Menu
Recruiting NCT06941870

Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

Phase IV Interventional Factor VIII Deficiency

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Efanesoctocog alfa.
Who it may be relevant to
Registry conditions: Factor VIII Deficiency. Basic parameters: from 12 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Canada, Japan, Taiwan
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

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

Primary outcome measures

  • Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score [Time frame: From Baseline up to Week 52]
Secondary outcome measures (12)
  • Change from baseline in the distribution of joint HEAD-US synovitis score [Time frame: From Baseline up to Week 52]
  • Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened [Time frame: From Baseline up to Week 52]
  • Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: 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 [Time frame: From Baseline up to Week 52]
  • Change from baseline in average HEAD-US synovitis domain score across all index joints [Time frame: From Baseline up to Week 52]
  • Change from baseline in total/ domain scores of the HEAD-US [Time frame: From Baseline up to Week 26]
  • Change from baseline in total/domain scores of the HJHS [Time frame: From Baseline up to Week 26]
  • Change from baseline in total/domain scores of the HJHS [Time frame: From Baseline up to Week 52]
  • Change from baseline in total/ domain scores of magnetic resonance imaging (MRI) [Time frame: From Baseline up to Week 52]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 4 centers
  • 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
Japan · 4 centers
  • Investigational Site Number : 3920003 — Kawasaki
  • Investigational Site Number : 3920002 — Hiroshima
  • Investigational Site Number : 3920001 — Saitama
  • Investigational Site Number : 3920004 — Tokyo
Taiwan · 3 centers
  • Investigational Site Number : 1580003 — Kaohsiung City
  • Investigational Site Number : 1580001 — Taichung
  • Investigational Site Number : 1580002 — Taipei
Canada · 2 centers
  • Investigational Site Number : 1240003 — Hamilton
  • Investigational Site Number : 1240004 — Hamilton

Identifiers

NCT: NCT06941870 · LPS18168 · U1111-1311-1701

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗