Phase 1 Haemophilia Trial, Recruiting NCT05709288 Sponsor: Institute of Hematology & Blood Diseases Hospital, China Condition: Haemophilia
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Phase 1 Haemophilia Trial, Recruiting

NCT05709288
Recruiting Phase 1

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • You (or your guardian) must be able to understand the study's purpose and risks, and sign a consent form
  • You must be male, between 12 and 17 years old, and weigh at least 50 kg (about 110 lbs)
  • You must have a confirmed diagnosis of hemophilia B with very low levels of the clotting factor FIX (2% or below), shown by laboratory tests
  • You must have previously received at least 75 treatment sessions using FIX protein products (either from donated blood or lab-made)
  • Your blood must show only low levels of certain antibodies that could interfere with the gene therapy being studied (confirm with trial site)
  • You must have had a bleeding episode or needed a FIX treatment within the 12 weeks before screening
  • You must have never had a serious allergic reaction to FIX products or IV immunoglobulin
  • You must have no detectable antibodies that block FIX from working (called inhibitors), and no signs that FIX treatments have stopped working for you
  • Your blood test results must fall within acceptable ranges for red blood cells, platelets, liver function, and kidney function
  • If you are sexually active, you must agree to use reliable barrier contraception (such as condoms) for up to 52 weeks
  • You must be willing and able to attend scheduled visits and keep a study diary

Who may not be able to join:

  • You have active hepatitis B or hepatitis C, shown by blood tests
  • You are currently taking antiviral medicines for hepatitis B or C
  • You have a blood clotting disorder other than hemophilia B
  • You have taken certain immune-suppressing medications (other than steroids or approved alternatives) within the past 30 days
  • You have received a vaccine in the past 30 days, or have a vaccination planned during the 52-week study period
  • You have a significant liver condition such as portal hypertension, an enlarged spleen, or liver-related brain problems, or any other liver condition the doctor feels makes gene therapy unsafe
  • You have surgery planned within 52 weeks after receiving the gene therapy
  • You have a history of serious ongoing infection, or a high risk of infection that the study doctor considers too risky
  • You have taken part in a gene therapy study in the past 52 weeks, or any other clinical drug trial in the past 12 weeks
  • You have taken herbal products that may affect liver function within the past 4 weeks
  • You have a history of a life-threatening bleeding episode, such as bleeding in the brain
  • You have any other serious illness or condition that the study doctor believes makes participation unsafe

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 27 July 2026
Phase 1: approximately ~10% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Lei Zhang, MD, Insitute of haematology and blood diseases hospital, chinese academy of medical sciences

Phone: +862223909240

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Phase 1
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
Registry
ClinicalTrials.gov
Start date
23 March 2023
Est. completion
1 June 2035

Where this trial is recruiting

🇨🇳 China

Primary endpoints

The incidence of treatment related adverse events deemed related to BBM-H901 within 10 weeks after vector administration; The incidence of adverse events and serious adverse events within 52 weeks after BBM-H901 administration; Change from baseline aspartate amino transferase; Change from baseline alanine aminotransferase

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov