Factor VIII Deficiency study: Efanesoctocog alfa
Official title Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy
ClinicalTrials.gov ID: NCT06941870
What this study is testing
What is Efanesoctocog alfa?
An investigational once‑weekly injectable medicine being studied for treating hemophilia A by reducing joint swelling.
Also referred to as BIVV001, ALTUVIIIO®.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Find out if a weekly dose of efanesoctocog alfa improves joint swelling over 12 months.
- Who it's for
- People with hemophilia A who have joint swelling.
- What's involved
- Participants receive a weekly dose of efanesoctocog alfa and have joint exams and tests.
- Time commitment
- About 12 months, with regular visits each week for treatment and periodic check‑ups.
- Phase 4: studies an already-approved treatment
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting...
- 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...
- Participant has at least 1 eligible\ joint without planned future major orthopedic procedures (example, arthroscopic synovectomy, radioisotopic or...
- Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2).
You likely can't join if
- 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 treatment study or exposed to other investigational drug(s) within 3 months prior to screening for this...
- Is currently in an institution because of regulatory or legal order (that is, is a prisoner or a patient who is legally institutionalized).
See the full eligibility 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...
- 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.
- Is a women of nonchildbearing potential (WONCBP)
- Is a 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)
- 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 treatment 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.
The study team makes the final eligibility decision.
Where it is enrolling
- Sacramento, California, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- Portland, Oregon, United States
- Seattle, Washington, United States
- Calgary, Alberta, Canada
- St. John's, Newfoundland and Labrador, Canada
- Hamilton, Ontario, Canada
- Kawasaki, Kanagawa, Japan
- Saitama, Japan
- Kaohsiung City, Taiwan
- Taichung, Taiwan
- Taipei, Taiwan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Sacramento, California, United States; Indianapolis, Indiana, United States; Iowa City, Iowa, United States; Portland, Oregon, United States; Seattle, Washington, United States; Calgary, Alberta, Canada and 7 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.