A Study Investigating Intravenous Human Normal Immunoglobulin 10% in Adults With Chronic Immune Thrombocytopenia (ITP)

Официальное название

A Phase III, Open-label, Single Arm, Prospective, Multicenter Study to Assess Efficacy and Safety of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Adult Patients With Chronic Immune Thrombocytopenia (ITP)

Обзор

The purpose of this study is to evaluate the efficacy and safety of KIg 10 (Intravenous Immunoglobulin 10%) in adult patients with chronic primary ITP

Вмешательства

• Биопрепарат Kedrion IVIG 10%
  (Intravenous) Human Normal Immunoglobulin (IVIg) 10%

Первичные конечные точки

• Rate of subjects with response (R) [Срок оценки: Treatment to day 14]

Вторичные конечные точки (6)

• Number and rate of subjects with complete response (CR) [Срок оценки: Treatment to day 14]
• Time to platelet count response [Срок оценки: Treatment to day 14]
• Duration of response [Срок оценки: 14 days after treatment to end of study]
• Regression of hemorrhages [Срок оценки: Day 1 (Visit 1) to Day 30 (End of Study Visit)]
• Platelet count assessment [Срок оценки: Day 1 (Visit 1) to Day 30 (End of Study Visit)]
• Assess safety and tolerability [Срок оценки: Day 1 (Visit 1) to Day 30 (End of Study Visit)]

Критерии участия

Критерии включения

• Male or female, 18-70 years of age.
• Patient has signed the ICF.
• Diagnosis of chronic (> 12 months duration) ITP as defined by the International Working Group.
• Mean screening platelet count of < 30 × 10\^9/L from two qualifying counts measured at least one calendar day apart. The first qualifying count can be from historical data if measured within 7 days prior to screening. The second qualifying count will be measured within 7 days before the first KIg10 infusion.
• A pre-infusion platelet count of < 30 × 10\^9/L at the Baseline Visit.
• Patient is willing to comply with all requirements of the protocol.
• Women of childbearing potential (WOCBP) must have a negative urine pregnancy test at screening and agree to employ adequate birth control measures during the study.
• Authorization to access personal health information.

Критерии исключения

• Patients incapable of giving informed consent.
• Patients with secondary ITP (all forms of immune-mediated thrombocytopenia except primary ITP). e.g., lupus erythematosus, rheumatoid arthritis, drug-related ITP, and Human Immunodeficiency Virus (HIV).
• Patients with Evans Syndrome.
• Patients known to be infected with hepatitis B virus, hepatitis C virus, or HIV.
• History of thrombotic events including deep vein thrombosis, cerebrovascular accident, pulmonary embolism, transient ischemic attacks, or myocardial infarction.
• Patient with a history of hypersensitivity to IVIg, other injectable forms of IVIg, or to any of the excipients.
• Patient unresponsive previously to IVIg or anti-D Ig treatment.
• Patient with known Immunoglobulin A (IgA) deficiency and antibodies against IgA.
• Splenectomy within 4 weeks of the Baseline Visit or planned splenectomy throughout the study period.
• Participants with known inherited thrombocytopenia. e.g., MYH-9 disorders.
• Participants with myelodysplastic syndrome (MDS).
• Administration of IVIg, anti-D immunoglobulin, Mercaptopurine, Vinca alkaloid, or platelet enhancing drugs (including thrombopoietin receptor agonists \[TPO-RA\], immunosuppressive, or other immunomodulatory drugs) within 3 weeks of the Baseline Visit, except for:

• patients on a stable dose of TPO-RA within 4 weeks of the Baseline Visit
• patients on a stable dose of Mycophenolate Mofetil within 3 months of the Baseline Visit
• patients on stable dose of Danazol within 3 months of the Baseline Visit
• long-term corticosteroid therapy for ITP, when the dose had been stable within 3 weeks of the Baseline Visit and no dosage change was planned until the EOS Visit
• long-term azathioprine, cyclophosphamide, or attenuated androgen therapy when the dose had been stable within 3 months of the Baseline Visit, and no dosage change was planned until after study completion. Treatment with any other products licensed for primary chronic ITP is also exclusive. An appropriate wash-out period must be determined in case of patients who might be eligible for treatment with IVIg.
• Received any blood, blood product, or blood derivative within 1 month of the Baseline Visit.
• Received rituximab within 6 months of the Baseline Visit.
• Had a platelet transfusion or receipt of blood products containing platelets within 7 days of Visit 1 (Day 1).
• Received recombinant activated factor VII within 7 days of the Baseline Visit.
• Had therapy with live attenuated virus vaccines within 3 months of the Baseline Visit.
• Use of loop diuretics within 1 week of the Baseline Visit.
• Patients at high risk of thrombotic events.
• Uncontrolled hypertension \[i.e., diastolic blood pressure >100 mmHg and/or systolic blood pressure >160 mmHg\]. If a single measure exceeds this limit, a triple repeat measurement may be performed and the average of the three measurements used.
• Congestive heart failure as per New York Heart Association III/IV, cardiomyopathy, cardiac arrhythmia associated with thromboembolic events (e.g., atrial fibrillation), unstable or advanced ischemic heart disease, hyperviscosity.
• Patients with significant protein losing enteropathy, nephrotic syndrome, or lymphangiectasia.
• Patients with hyperproteinemia, increased serum viscosity, and/or hyponatremia.
• Severe liver or kidney disease (normal reference ranges of laboratory doing the analysis):

• alanine aminotransferase (ALT) or aspartate amino transferase (AST) 2.5x > upper limit of normal (ULN)
• creatinine > 120 μmol/L
• blood urea nitrogen (BUN) > 2.5x ULN
• Signs of severe anemia: Hemoglobin of less than 7 g/dL, hemodynamically unstable due to active bleeding, and/or when evidence of end-organ ischemia secondary to severe anemia is present.
• Body mass index > 40 kg/m2 or an IVIg dose that puts the patient at risk of fluid overload.
• History of a malignant disease within 3 years of the Baseline Visit other than properly treated carcinoma in situ of the cervix or basal cell or squamous cell carcinoma of the skin.
• Patient has participated in an interventional, investigational clinical study within 30 days of the Baseline Visit or within 5 half-lives of the investigational medicinal product (IMP) under investigation.
• Any condition that the Investigator believes is likely to interfere with evaluation of the IMP or with satisfactory conduct of the trial.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Turkey (Türkiye) · 7 центров

• Ankara Üniversitesi Tip Fakültesi - Cebeci Arastirma ve Uygulama Hastanesi — Ankara
• Kocaeli Üniversitesi Arastirma ve Uygulama Hastanesi — Ankara
• Dr. Abdurrahman Yurtaslan Ankara Onkoloji Egitim ve Arastirma Hastanesi — Yenimahalle
• Trakya Üniversitesi Saglik Arastirma ve Uygulama Merkezi — Edirne
• Ege Universitesi Tip Fakultesi — Izmir
• VM Medical Park Mersin Hastanesi — Mersin
• Sakarya Universitesi Egitim ve Arastirma Hastanesi — Adapazarı

Италия · 5 центров

• Azienda Ospedaliero - Universitaria Careggi — Florence
• Azienda Ospedaliero-Universitaria Maggiore della Carità di Novara — Novara
• Azienda Sanitaria Universitaria Giuliano Isontina — Trieste
• Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino — Torino
• AULSS 8 Berica - Ospedale San Bortolo Di Vicenza — Vicenza

Испания · 4 центра

• Complejo Asistencial Universitario de Burgos - Hospital Universitario de Burgos — Burgos
• Instituto De Investigacion Biomedica De A Coruna - Virologia Clinica — A Coruña
• Complejo Hospitalario Ruber Juan Bravo — Madrid
• Hospital General Universitario Gregorio Marañón — Madrid

США · 3 центра

• University of Southern California — Los Angeles
• NY Cancer and Blood Specialists — Shirley
• East Carolina University — Greenville

Румыния · 3 центра

• Coltea - Spital Clinic — Bucharest
• Institutul Oncologic Prof. Dr. Ion Chiricuta — Cluj-Napoca
• Spitalul Filantropia - Craiova — Craiova

Чехия · 2 центра

• Vseobecna Fakultni Nemocnice v Praze — Prague
• Fakultni Nemocnice Brno — Brno

Германия · 2 центра

• Onkologisches Zentrum Donauwörth Neudegger - Onkomedeor Onkologische Zentren — Donauwörth
• Universitätsklinikum Frankfurt — Frankfurt am Main

Сербия · 2 центра

• Univerzitetski Klinicki Centar Srbije — Belgrade
• Klinicko-Bolnicki Centar Zemun — Belgrade

Идентификаторы

NCT: NCT07059000 · KB072 · 2023-507115-35-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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