Hemophilia BPhase 1/2NCT06611436
BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
A Phase 1/2 study testing BE-101 in people with Hemophilia B and Hemophilia B, Moderately Severe or Severe, run by Be Biopharma at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia B, Hemophilia B, Moderately Severe or Severe
- What is being tested?
- BE-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 5 sitesUnited States
- Study dates
- 4 Dec 2024to 29 May 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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)
Show all 14Show fewer
- 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 and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
University of Michigan
Ann Arbor, Michigan, 48109
University of California, Davis
Davis, California, 95616
University of Minnesota
Minneapolis, Minnesota, 55455
Washington Center for Bleeding Disorders
Seattle, Washington, 98101
Georgetown University
Washington D.C., District of Columbia, 20057
About this study
Official title: 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
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.
Read the detailed description
The study includes 2 distinct parts: Part 1 and Part 2. In Part 1, an ascending-dose design will be utilized to enable evaluation of increasing doses in a stepwise manner. The objective for this dose escalation is to identify the dose of BE-101 required to achieve desired FIX activity 28 days after infusion. Upon identification of a safe and efficacious dose in Part 1, an expansion phase (Part 2) will initiate. The initial cohort in the Part 2 expansion (Part 2a) phase will include up to 6 adult participants to further characterize the safety and activity of BE-101 at the selected dose. Additional cohorts for adolescents and redosing for participants in Part 1 of the study will occur following data availability of Part 1.
Up to 24 participants will be enrolled across Part 1 (up to 18) and Part 2a (up to 6). Consented participants will complete a screening period to assess eligibility and upon enrollment will undergo leukapheresis collection to support BE-101 manufacturing. Following administration, participants will be monitored for safety and clinical activity. The total duration of study participation is approximately 52 weeks post IV administration of BE-101.
- Being tested
- BE-101
- Keywords
- Hemophilia B, Dose Escalation
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BE-101Drug
IV Infusion of BE-101 dose from Dose Level 1
BE-101Drug
IV Infusion of BE-101 dose from Dose Level 2
BE-101Drug
IV Infusion of BE-101 dose from Dose Level 3
BE-101Drug
IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation
| Study group | Role | What they receive |
|---|---|---|
| Part 1 - Dose Escalation, Dose Level 1 | Experimental | Not described Receives: Drug: BE-101 |
| Part 1 - Dose Escalation, Dose Level 2 | Experimental | Not described Receives: Drug: BE-101 |
| Part 1 - Dose Escalation, Dose Level 3 | Experimental | Not described Receives: Drug: BE-101 |
| Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation | Experimental | Not described Receives: Drug: BE-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events (AEs) and Serious Adverse Events (SAEs)
Incidence of AEs and SAEs
Measured: 1 year post dose
Secondary outcomes (8)
FIX Activity
Change From Baseline in FIX Activity
Measured: Baseline to 1 year post dose
Annualized Bleed Rate (ABR)
ABR (spontaneous and traumatic) for all bleeds. Annualized bleed rates overall and by severity will be presented.
Measured: 1 year post dose
Bleeding Episodes
Number of bleeding episodes (total, spontaneous, and traumatic). Bleeding episodes will be summarized by subject and overall, and will include number, duration, and severity.
Measured: 1 year post dose
Target Joints
Number of target joints. A target joint is defined as a major joint (e.g. hip, elbow, wrist, shoulder, knee, ankle) into which repeated bleeding occurs (frequency of 3 or more bleeding episodes into the same joint in a consecutive 12 week period) and with symptoms of pre-existing target joint involvement (eg, synovitis, persistent swelling, effusion, limitation of range of motion).
Measured: 1 year post dose
FIX Antigen Concentration
Change From Baseline in FIX Antigen concentration
Measured: Baseline to 1 year post dose
Exogenous FIX Concentrate
Number of infusions of exogenous FIX concentrate
Measured: 1 year post dose
FIX Replacement Therapy
Total consumption of exogenous FIX replacement therapy (IU) after BE-101 administration
Measured: 1 year post dose
FIX Inhibitor
FIX inhibitor development post BE-101 administration
Measured: 1 year post dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2024
- Study start4 Dec 2024
- Primary completion29 May 2026
- Study completion29 May 2026
Why it stopped: The study was terminated due to a strategic business decision and not due to safety concerns
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Be Biopharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06611436
- Sponsor's protocol ID
- BE-101-01
- Status last verified
- Jun 2026
- First submitted
- 18 Sep 2024
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06611436
- Acronym / sponsor ID
- BE-101-01
- Keywords
- Hemophilia B, Dose Escalation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated due to a strategic business decision and not due to safety concerns
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Be BiopharmaIndustry (pharma, biotech, devices)
- Study start
- 4 Dec 2024 (actual)
- Primary completion
- 29 May 2026 (actual)
- Study completion
- 29 May 2026 (actual)
- First posted
- 25 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The study includes 2 distinct parts: Part 1 and Part 2. In Part 1, an ascending-dose design will be utilized to enable evaluation of increasing doses in a stepwise manner. The objective for this dose escalation is to identify the dose of BE-101 required to achieve desired FIX activity 28 days after infusion. Upon identification of a safe and efficacious dose in Part 1, an expansion phase (Part 2) will initiate. The initial cohort in the Part 2 expansion (Part 2a) phase will include up to 6 adult participants to further characterize the safety and activity of BE-101 at the selected dose. Additional cohorts for adolescents and redosing for participants in Part 1 of the study will occur following data availability of Part 1.
Up to 24 participants will be enrolled across Part 1 (up to 18) and Part 2a (up to 6). Consented participants will complete a screening period to assess eligibility and upon enrollment will undergo leukapheresis collection to support BE-101 manufacturing. Following administration, participants will be monitored for safety and clinical activity. The total duration of study participation is approximately 52 weeks post IV administration of BE-101.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part 1 - Dose Escalation, Dose Level 1 | Experimental | Drug: BE-101 |
| Part 1 - Dose Escalation, Dose Level 2 | Experimental | Drug: BE-101 |
| Part 1 - Dose Escalation, Dose Level 3 | Experimental | Drug: BE-101 |
| Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation | Experimental | Drug: BE-101 |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (14 listed)
- Pre-existing or history of specific diseases
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BE-101IV Infusion of BE-101 dose from Dose Level 1 | Drug | Part 1 - Dose Escalation, Dose Level 1 |
| BE-101IV Infusion of BE-101 dose from Dose Level 2 | Drug | Part 1 - Dose Escalation, Dose Level 2 |
| BE-101IV Infusion of BE-101 dose from Dose Level 3 | Drug | Part 1 - Dose Escalation, Dose Level 3 |
| BE-101IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation | Drug | Part 2 - Dose Expansion, Cohort 2a Adult Expansion, Optimal Dose Selected in Part 1 Dose Escalation |
Primary outcome
Adverse events (AEs) and Serious Adverse Events (SAEs)
Time frame: 1 year post dose
Incidence of AEs and SAEs
8 secondary outcomes
FIX Activity
Time frame: Baseline to 1 year post dose
Annualized Bleed Rate (ABR)
Time frame: 1 year post dose
Bleeding Episodes
Time frame: 1 year post dose
Target Joints
Time frame: 1 year post dose
FIX Antigen Concentration
Time frame: Baseline to 1 year post dose
Exogenous FIX Concentrate
Time frame: 1 year post dose
FIX Replacement Therapy
Time frame: 1 year post dose
FIX Inhibitor
Time frame: 1 year post dose
Results and safety
The study was stopped early (reason given: The study was terminated due to a strategic business decision and not due to safety concerns). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BE-101 in people with Hemophilia B and Hemophilia B, Moderately Severe or Severe, run by Be Biopharma at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The study was terminated due to a strategic business decision and not due to safety concerns). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (AEs) and Serious Adverse Events (SAEs) (time frame: 1 year post dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jun 2026.