Идёт набор NCT06544499
A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: Efgartigimod IV, Placebo IV.
- Кому может быть актуально
- Состояния в реестре: Primary Immune Thrombocytopenia (ITP). Базовые параметры: от 18 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- США, Австрия, Болгария, Китай, Хорватия +12
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm Study Followed by an Open-Label Arm to Evaluate the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
Обзор
The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively during the double-blinded treatment period (DBTP). At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first 52-week open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second 52-week OLTP2. After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks. More information can be found here: https://clinicaltrials.argenx.com/advancenext
Вмешательства
- Биопрепарат Efgartigimod IV
Intravenous infusion of efgartigimod - Другое Placebo IV
Intravenous infusion of placebo
Первичные конечные точки
- Extent of disease control, defined as the number of cumulative weeks during the 24-week Double-Blinded Treatment Period with platelet counts of at least 50 × 10^9/L [Срок оценки: Up to 24 weeks]
Вторичные конечные точки (12)
- Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 study visits between study weeks 19 and 24 of the DBTP [Срок оценки: Up to 6 weeks]
- Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 study visits between study weeks 17 and 24 of the DBTP [Срок оценки: Up to 8 weeks]
- Proportion of participants achieving a platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the DBTP [Срок оценки: Up to 12 weeks]
- Proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions at any time until study week 12 during the DBTP [Срок оценки: Up to 12 weeks]
- Time to response, defined as the time to achieve 2 consecutive platelet counts of at least 50 × 10^9/L at any time during the DBTP [Срок оценки: up to 24 weeks]
- Proportion of participants with an IWG response during the DBTP [Срок оценки: Up to 24 weeks]
- Proportion of participants with initial response, defined as a platelet count of at least 30 × 10^9/L and a 2-fold increase from baseline in platelet count at study week 5 of the DBTP [Срок оценки: Up to 20 weeks]
- Time to achieve a platelet count of at least 30 × 10^9/L and a 2-fold increase from baseline in platelet count during the DBTP [Срок оценки: Up to 24 weeks]
- Number of cumulative weeks during the DBTP with platelet counts of at least 30 × 10^9/L and ≥20 × 10^9/L above baseline [Срок оценки: Up to 24 weeks]
- Number of cumulative weeks during the DBTP with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline in participants with baseline platelet counts of <15 × 10^9/L [Срок оценки: Up to 24 weeks]
- Extent of disease control, defined as the percentage of weeks with platelet counts of at least 50 × 10^9/L [Срок оценки: Up to 76 weeks]
- In participants receiving placebo IV in the DBTP, the extent of disease control, defined as the number of cumulative weeks with platelet counts of at least 50 × 10^9/L [Срок оценки: Up to 76 weeks]
Критерии участия
Критерии включения
- Is at least 18 years of age and the local legal age of consent for clinical studies when signing the informed consent form (ICF).
- Has documented baseline mean platelet count of <30 x 10\^9/L before randomization
- Has a documented duration of primary immune thrombocytopenia (ITP) of more than 12 months on the date of informed consent form (ICF) signature.
- Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab.
- Has documented insufficient response to a prior ITP treatment (the specific criteria can be found in the protocol).
- Has documented prior response defined as 1 platelet count of ≥50 × 109/L to at least 1 of the following ITP treatments in the 3 years before the date of ICF signature: prednisone, dexamethasone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin
Критерии исключения
- Other than the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of ITP, confound the results of the study or put the participant at undue risk.
- Secondary ITP
- Nonimmune thrombocytopenia
- Autoimmune hemolytic anemia
- ITP-associated critical or severe bleeding The complete list of criteria can be found in the protocol.
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Рандомизированное
- Модель
- Параллельные группы
- Маскирование
- Четверное слепое
- Основная цель
- Лечение
Центры проведения
США · 18 центров
- Mayo Clinic Hospital Scottsdale — Phoenix
- University of Southern California Norris Comprehensive Cancer Center — Los Angeles
- Sharp Memorial Hospital — Oceanside
- University of Colorado Hospital — Aurora
- Yale University School of Medicine — New Haven
- Georgetown University Hospital — Washington D.C.
- The University of Chicago Medicine — Chicago
- The University of Iowa Hospitals & Clinics — Iowa City
- … и ещё 10 центров
Италия · 12 центров
- Azienda ULSS 7 Pedemontana — Bassano del Grappa
- Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC) — Florence
- Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico — Milan
- Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico — Milan
- ASST Grande Ospedale Metropolitano Niguarda — Milan
- AO Maggiore Della Carita — Novara
- U.O.C. Ematologia Ospedale Santa Maria delle Croci — Ravenna
- Arcispedale Santa Maria Nuova (ASMN) Istituto di Ricovero e Cura a Carattere Scientifico ( — Reggio Emilia
- … и ещё 4 центра
Китай · 11 центров
- Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University — Пекин
- Peking University Third Hospital — Пекин
- The First Affiliated Hospital Of Bengbu Medical College — Bengbu
- The Affiliated Hospital of Chengde Medical University — Chengde
- The Affiliated Hospital of Inner Mongolia Medical University — Hohhot
- The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University — Huai'an
- Huizhou Central People's Hospital — Huizhou
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & P — Тяньцзинь
- … и ещё 3 центра
Великобритания · 8 центров
Список центров уточняется — проверьте первичный протокол.
Польша · 7 центров
- Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy — Bydgoszcz
- Pratia Onkologia Katowice — Katowice
- Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodz — Lodz
- NZOZ Neuromed M. i M. Nastaj Sp.P. — Lublin
- Uniwersytecki Szpital Kliniczny Nr 4 w Lublinie Centrum Innowacyjnych Terapii — Lublin
- Szpital Specjalistyczny im. Jedrzeja Sniadeckiego w Nowym Saczu — Nowy Sącz
- Wojewodzki Szpital Zespolony im. Ludwika Rydygiera w Toruniu — Torun
Болгария · 6 центров
- Acibadem City clinic Tokuda hospital — Sofia
- UMHAT Aleksandrovska — Sofia
- University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski — Sofia
- National Specialized Hospital for Active Treatment of Hematology Diseases — Sofia
- UMHAT SofiaMed — Sofia
- University Hospital Prof. Dr. Stoyan Kirkovich AD — Stara Zagora
Испания · 6 центров
Список центров уточняется — проверьте первичный протокол.
Сербия · 5 центров
- Clinical Center of Serbia — Belgrade
- University Clinical Centre of Kragujevac — Kragujevac
- Clinical Center of Nis — Niš
- University Clinical Hospital Center Zemun — Zemun
- General Hospital Djordje Joanovic — Zrenjanin
Румыния · 4 центра
- Spitalul Clinic Coltea, Clinica de Hematologie — Bucharest
- Spitalul Clinic Colentina — Bucharest
- Spitalul Clinic Judetean de Urgenta Sibiu (SCJS) (Clinical County Hospital Sibiu) — Sibiu
- Spitalul Clinic Judetean de Urgenta Targu-Mures — Târgu Mureş
Австрия · 3 центра
- Allgemeines Krankenhaus der Stadt Linz — Linz
- Ordensklinikum Linz GmbH Elisabethinen — Linz
- Klinikum Kreuzschwestern Wels — Wels
Франция · 3 центра
- CHU d'Amiens-Picardie - Hopital SUD — Amiens
- Centre Hospitalier Universitaire (CHU) - Hopital Henri Mondor — Créteil
- Centre Hospitalier Universitaire Dijon Bourgogne Medecine Interne et Immunologie Clinique — Dijon
Чехия · 2 центра
- Fakultni Nemocnice Ostrava — Ostrava
- Fakultni nemocnice Kralovske Vinohrady — Prague
Германия · 2 центра
- University Hospital Carl Gustav Carus — Dresden
- Universitaetsklinikum Essen (AoR) — Essen
Венгрия · 2 центра
- Semmelweis University-Internal Medicine and Haematology — Budapest
- Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz — Győr
Ирландия · 2 центра
- Mater Misericordiae University Hospital — Dublin
- St James's Hospital - Cancer Clinical Trials Office — Dublin
Португалия · 2 центра
- Unidade Local de Saude de Braga — Braga
- Unidade Local de Saude Gaia e Espinho, E.P.E. — Vila Nova de Gaia
Хорватия · 1 центр
- University Hospital Center Zagreb — Zagreb
Идентификаторы
NCT: NCT06544499 · ARGX-113-2402 · 2024-515451-38-00