Haemophilia Trial, Recruiting NCT04728841 Sponsor: Institute of Hematology & Blood Diseases Hospital, China Condition: Haemophilia
Back to Haemophilia

Haemophilia Trial, Recruiting

NCT04728841
Recruiting Not Applicable

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 are able to understand what the study involves, including its risks, and are willing to sign a consent form
  • You are male and at least 18 years old
  • You have been diagnosed with hemophilia A with very low or absent Factor VIII activity (1% or less), confirmed by a lab test or past medical records
  • You have never had a serious allergic reaction (such as anaphylaxis) to a Factor VIII product
  • You have no detectable Factor VIII inhibitors (antibodies that block Factor VIII), confirmed by at least two lab tests taken at least one week apart, or you have a documented history of no inhibitors after extensive Factor VIII treatment
  • Your blood test results at screening meet specific healthy ranges for hemoglobin, platelets, liver function, and kidney function (confirm specific values with trial site)
  • You agree to use reliable barrier contraception (such as condoms) until the end of the 52-week observation period and until three semen samples test negative for the study's gene therapy material

Who may not be able to join:

  • You currently have active, clinically significant Hepatitis B or C (people who have naturally cleared the virus or successfully treated it may still be eligible — confirm with trial site)
  • You are currently taking antiviral medications for Hepatitis B or C
  • You have a history of long-term or ongoing infections or other chronic illnesses that could make participation unsafe
  • You have taken part in a gene therapy trial in the past 52 weeks, or any other experimental drug study in the past 30 days
  • You have a condition that means you cannot safely take steroid medications such as prednisone or prednisolone
  • You have a history of blood clots, including deep vein thrombosis, stroke (non-bleeding type), pulmonary embolism, heart attack, or arterial blockage
  • You have a known inherited or acquired condition that increases your risk of blood clots, including atrial fibrillation
  • You are planning to have major surgery within the year following the study treatment
  • You have a known allergy or sensitivity to the study's gene therapy carrier (vector)
  • You have any other serious medical condition that, in the investigator's opinion, makes participation unsafe or unsuitable
  • You are unable or unwilling to attend all required study visits and follow the study schedule
  • You have a bleeding disorder that is not related to hemophilia A

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 28 July 2026
This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Principal Investigator: Lei Zhang, MD, Chinese Academy of Medical Science and Blood Disease Hospital

Phone: +82223909240

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
Not Applicable
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
Registry
ClinicalTrials.gov
Start date
4 March 2021
Est. completion
31 July 2028

Where this trial is recruiting

🇨🇳 China

Primary endpoints

Incidence of treatment- related adverse events; Percentage of subjects in each dose group with newly occurred clinically significant abnormalities in physical examination compared to the baseline.; Changes of Weighted Mean of vital signs (systolic blood pressure [SBP] and diastolic blood pressure [DBP], pulse rate, temperature, respiratory rate) from baseline at each assessment time point for each dose group; Changes of Mean Alkaline Phosphatase (ALP), Alanine Amino Transferase (ALT), Aspartate Amio Transferase (AST) from baseline at each assessment time point for each dose group; Immune respo...

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: 28 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