Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

Официальное название

Prospective Interventional Study of Effectiveness of Efanesoctocog Alfa Prophylaxis on Synovial Hypertrophy in Patients With Hemophilia A

Обзор

The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A. The study duration for each participant is approximately 12 months.

Вмешательства

• Препарат Efanesoctocog alfa
  Pharmaceutical form:Lyophilized powder in a sterile vial that requires reconstitution with sterile water for injection (diluent)-Route of administration:Intravenous

Первичные конечные точки

• Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score [Срок оценки: From Baseline up to Week 52]

Вторичные конечные точки (12)

• Change from baseline in the distribution of joint HEAD-US synovitis score [Срок оценки: From Baseline up to Week 52]
• Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened [Срок оценки: From Baseline up to Week 52]
• Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened [Срок оценки: From Baseline up to Week 52]
• Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened [Срок оценки: From Baseline up to Week 52]
• Percentage of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened [Срок оценки: From Baseline up to Week 52]
• Number of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened [Срок оценки: From Baseline up to Week 52]
• Percentage of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened [Срок оценки: From Baseline up to Week 52]
• Change from baseline in average HEAD-US synovitis domain score across all index joints [Срок оценки: From Baseline up to Week 52]
• Change from baseline in total/ domain scores of the HEAD-US [Срок оценки: From Baseline up to Week 26]
• Change from baseline in total/domain scores of the HJHS [Срок оценки: From Baseline up to Week 26]
• Change from baseline in total/domain scores of the HJHS [Срок оценки: From Baseline up to Week 52]
• Change from baseline in total/ domain scores of magnetic resonance imaging (MRI) [Срок оценки: From Baseline up to Week 52]

Критерии участия

Inclusion Criteria: Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting activity) at the time of consent/assent.

Participant is more than or equal to (≥)12 years old at the time of consent/assent.

Participant has existing synovial hypertrophy, defined as at least 1 eligible\* joint by the HEAD-US score (Synovitis score: 1 or 2) at the time of consent/assent.

Participant has at least 1 eligible\* joint without planned future major orthopedic procedures (example, arthroscopic synovectomy, radioisotopic or chemical synoviorthesis), or major orthopedic procedures in the past 3 months prior to the screening visit (Visit 1).

\*An eligible joint is a joint with existing synovial hypertrophy, as defined by a HEAD-US synovitis score of 1 or 2, considering hypertrophic synovium as an indication of the presence of synovitis Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2).

Participant is capable of understanding the written Informed Consent Form (ICF)/assent form, provides a signed and witnessed written ICF/assent form, and agrees to comply with the protocol requirements.

If male, no contraceptive measures are required for this study.

If female, is not pregnant or breastfeeding, and one of the following conditions applies:

Is a women of nonchildbearing potential (WONCBP) Is a women of childbearing potential (WOCBP) and agrees to use a highly effective contraceptive method, with a failure rate of less than (<)1 percent during the study treatment period (at least until the Week 52/ End of Treatment \[EoT\] visit).

A WOCBP must have a negative serum pregnancy test at the screening visit (Visit 1) Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:

Has other associated clotting disorders at the time of consent/assent. Is already under efanesoctocog alfa treatment. Has a current diagnosis of an factor VIII (FVIII) inhibitor, defined as inhibitor titer ≥0.60 BU/mL.

Has ITI within the last 2 years prior to the baseline visit (Visit 2). Has been enrolled in a concurrent clinical interventional study or exposed to other investigational drug(s) within 3 months prior to screening for this study.

Is currently in an institution because of regulatory or legal order (that is, is a prisoner or a patient who is legally institutionalized).

Is not suitable for participation, whatever the reason, as judged by the investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.

Is an employee or family member of the investigator or site personnel. Is involved in a specific situation during study implementation or the course of the study that may raise ethics considerations.

Has hypersensitivity to efanesoctocog alfa or its components or any of its excipients that, in the opinion of the investigator, contraindicates participation in the study.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 4 центра

• Orthopedic Institute for Children- Site Number : 8400009 — Los Angeles
• Innovative Hematology, Inc.- Site Number : 8400006 — Indianapolis
• University of Iowa- Site Number : 8400004 — Iowa City
• Ohio State University Hospital East- Site Number : 8400001 — Columbus

Япония · 4 центра

• Investigational Site Number : 3920003 — Kawasaki
• Investigational Site Number : 3920002 — Hiroshima
• Investigational Site Number : 3920001 — Saitama
• Investigational Site Number : 3920004 — Tokyo

Тайвань · 3 центра

• Investigational Site Number : 1580003 — Kaohsiung City
• Investigational Site Number : 1580001 — Taichung
• Investigational Site Number : 1580002 — Taipei

Канада · 2 центра

• Investigational Site Number : 1240003 — Hamilton
• Investigational Site Number : 1240004 — Hamilton

Идентификаторы

NCT: NCT06941870 · LPS18168 · U1111-1311-1701

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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