Fabry DiseaseEarly Phase 1NCT06207552
Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
A Early Phase 1 study testing BBM-F101 injection in people with Fabry Disease, run by Children's Hospital of Fudan University at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 7 to 18 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- BBM-F101 injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 20 Feb 2024to Jun 2029
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 7 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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;
- 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;
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- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Children's Hospital of Fudan University
Shanghai
Qian Shen, MD,PhD, +86 13701923307, shenqian@shmu.edu.cn
About this study
Official title: A Single-arm, Open Label, Single-dose Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-F101 Injection in the Treatment of Pediatric Fabry Disease
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.
- Conditions
- Fabry Disease
- Being tested
- BBM-F101 injection
- Keywords
- Fabry
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BBM-F101 injectionGenetic
The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion.
| Study group | Role | What they receive |
|---|---|---|
| Arm of BBM-F101 injection | Experimental | The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion. Receives: Genetic: BBM-F101 injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose limited toxicity
The incidence of dose limited toxicity (DLT) events as determined by the safety review committee (SRC) within DLT observation period following the BBM-F101 injection
Measured: 12 weeks
Incidence of adverse events and serious adverse events
The incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following the BBM-F101 injection
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2024
- Study start20 Feb 2024
- Primary completionJun 2025 (estimated)
- Study completionJun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by Children's Hospital of Fudan UniversityThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Hong Xu, MD,PhD, Principal investigator, Children's Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06207552
- Sponsor's protocol ID
- BBM017-IIT1001
- Status last verified
- Jul 2024
- First submitted
- 18 Dec 2023
- Last updated
- 9 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Open Label, Single-dose Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-F101 Injection in the Treatment of Pediatric Fabry Disease
- ClinicalTrials.gov ID
- NCT06207552
- Acronym / sponsor ID
- BBM017-IIT1001
- Conditions
- Fabry Disease
- Keywords
- Fabry
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Study start
- 20 Feb 2024 (actual)
- Primary completion
- Jun 2025 (estimated)
- Study completion
- Jun 2029 (estimated)
- First posted
- 17 Jan 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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm of BBM-F101 injection | Experimental | Genetic: BBM-F101 injectionThe dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion. |
Who may qualify
- Ages
- 7 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- 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;
- 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;
Key exclusion criteria (9 listed)
- 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;
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 |
|---|---|---|
| BBM-F101 injectionThe dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion. | Genetic | Arm of BBM-F101 injection |
Primary outcome
Incidence of dose limited toxicity
Time frame: 12 weeks
The incidence of dose limited toxicity (DLT) events as determined by the safety review committee (SRC) within DLT observation period following the BBM-F101 injection
Incidence of adverse events and serious adverse events
Time frame: 52 weeks
The incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following the BBM-F101 injection
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2024.
- 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.
Hong Xu, MD,PhD principal investigator
Children's Hospital of Fudan University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing BBM-F101 injection in people with Fabry Disease, run by Children's Hospital of Fudan University at 1 site in China.
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 10 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 9 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Qian Shen, MD,PhD (+86 13701923307, shenqian@shmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of dose limited toxicity (time frame: 12 weeks). The study lists 2 primary outcomes in all. 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 9 Jul 2024; AzTrials last refreshed its copy on 8 Nov 2026.