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Tests treatment safety and results for Fabry Disease
Official title Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
ClinicalTrials.gov ID: NCT06207552
What this study is testing
What is BBM-F101 injection?
BBM-F101 injection is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fabry disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a single-arm, open label, single-dose clinical study to evaluate the safety, tolerability and efficacy of BBM-F101 injection in the pediatric Fabry disease participants up to 52 weeks after infusion, and the long-term safety and efficacy of BBM-F101 injection up to 5 years after infusion. BBM-F101 injection is an adeno-associated virus (AAV) gene therapy product for the treatment of pediatric Fabry disease.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 7 to 18
You may be able to join if
- The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written informed...
- Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
- Males or females aged ≥7 years and \<18 years old;
- Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
- Participants had at least one of the clinical manifestations for Fabry disease;
You likely can't join if
- 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...
- Have potential liver diseases;
- Heart failure and severe arrhythmias;
- Severe allergic reactions for enzyme replacement drugs or other medications;
- Acute/chronic infections;
- End-stage renal disease;
See the full eligibility criteria
- 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...
- Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
- Males or females aged ≥7 years and \<18 years old;
- Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
- Participants had at least one of the clinical manifestations for Fabry disease;
- Acceptable capsid antibody titers;
- Acceptable anti α-Gal A antibody titers;
- Acceptable laboratory values;
- Participant's legal guardian and participant with good cooperation and compliance;
- Use of reliable contraception methods during the study for adolescence.
- 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;
- Have potential liver diseases;
- Heart failure and severe arrhythmias;
- Severe allergic reactions for enzyme replacement drugs or other medications;
- Acute/chronic infections;
- End-stage renal disease;
- Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period;
- Have received gene therapy or used other investigational drugs within four weeks prior to dosing;
- Other conditions that make the participant not eligible for the study according to the investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 7 years to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.