What is the BEYOND-9 clinical research study?
If you live with hemophilia B, please know that you are not alone in your medical journey. Hemophilia B is a rare genetic disease that affects about 4 in every 100,000 males worldwide according to the World Federation of Hemophilia (WFH).
Currently, we are working on developing potential treatments for hemophilia B. BEYOND-9 is a phase 1/2A phase 1 clinical research study is a clinical research study investigating the overall safety and tolerability of an investigational treatment. In a phase 2 clinical research study, investigators assess the safety of the potential treatment and the best dose. Researchers also study how it works and possible side effects. clinical study that aims to see if an investigational CRISPR-based targeted gene insertion therapy may be effective and well-tolerated in males with hemophilia B. This treatment is called “investigational” as it has not yet been approved to treat hemophilia B.
Am I eligible?
What is the study treatment?
The study treatment is an investigational CRISPR/Cas9-based gene insertion therapy. It is being developed to insert a functional F9 (FIX) sequence into the DNA in liver cells, the cells that naturally produce the FIX protein. The insertion of the sequence into the DNA of liver cells is intended to prevent bleeding by restoring normal levels of functional FIX protein in the blood.
CRISPR stands for “clustered regularly interspaced short palindromic repeats.” Cas9 is a CRISPR-associated protein 9. CRISPR technology has two parts which work together for gene insertion: a guide RNA strand and a Cas9 enzyme. The guide RNA strand guides the Cas9 enzyme precisely to a matching location within the DNA sequence. Only then will the Cas9 enzyme create a small opening for the new gene to be inserted.
To learn more about gene therapy, please visit the What is gene therapy? section.
Once the study doctor confirms that you qualify, you will be assigned to receive a single dose of the study treatment via an IV (intravenous – into a vein) infusion.
What can I expect if I participate?
participation is 100% voluntary
approximately 2 years and 8 months
Study participation is 100% voluntary (your choice). You will receive information on what to expect, as well as your role and responsibilities if you join the BEYOND-9 study. You may leave the study at any time without it affecting your regular health care.
If you join the study, there are certain things you will be expected to do. This includes attending study visits and undergoing certain assessments and procedures. Your participation may last for up to approximately 2 years and 8 months. Participation may last longer (up to 4 years and 2 months) depending on time spent in the lead-in period.
There is an expectation to be followed-up for up to 15 years following receipt of the study treatment. This is so you can continue to be checked for safety as this is a new therapy and it is possible that some effects may not be seen until later. For this purpose, you will be asked to join a long-term follow-up study at the end of this study.
You will be given an e-diary to record bleeding events and FIX replacement therapy usage for the duration of the study. You may have the option to have certain visits take place at your home or another acceptable location if you are unable to access the study site. This option depends on several factors, such as your country’s local regulations. The study team will discuss all of this with you.
Study flow diagram
* Not required for participants who enroll following at least 6-months in a separate lead-in study (RO00-HEMB-2187)
^ Liver function tests monitored twice weekly during weeks 4-12
This study will have a lead-in period before you receive the study treatment, where you will continue to take your standard of care FIX prophylaxis and record your bleeding events for at least 6 months and up to 2 years. If you have participated in HONEY-B (R000-HEMB-2187), then you do not need to participate in the lead-in period. HONEY-B is a study evaluating the disease characteristics, including bleeding events, in people with hemophilia B that are receiving FIX prophylaxis.
Participants will be reimbursed for reasonable travel expenses to and from study visits, and medical care related to their participation, including the study treatment, will be provided at no cost. The study team will discuss all of this with you.
How can I take part?
If you wish to take part in BEYOND-9, you can fill in a short pre-screener questionnaire to see if you may be eligible.
Am I eligible?
What else should I know?
As with all medicines, there are possible risks when taking the investigational treatment. If you qualify and choose to participate, you will be provided with an Informed Consent Form that explains any possible risks and side effects. It is also possible that the study treatment may affect you in unknown ways. Your health and safety are our top priorities and will be closely monitored throughout your participation.
There is no guarantee that you will receive a medical benefit from participating in this study. Your condition may get better, stay the same, or may even get worse. You are free to withdraw from the study for any reason and at any time.
Find a study site near you
To find out more about BEYOND-9 and whether it may be right for you, please contact your closest study site – they will be more than happy to help.
English