Gene therapy for treating pediatric Fabry disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the safety, tolerability, and efficacy of a single-dose gene therapy drug, BBM-F101, in children diagnosed with Fabry disease. It is a single-arm, open-label study that will monitor participants for up to 52 weeks post-infusion, with long-term follow-up extending to five years. The study aims to assess how well the gene therapy works in improving symptoms and overall health in pediatric patients with this rare genetic condition.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written informed consent; If the participant is \>= 8 years old, the participant must also agree to participate in the study and sign a written informed consent;
2. Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
3. Males or females aged ≥7 years and \<18 years old;
4. Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
5. Participants had at least one of the clinical manifestations for Fabry disease;
6. Acceptable capsid antibody titers;
7. Acceptable anti α-Gal A antibody titers;
8. Acceptable laboratory values;
9. Participant's legal guardian and participant with good cooperation and compliance;
10. Use of reliable contraception methods during the study for adolescence.

Exclusion Criteria:

1. Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA), positive for hepatitis C virus RNA (HCV-RNA), positive for HIV or syphilis;
2. Have potential liver diseases;
3. Heart failure and severe arrhythmias;
4. Severe allergic reactions for enzyme replacement drugs or other medications;
5. Acute/chronic infections;
6. End-stage renal disease;
7. Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period;
8. Have received gene therapy or used other investigational drugs within four weeks prior to dosing;
9. Other conditions that make the participant not eligible for the study according to the investigator.

Where it's running

• Children's Hospital of Fudan University
  Shanghai, China Recruiting

Study contacts

• Principal investigator: Hong Xu, MD,PhD — Children's Hospital of Fudan University
• Study coordinator: Qian Shen, MD,PhD
• Email: shenqian@shmu.edu.cn
• Phone: +86 13701923307

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Lucerastat in untreated adult men with Fabry disease shows how well it works and safety

  This study tests lucerastat to see if it can reduce Gb3 buildup and be safe in untreated adult men with Fabry disease.

  Recruiting Phase 3 Idorsia Pharmaceuticals Ltd. · Birmingham, Alabama and 15 other locations · NCT07778667

• Periodontal Health in Rare Kidney Conditions and Controls

  This study tests how common periodontitis is in people with rare kidney disorders and compares it to CKD patients and people without kidney disease.

  Recruiting Institutul Clinic Fundeni · Bucharest · NCT07575347

• Anderson-Fabry disease fitness improvement program

  This study tests whether a 12-week physiotherapy-led exercise program can improve function, fitness, strength, and quality of life in adults with Fabry disease.

  Recruiting General University Hospital, Prague · Prague · NCT07495410

• Using advanced heart MRI to improve diagnosis and risk prediction for rare cardiomyopathies

  This project tests whether advanced cardiac MRI techniques can help diagnose and predict future risk for people with rare heart muscle diseases like Danon, Fabry, cardiac amyloidosis, Noonan syndrome, and cardiac sarcoidosis.

  Recruiting Chinese Academy of Medical Sciences, Fuwai Hospital · Beijing · NCT07336394

• Quality of life for people 65+ with Fabry disease, treated or untreated

  This project will see if starting or continuing Fabry disease treatment changes quality of life over two and five years in people aged 65 and older.

  Recruiting Groupe Hospitalier Diaconesses Croix Saint-Simon · Paris, France · NCT07277361

• Agalsidase alfa for heart inflammation in Fabry disease measured by PET‑CMR

  This study will test whether agalsidase alfa reduces heart inflammation in people with Fabry disease by comparing PET‑CMR scans before and after 12 months of treatment.

  Recruiting Yonsei University · Seoul · NCT07235709

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.