BeCoMe-9: A Phase 1/2 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
New Gene Therapy Trial for Hemophilia B: BE-101
Plain English Summary
BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B is a Phase 2 clinical trial sponsored by Be Biopharma studying Hemophilia B, Hemophilia B, Moderately Severe or Severe. This trial tests a new gene therapy called BE-101 for adults with moderately severe to severe Hemophilia B. It is for adult males with Hemophilia B who are currently on standard treatment and have had at least 50 exposure days to Factor IX products. Participation involves receiving a single IV infusion of BE-101, which is made from the patient's own cells that have been genetically modified. Standard treatment for Hemophilia B involves regular infusions of Factor IX concentrate. The trial aims to enroll 24 participants.
Official Summary
The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately severe or severe Hemophilia B. Once infused, BE-101 is designed to engraft and continuously secrete FIX into the circulation to restore clinically meaningful levels of active FIX. BE-101 is an autologous (person's own cells) B Cell Medicine (BCM) which uses CRISPR/Cas9 gene editing to precisely insert human FIX gene into those cells.
Who Can Participate
Here is what you need to know about eligibility for this trial. Adult males aged 18 and older with moderately severe or severe Hemophilia B. Must have received at least 50 doses of Factor IX products and be currently on prophylaxis. Cannot have certain pre-existing conditions like B-cell cancers, immune deficiencies, recent blood clots, or active infections. Cannot have a history of inhibitors to Factor IX or allergic reactions to Factor IX products, or have previously received gene therapy. This trial is studying Hemophilia B, Hemophilia B, Moderately Severe or Severe, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.
What They're Measuring
The primary outcome measures will track any side effects and serious adverse events to ensure the treatment is safe for patients. The specific primary outcome measures are: Adverse events (AEs) and Serious Adverse Events (SAEs) (1 year post dose). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.
About This Phase
This trial is in Phase 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.
Why This Trial Matters
This trial aims to address the need for a long-lasting treatment for Hemophilia B by using a patient's own cells to continuously produce the missing clotting factor. Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets Hemophilia B, Hemophilia B, Moderately Severe or Severe, where improved treatment options are needed.
Investor Insight
This trial represents an early-stage investment in a novel gene therapy platform for rare bleeding disorders, with potential for significant market impact if successful. Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.
Is This Trial Right for Me?
Ask your doctor about the potential benefits and risks of BE-101 compared to your current treatment. Participation involves a procedure to collect your cells (leukapheresis) followed by a single IV infusion of the investigational therapy. You will need to attend regular follow-up appointments for about a year to monitor your safety and treatment effectiveness. This trial is currently recruiting participants. The trial is being conducted at 4 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: NON_RANDOMIZED
- Model: SEQUENTIAL
- Masking: NONE
- Enrollment: 24 participants
Interventions
- DRUG: BE-101 — IV Infusion of BE-101 dose from Dose Level 1
- DRUG: BE-101 — IV Infusion of BE-101 dose from Dose Level 2
- DRUG: BE-101 — IV Infusion of BE-101 dose from Dose Level 3
- DRUG: BE-101 — IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation
Primary Outcomes
- Adverse events (AEs) and Serious Adverse Events (SAEs) (1 year post dose)
Secondary Outcomes
- FIX Activity (Baseline to 1 year post dose)
- Annualized Bleed Rate (ABR) (1 year post dose)
- Bleeding Episodes (1 year post dose)
- Target Joints (1 year post dose)
- FIX Antigen Concentration (Baseline to 1 year post dose)
Full Eligibility Criteria
Inclusion Criteria: * Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency) * Received ≥50 exposure days to Factor IX products preceding enrollment. * Currently receiving prophylaxis treatment * Adequate organ function and clinical labs * Able to tolerate study procedures including leukapheresis. Exclusion Criteria: * Pre-existing or history of specific diseases * B-Cell malignancy, EBV lymphoproliferative disease * Primary immunodeficiency disease or disorder (PIDD) or systemic immuno-suppression * Arterial and/or venous thromboembolic events within 2 years prior to dosing * History of anaphylaxis or nephrotic syndrome * Active infection (HIV, Hep B or C) * History of inhibitor to FIX or inhibitor * History of an allergic reaction or anaphylaxis to FIX products * Planned surgical procedure within 6 months from BE-101 administration * Previously dosed with gene therapy * Participated in an interventional study and/or received an interventional study drug within 30 days or five-half-lives (whichever is longer) of consent into BeCoMe-9 and for the duration of the study * Planned participation in clinical trial within one year after BE-101 * Administration of an investigational agent or vaccine within 28 days of leukapheresis and dosing of BE-101 Other protocol-defined inclusion/exclusion criteria may apply.
Trial Locations
- University of California, Davis, Davis, California, United States
- University of Michigan, Ann Arbor, Michigan, United States
- University of Minnesota, Minneapolis, Minnesota, United States
- Washington Center for Bleeding Disorders, Seattle, Washington, United States
Frequently Asked Questions
What is clinical trial NCT06611436?
NCT06611436 is a Phase 2 INTERVENTIONAL study titled "BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B." It is currently recruiting and is sponsored by Be Biopharma. The trial targets enrollment of 24 participants.
What conditions does NCT06611436 study?
This trial investigates treatments for Hemophilia B, Hemophilia B, Moderately Severe or Severe. The primary condition under study is Hemophilia B.
What treatments are being tested in NCT06611436?
The interventions being studied include: BE-101 (DRUG), BE-101 (DRUG), BE-101 (DRUG), BE-101 (DRUG). IV Infusion of BE-101 dose from Dose Level 1
What does Phase 2 mean for NCT06611436?
Phase 2 trials test whether the treatment works for the intended condition. They involve 100-300 patients and continue to evaluate safety while measuring effectiveness.
What is the current status of NCT06611436?
This trial is currently "Recruiting." It started on 2024-12-04. The estimated completion date is 2027-07.
Who is sponsoring NCT06611436?
NCT06611436 is sponsored by Be Biopharma. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT06611436?
The trial aims to enroll 24 participants. The trial is currently recruiting and accepting new participants.
How is NCT06611436 designed?
This is a interventional study, uses non_randomized allocation, follows a sequential design, employs none masking.
What are the primary outcomes being measured in NCT06611436?
The primary outcome measures are: Adverse events (AEs) and Serious Adverse Events (SAEs) (1 year post dose). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT06611436 being conducted?
This trial is being conducted at 4 sites, including Davis, California; Ann Arbor, Michigan; Minneapolis, Minnesota; Seattle, Washington (United States).
Where can I find official information about NCT06611436?
The official record for NCT06611436 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT06611436. This government database provides the most up-to-date and detailed information about the trial.
What is NCT06611436 testing in simple terms?
This trial tests a new gene therapy called BE-101 for adults with moderately severe to severe Hemophilia B. It is for adult males with Hemophilia B who are currently on standard treatment and have had at least 50 exposure days to Factor IX products.
Why is this trial significant?
This trial aims to address the need for a long-lasting treatment for Hemophilia B by using a patient's own cells to continuously produce the missing clotting factor.
What are the potential risks of participating in NCT06611436?
The most common risks are related to the cell collection procedure and the infusion itself. Potential side effects may include infusion reactions, temporary changes in blood counts, or the development of antibodies. As this is a gene therapy, long-term effects are still being studied. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT06611436?
Ask your doctor about the potential benefits and risks of BE-101 compared to your current treatment. Participation involves a procedure to collect your cells (leukapheresis) followed by a single IV infusion of the investigational therapy. You will need to attend regular follow-up appointments for about a year to monitor your safety and treatment effectiveness. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT06611436 signal from an investment perspective?
This trial represents an early-stage investment in a novel gene therapy platform for rare bleeding disorders, with potential for significant market impact if successful. This is a Phase 2 trial, which is focused on confirming efficacy before larger pivotal studies.
What happens if the treatment in this trial doesn't work?
Participation involves receiving a single IV infusion of BE-101, which is made from the patient's own cells that have been genetically modified. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.
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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.