITING

Univ of Pennsylvania Medical Center

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Prolato Clinical Research Center

Houston, Texas, United States

Site Status RECRUITING

Baylor Scott and White Research

Temple, Texas, United States

Site Status RECRUITING

University of Utah

Salt Lake City, Utah, United States

Site Status RECRUITING

University of Wisconsin

Madison, Wisconsin, United States

Site Status RECRUITING

Novartis Investigative Site

CABA, Buenos Aires, Argentina

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CABA, Buenos Aires, Argentina

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Buenos Aires, , Argentina

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CABA, , Argentina

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Brasília, Federal District, Brazil

Site Status WITHDRAWN

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Belo Horizonte, Minas Gerais, Brazil

Site Status RECRUITING

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Recife, Pernambuco, Brazil

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Niterói, Rio de Janeiro, Brazil

Site Status WITHDRAWN

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Rio de Janeiro, Rio de Janeiro, Brazil

Site Status WITHDRAWN

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Porto Alegre, Rio Grande do Sul, Brazil

Site Status RECRUITING

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Botucatu, São Paulo, Brazil

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Santo André, São Paulo, Brazil

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Sao Jose Rio Preto, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

Site Status RECRUITING

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Salvador, , Brazil

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Etobicoke, Ontario, Canada

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Toronto, Ontario, Canada

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Montreal, Quebec, Canada

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Montreal, Quebec, Canada

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Prague, , Czechia

Site Status ACTIVE_NOT_RECRUITING

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Marseille, , France

Site Status RECRUITING

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Montpellier, , France

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Paris, , France

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Rennes, , France

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Toulouse, , France

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Munich, Bavaria, Germany

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Würzburg, Bavaria, Germany

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Dresden, Saxony, Germany

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Jena, Thuringia, Germany

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Berlin, , Germany

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Essen, , Germany

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Hamburg, , Germany

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Hanover, , Germany

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Mainz, , Germany

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Ulm, , Germany

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Athens, , Greece

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Chaïdári, , Greece

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Heraklion Crete., , Greece

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Ioannina, , Greece

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Pátrai, , Greece

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Thessaloniki, , Greece

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Bangalore, Karnataka, India

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Nagpur, Maharashtra, India

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Pune, Maharashtra, India

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New Delhi, National Capital Territory of Delhi, India

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Hyderabad, Telangana, India

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Haifa, , Israel

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Jerusalem, , Israel

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Petah Tikva, , Israel

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Bari, BA, Italy

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Ranica, BG, Italy

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Brescia, BS, Italy

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Milan, MI, Italy

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Roma, RM, Italy

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Torino, TO, Italy

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Napoli, , Italy

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Fuchū, Tokyo, Japan

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Hachiōji, Tokyo, Japan

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Okayama, , Japan

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Osaka, , Japan

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Groningen, , Netherlands

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Poznan, Greater Poland Voivodeship, Poland

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Bialystok, , Poland

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Krakow, , Poland

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Olsztyn, , Poland

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Warsaw, , Poland

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Warsaw, , Poland

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Wroclaw, , Poland

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Martin, Slovakia, Slovakia

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Košice, , Slovakia

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Seoul, Korea, South Korea

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Seoul, , South Korea

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Pamplona, Navarre, Spain

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Almería, , Spain

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Barcelona, , Spain

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Barcelona, , Spain

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Madrid, , Spain

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Madrid, , Spain

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Madrid, , Spain

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Salamanca, , Spain

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Bern, , Switzerland

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Lausanne, , Switzerland

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Zurich, , Switzerland

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Kaohsiung City, , Taiwan

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Istanbul, Fatih, Turkey (Türkiye)

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Izmir, Karsiyaka, Turkey (Türkiye)

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Köseköy, Kocaeli, Turkey (Türkiye)

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Kayseri, Melikgazi, Turkey (Türkiye)

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Ankara, Yenimahalle, Turkey (Türkiye)

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Mersin, Yenisehir, Turkey (Türkiye)

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Belfast, , United Kingdom

Site Status RECRUITING

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Cardiff, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

Site Status RECRUITING

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Salford, , United Kingdom

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Novartis Investigative Site

Ho Chi Minh City, , Vietnam

Site Status RECRUITING

Countries

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United States Argentina Brazil Canada Czechia France Germany Greece India Israel Italy Japan Netherlands Poland Slovakia South Korea Spain Switzerland Taiwan Turkey (Türkiye) United Kingdom Vietnam

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

Phone: 1-888-669-6682

Email: [email protected]

Novartis Pharmaceuticals

Role: CONTACT

Phone: +41613241111

Facility Contacts

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Ahad Qureshi

Role: primary

Nancy Ortega

Role: primary

Juan Espinoza

Role: primary

Rosario Machicado

Role: primary

Kati Dugan

Role: primary

Moya Chang

Role: primary

Andrew Cao

Role: primary

Beth Hakamy

Role: primary

Karen Omlung

Role: primary

Susan L Tigert

Role: primary

Anup Pradhan

Role: primary

Fabrizio Angeles

Role: primary

Romeo Parada

Role: primary

Stephanie McMeen

Role: primary

Gabriella Gourdin

Role: primary

Alana Quackenbush

Role: primary

Related Links

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https://doi.org/10.1016/j.ekir.2024.02.240

WCN24-1146 SAFETY AND EFFICACY OF IPTACOPAN IN ADOLESCENT PATIENTS WITH IC-MPGN in Elsivier Journal

Other Identifiers

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2022-002328-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CLNP023B12302

Identifier Type: -

Identifier Source: org_study_id