BridgeMD
A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.
EU CTIS ID: 2024-510738-42-00
What this study is testing
Demonstrate the non-inferiority of CSL222 (formerly AMT-061 [2 x 10^13 gc/kg]) during the 52 weeks following establishment of stable factor IX expression (months 6 to 18) post-treatment (CSL222) followup compared to standard of care continuous routine factor IX prophylaxis during the lead-in phase, as measured by the annualized bleeding rate (ABR).
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male
- Age ≥18 years
- Subjects with congenital hemophilia B with known severe or moderately severe factor IX deficiency (≤2% of normal circulating factor IX ) for which the subject is on continuous routine factor IX prophylaxis (continuous routine prophylaxis is defined as the intent of treating with an a priori defined frequency of infusions [e.g., twice weekly, once every two weeks, etc.] as documented in the medical records).
- >150 previous exposure days of treatment with factor IX protein
- Have been on stable prophylaxis for at least 2 months prior to screening
- Have demonstrated capability to independently, accurately and in a timely manner complete the diary during the lead-in phase as judged by the investigator
You likely can't join if
- History of factor IX inhibitors
- Known history of an allergic reaction or anaphylaxis to factor IX products
- Known uncontrolled allergic conditions or allergy/hypersensitivity to any component of the IMP excipients
- Known history of allergy to corticosteroids
- Known medical condition that would require chronic administration of steroids
- Previous gene therapy treatment
See the full eligibility criteria
- Male
- Age ≥18 years
- Subjects with congenital hemophilia B with known severe or moderately severe factor IX deficiency (≤2% of normal circulating factor IX ) for which the subject is on continuous routine factor IX prophylaxis (continuous routine prophylaxis is defined as the intent of treating with an a priori defined frequency of infusions [e.g., twice weekly, once every two weeks, etc.] as documented in the medical records).
- >150 previous exposure days of treatment with factor IX protein
- Have been on stable prophylaxis for at least 2 months prior to screening
- Have demonstrated capability to independently, accurately and in a timely manner complete the diary during the lead-in phase as judged by the investigator
- Acceptance to use a condom during sexual intercourse in the period from IMP administration until AAV5 has been cleared from semen, as evidenced by the central laboratory from negative analysis results for at least 3 consecutively collected semen samples (this criterion is applicable also for subjects who are surgically sterilized)
- Able to provide informed consent following receipt of verbal and written information about the trial.
- History of factor IX inhibitors
- Known history of an allergic reaction or anaphylaxis to factor IX products
- Known uncontrolled allergic conditions or allergy/hypersensitivity to any component of the IMP excipients
- Known history of allergy to corticosteroids
- Known medical condition that would require chronic administration of steroids
- Previous gene therapy treatment
- Receipt of an experimental agent within 60 days prior to screening
- Current participation or anticipated participation within one year after IMP administration in this trial in any other interventional clinical trial involving drugs or devices.
- Positive factor IX inhibitor test at screening and Visit L-Final (based on local laboratory results)
- Screening and Visit L-Final laboratory values (based on central laboratory results): a) ALT >2 times upper normal limit b) Aspartate aminotransferase (AST) >2 times upper normal limit c) Total bilirubin >2 times upper normal limit (except if this is caused by Gilbert disease) d) Alkaline phosphatase (ALP) >2 times upper normal limit e) Creatinine >2 times upper normal limit
- Positive human immunodeficiency virus (HIV) serological test at E7 screening and Visit LFinal, not controlled with anti-viral therapy asshown by CD4+ counts ≤ 200/μL (based on central laboratory results)
- Hepatitis B or C infection with the following criteria present at screening: i. Currently receiving antiviral therapy for this/these infection(s) and/or ii. Positive for any of the following (based on central laboratory results): • Hepatitis B surface antigen (HBsAg), except if in the opinion of the investigator this is due to a previous Hepatitis B vaccination rather thanactive Hepatitis B infection • Hepatitis B virus deoxyribonucleic acid (HBV DNA) • Hepatitis C virus ribonucleic acid (HCV RNA)
- Known coagulation disorder other than hemophilia B
- Thrombocytopenia, defined as a platelet count below 50 × 10^9/L, at screening and Visit L-Final (based on central laboratory results)
- Known severe infection or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, cardiovascular, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, alcoholism, drug dependency or any other psychological disorder valuated by the investigator to interfere with adherence to the protocol procedures or with the degree of tolerance to the IMP
- Known significant medical condition that may significantly impact the intended transduction of the vector and/or expression and activity of the protein, including but not limited to: • Disseminated intravascular coagulation • Accelerated fibrinolysis • Advanced liver fibrosis (suggestive of or equal to METAVIR Stage 3 disease; e.g. a FibroScan™ score of ≥9 kPa is considered equivalent)
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
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 - 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 male, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.