Krabbe DiseaseNCT06308718
Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
An observational study of Krabbe Disease, run by Forge Biologics, Inc at 1 site in United States.
Trial summary
This is an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Krabbe Disease
- What is being tested?
- FBX-101
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Aug 2024to 11 Apr 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This is an observational long-term follow-up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101.
Who may be able to join
- Participants that have completed a prior clinical trial involving the administration of FBX-101.
- Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.
Who cannot join
- •Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of FBX-101 during this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109
About this study
Official title: A Long-term Follow-up Study to Evaluate Safety and Efficacy of Krabbe Patients From Gene Therapy Clinical Trials Involving the Administration of FBX-101 (AAVrh.10-hGALC)
This is an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101.
Read the detailed description
FBX-101-LTFU is a multicenter, non-interventional, Long-Term Follow-Up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101. Eligible participants will undergo clinical evaluations at prespecified intervals for at least 3 years from the last visit in the prior clinical trial (up to 5 years post-FBX-101 treatment). Overall safety and additional signs of efficacy will be collected with a series of laboratory tests, diagnostic tests, and performance surveys. Additionally, children participating in interventional trials that are terminated early will be transferred to this LTFU study and will complete any pending visits from the interventional trial before starting the clinical evaluations included in this protocol.
- Conditions
- Krabbe Disease
- Being tested
- FBX-101
- Keywords
- Lysosomal Storage Disorder, Globoid Cell Leukodystrophy, Leukodystrophy, GALC
- Medical subject headings
- Leukodystrophy, Globoid Cell, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FBX-101Biological
A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (hGALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein.
| Study group | Role | What they receive |
|---|---|---|
| Patients enrolled in the FBX-101-LTFU study | The participants will be followed for 36 months after they have concluded their participation in the interventional trial. They will complete 5 scheduled visits with assessments as specified in the schedule of assessments, to collect data for safety and additional signs of efficacy for FBX-101. Those patients enrolled from any other early terminated trial, will first complete pending evaluations from that trial. Receives: Biological: FBX-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long Term safety as assessed by incidence of Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) that are attributed to FBX-101
Measured: 36 months
Secondary outcomes (26)
Efficacy as assessed by change of gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Mullen Scales of Early Learning (MSEL)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Beery VMI Sixth Edition (VMI)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by Mullen Scales of Early Learning (MSEL)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by the Differential Ability Scale II (DAS-II)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of adaptive behaviour function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of language function measured longitudinally by Mullen Scales of Early Learning (MSEL).
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of language function measured longitudinally by Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of language function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of language function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome.
Measured: 36 months
Efficacy as assessed by change of quality of life measured longitudinally by the Pediatric Quality of Life (PedsQL) Generic Core Scales with Family Impact Module and Multidimensional Fatigue Scale
Measured: 36 months
Efficacy as assessed by change of developmental milestones such as language and motor skills as measured longitudinally by an specific Developmental Milestones questionnaire
Measured: 36 months
Efficacy as assessed by change of hearing function measured longitudinally by Auditory Brainstem Responses (ABRs)
Absolute values for Waves I, III and V in milliseconds will be recorded along interpretation and waveform morphology; and also Auditory Brainstem Responses (corrected).
Measured: 36 months
Efficacy as assessed by change of hearing function measured longitudinally by Behavior Audiometry (BAUD)
Measured: 36 months
Efficacy as assessed by change of peripheral nerve conduction velocity measured longitudinally by Nerve Conduction Velocity (NCV) assessments
Measured: 36 months
Efficacy as assessed by change of Fractional Anisotropy (FA) as measured longitudinally by brain Magnetic Resonance Imaging (MRI) with Diffusion Tensor Imaging (DTI)
Measured: 36 months
Efficacy as assessed by change of visual function as measured longitudinally by visual evoked potentials (VEP)
Measured: 36 months
Efficacy as assessed by change of visual function as measured longitudinally by visual acuity
Measured: 36 months
Efficacy as assessed by change of whole blood donor chimerism
Measured: 36 months
Efficacy as assessed by change of psychosine in whole blood and plasma
Measured: 36 months
Efficacy as assessed by change of Galactosylceramidase (GALC) levels in plasma and Cerebrospinal Fluid (CSF)
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2024
- Study start6 Aug 2024
- Primary completion10 Apr 2025
- Study completion11 Apr 2025
Why it stopped: The FBX-101 clinical program has been discontinued by Forge Biologics
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for Krabbe DiseaseStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Forge Biologics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06308718
- Sponsor's protocol ID
- FBX-101-LTFU
- Status last verified
- Aug 2025
- First submitted
- 6 Mar 2024
- Last updated
- 5 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Bascou N, DeRenzo A, Poe MD, Escolar ML. A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life. Orphanet J Rare Dis. 2018 Aug 9;13(1):126. doi: 10.1186/s13023-018-0872-9.
PubMed 30089515Beltran-Quintero ML, Bascou NA, Poe MD, Wenger DA, Saavedra-Matiz CA, Nichols MJ, Escolar ML. Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months. Orphanet J Rare Dis. 2019 Feb 18;14(1):46. doi: 10.1186/s13023-019-1018-4.
PubMed 30777126Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, Wenger DA, Pietryga D, Wall D, Champagne M, Morse R, Krivit W, Kurtzberg J. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005 May 19;352(20):2069-81. doi: 10.1056/NEJMoa042604.
PubMed 15901860Yoon IC, Bascou NA, Poe MD, Szabolcs P, Escolar ML. Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood. 2021 Apr 1;137(13):1719-1730. doi: 10.1182/blood.2020005477.
PubMed 33150395Wright MD, Poe MD, DeRenzo A, Haldal S, Escolar ML. Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study. Neurology. 2017 Sep 26;89(13):1365-1372. doi: 10.1212/WNL.0000000000004418. Epub 2017 Aug 30.
PubMed 28855403Gupta A, Poe MD, Styner MA, Panigrahy A, Escolar ML. Regional differences in fiber tractography predict neurodevelopmental outcomes in neonates with infantile Krabbe disease. Neuroimage Clin. 2014 Sep 26;7:792-8. doi: 10.1016/j.nicl.2014.09.014. eCollection 2015.
PubMed 25844309Bradbury AM, Bagel J, Swain G, Miyadera K, Pesayco JP, Assenmacher CA, Brisson B, Hendricks I, Wang XH, Herbst Z, Pyne N, Odonnell P, Shelton GD, Gelb M, Hackett N, Szabolcs P, Vite CH, Escolar M. Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy. Mol Ther. 2024 Jan 3;32(1):44-58. doi: 10.1016/j.ymthe.2023.11.014. Epub 2023 Nov 11.
PubMed 37952085Siddiqi ZA, Sanders DB, Massey JM. Peripheral neuropathy in Krabbe disease: effect of hematopoietic stem cell transplantation. Neurology. 2006 Jul 25;67(2):268-72. doi: 10.1212/01.wnl.0000230156.01228.33.
PubMed 16864820Vander Lugt MT, Chen X, Escolar ML, Carella BA, Barnum JL, Windreich RM, Hill MJ, Poe M, Marsh RA, Stanczak H, Stenger EO, Szabolcs P. Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Adv. 2020 Jul 14;4(13):3041-3052. doi: 10.1182/bloodadvances.2020001940.
PubMed 32634238
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Follow-up Study to Evaluate Safety and Efficacy of Krabbe Patients From Gene Therapy Clinical Trials Involving the Administration of FBX-101 (AAVrh.10-hGALC)
- ClinicalTrials.gov ID
- NCT06308718
- Acronym / sponsor ID
- FBX-101-LTFU
- Conditions
- Krabbe Disease
- Keywords
- Lysosomal Storage Disorder, Globoid Cell Leukodystrophy, Leukodystrophy, GALC
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: The FBX-101 clinical program has been discontinued by Forge Biologics
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Forge Biologics, IncIndustry (pharma, biotech, devices)
- Study start
- 6 Aug 2024 (actual)
- Primary completion
- 10 Apr 2025 (actual)
- Study completion
- 11 Apr 2025 (actual)
- First posted
- 13 Mar 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 an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101.
Detailed description
FBX-101-LTFU is a multicenter, non-interventional, Long-Term Follow-Up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101. Eligible participants will undergo clinical evaluations at prespecified intervals for at least 3 years from the last visit in the prior clinical trial (up to 5 years post-FBX-101 treatment). Overall safety and additional signs of efficacy will be collected with a series of laboratory tests, diagnostic tests, and performance surveys. Additionally, children participating in interventional trials that are terminated early will be transferred to this LTFU study and will complete any pending visits from the interventional trial before starting the clinical evaluations included in this protocol.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients enrolled in the FBX-101-LTFU study | – | Biological: FBX-101The participants will be followed for 36 months after they have concluded their participation in the interventional trial. They will complete 5 scheduled visits with assessments as specified in the schedule of assessments, to collect data for safety and additional signs of efficacy for FBX-101. Those patients enrolled from any other early terminated trial, will first complete pending evaluations from that trial. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This is an observational long-term follow-up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101.
Key inclusion criteria (2 listed)
- Participants that have completed a prior clinical trial involving the administration of FBX-101.
- Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.
Key exclusion criteria (1 listed)
- •Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of FBX-101 during this study.
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 |
|---|---|---|
| FBX-101A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (hGALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein. | Biological | Patients enrolled in the FBX-101-LTFU study |
Primary outcome
Long Term safety as assessed by incidence of Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) that are attributed to FBX-101
Time frame: 36 months
26 secondary outcomes
Efficacy as assessed by change of gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2)
Time frame: 36 months
Efficacy as assessed by change of gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)
Time frame: 36 months
Efficacy as assessed by change of gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88)
Time frame: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Mullen Scales of Early Learning (MSEL)
Time frame: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Beery VMI Sixth Edition (VMI)
Time frame: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
Time frame: 36 months
Efficacy as assessed by change of fine motor function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3)
Time frame: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by Mullen Scales of Early Learning (MSEL)
Time frame: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by the Differential Ability Scale II (DAS-II)
Time frame: 36 months
Efficacy as assessed by change of cognitive function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
Time frame: 36 months
Efficacy as assessed by change of adaptive behaviour function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
Time frame: 36 months
Efficacy as assessed by change of language function measured longitudinally by Mullen Scales of Early Learning (MSEL).
Time frame: 36 months
Efficacy as assessed by change of language function measured longitudinally by Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5)
Time frame: 36 months
Efficacy as assessed by change of language function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
Time frame: 36 months
Efficacy as assessed by change of language function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
Time frame: 36 months
Efficacy as assessed by change of quality of life measured longitudinally by the Pediatric Quality of Life (PedsQL) Generic Core Scales with Family Impact Module and Multidimensional Fatigue Scale
Time frame: 36 months
Efficacy as assessed by change of developmental milestones such as language and motor skills as measured longitudinally by an specific Developmental Milestones questionnaire
Time frame: 36 months
Efficacy as assessed by change of hearing function measured longitudinally by Auditory Brainstem Responses (ABRs)
Time frame: 36 months
Efficacy as assessed by change of hearing function measured longitudinally by Behavior Audiometry (BAUD)
Time frame: 36 months
Efficacy as assessed by change of peripheral nerve conduction velocity measured longitudinally by Nerve Conduction Velocity (NCV) assessments
Time frame: 36 months
Efficacy as assessed by change of Fractional Anisotropy (FA) as measured longitudinally by brain Magnetic Resonance Imaging (MRI) with Diffusion Tensor Imaging (DTI)
Time frame: 36 months
Efficacy as assessed by change of visual function as measured longitudinally by visual evoked potentials (VEP)
Time frame: 36 months
Efficacy as assessed by change of visual function as measured longitudinally by visual acuity
Time frame: 36 months
Efficacy as assessed by change of whole blood donor chimerism
Time frame: 36 months
Efficacy as assessed by change of psychosine in whole blood and plasma
Time frame: 36 months
Efficacy as assessed by change of Galactosylceramidase (GALC) levels in plasma and Cerebrospinal Fluid (CSF)
Time frame: 36 months
Results and safety
The study was stopped early (reason given: The FBX-101 clinical program has been discontinued by Forge Biologics). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2025.
- Background: PubMed 30089515Bascou N, DeRenzo A, Poe MD, Escolar ML. A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life. Orphanet J Rare Dis. 2018 Aug 9;13(1):126. doi: 10.1186/s13023-018-0872-9.
- Background: PubMed 30777126Beltran-Quintero ML, Bascou NA, Poe MD, Wenger DA, Saavedra-Matiz CA, Nichols MJ, Escolar ML. Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months. Orphanet J Rare Dis. 2019 Feb 18;14(1):46. doi: 10.1186/s13023-019-1018-4.
- Background: PubMed 15901860Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, Wenger DA, Pietryga D, Wall D, Champagne M, Morse R, Krivit W, Kurtzberg J. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005 May 19;352(20):2069-81. doi: 10.1056/NEJMoa042604.
- Background: PubMed 33150395Yoon IC, Bascou NA, Poe MD, Szabolcs P, Escolar ML. Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood. 2021 Apr 1;137(13):1719-1730. doi: 10.1182/blood.2020005477.
- Background: PubMed 28855403Wright MD, Poe MD, DeRenzo A, Haldal S, Escolar ML. Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study. Neurology. 2017 Sep 26;89(13):1365-1372. doi: 10.1212/WNL.0000000000004418. Epub 2017 Aug 30.
- Background: PubMed 25844309Gupta A, Poe MD, Styner MA, Panigrahy A, Escolar ML. Regional differences in fiber tractography predict neurodevelopmental outcomes in neonates with infantile Krabbe disease. Neuroimage Clin. 2014 Sep 26;7:792-8. doi: 10.1016/j.nicl.2014.09.014. eCollection 2015.
- Background: PubMed 37952085Bradbury AM, Bagel J, Swain G, Miyadera K, Pesayco JP, Assenmacher CA, Brisson B, Hendricks I, Wang XH, Herbst Z, Pyne N, Odonnell P, Shelton GD, Gelb M, Hackett N, Szabolcs P, Vite CH, Escolar M. Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy. Mol Ther. 2024 Jan 3;32(1):44-58. doi: 10.1016/j.ymthe.2023.11.014. Epub 2023 Nov 11.
- Background: PubMed 16864820Siddiqi ZA, Sanders DB, Massey JM. Peripheral neuropathy in Krabbe disease: effect of hematopoietic stem cell transplantation. Neurology. 2006 Jul 25;67(2):268-72. doi: 10.1212/01.wnl.0000230156.01228.33.
- Background: PubMed 32634238Vander Lugt MT, Chen X, Escolar ML, Carella BA, Barnum JL, Windreich RM, Hill MJ, Poe M, Marsh RA, Stanczak H, Stenger EO, Szabolcs P. Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Adv. 2020 Jul 14;4(13):3041-3052. doi: 10.1182/bloodadvances.2020001940.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Krabbe Disease, run by Forge Biologics, Inc at 1 site in United States.
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 2 inclusion and 1 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: stopped early, as of the sponsor's last update on 5 Sep 2025. 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 United States. 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.
Are results available for this study?
The study was stopped early (reason given: The FBX-101 clinical program has been discontinued by Forge Biologics). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long Term safety as assessed by incidence of Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) that are attributed to FBX-101 (time frame: 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06308718, which also lists 9 linked publications.
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 5 Sep 2025.