Krabbe DiseasePhase 1/2REKLAIMNCT05739643
Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT
A Phase 1/2 study testing FBX-101 in people with Krabbe Disease, run by Forge Biologics, Inc at 3 sites in United States.
Trial summary
This is a non-blinded, non-randomized dose escalation study of intravenous FBX-101 in which subjects will receive a single infusion of an adeno-associated virus gene therapy product, after more than 21 days of the HSCT (UCBT preferred HSCT source). Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Krabbe Disease
- What is being tested?
- FBX-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 3 sitesUnited States
- Study dates
- 3 Feb 2023to Nov 2026
- Phase
- Phase 1/2
- 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Group Infantile Krabbe: Subjects who are going to be transplanted or have already been transplanted for asymptomatic infantile onset Krabbe disease with initial diagnosis based on:
- Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of infantile Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING:
- Psychosine levels predictive of infantile onset by Dried Blood Spot (DBS); OR
- Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR
- Two GALC mutations predictive to result in infantile onset phenotype.
- Group Late Infantile Krabbe: Subjects who are going to be transplanted or have already been transplanted for symptomatic late infantile onset Krabbe with initial diagnosis based on:
Show all 19Show fewer
- Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of late infantile Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING:
- Psychosine levels predictive of late infantile onset by DBS; OR
- Imaging or neurophysiological findings consistent with Krabbe disease (CSF, MRI, NCV, ABR); OR
- Two GALC mutations predictive to result in late infantile onset phenotype; OR
- Neurological/developmental exam findings consistent with late infantile Krabbe disease
- Participants must be considered candidates for HSCT or have received HSCT at least 21 days prior to dosing date
- For patients already transplanted and followed for more than 3 months chimerism should reflect at least 30% of myeloid cells from the donor by month 3 post-transplant, from 30 to 10% between 3 months and one year post-transplant or 10% by one year post-transplant.
- Participant must have adequate organ function at time of screening or evaluation as measured by:
- Ejection fraction of > 50% by echocardiogram or other appropriate study without evidence of pulmonary hypertension.
- Pulmonary evaluation testing demonstrating resting pulse oximeter > 95% on room air.
- Absence of active aspiration
- Participant's parents or legal guardian consent to participate in the study and provide informed consent according to IRB/IEC guidelines prior to any study procedures being performed
- Parent(s) and/or legal guardian able to comply with the clinical protocol
Who cannot join
- Immunoassay with total anti-AAV10 antibody titers of >1:100. This criterion will not apply to children screened before they have received HSCT or for children who sign the inform consent within 6 months from HSCT. In children who test positive to anti-AAV10 antibody with titers of >1:100 under this exception, the ISR regime proposed by the PI and approved/modified by the ISR committee may include immunosuppressive drugs that prevent the potential development of a secondary immune response to AAVrh10 after FBX-101 administration.
- History of prior treatment with a gene therapy product
- Motor function evaluated by age with PDMS-II by a study physical therapist:
- Inability to hold head for patients older than 5 months; b. Inability to sit independently for patients older than 12 months; c. Inability to walk with assistance for patients older than 24 months.
- In patients that sign the informed consent before HSCT or up to 90 days post-HSCT, abnormalities in white count, hemoglobin and platelets found from conditioning regime to Day -1 (the day before FBX-101 administration) will be evaluated by the PI (with referral to the DSMB if indicated). If abnormal, they will not be considered an exclusion criteria if the PI considers they are consistent with expected consequences of the HSCT (and related management) and upon confirmation they were not present before commencement of the conditioning regime.
- Grade 2 or higher abnormalities in LFTs, bilirubin, creatinine, white count, hemoglobin, platelets, PT/INR and PTT according to latest version of CTCAE
Show all 14Show fewer
- Presence of any neurocognitive deficit, motor deficit, or brain damage not attributable to Krabbe disease
- Signs of active infections or disease from cytomegalovirus, adenovirus, EBV, hepatitis B or C, and HIV or other viruses excluding rhinovirus from RVP and asymptomatic norovirus presence in stool. Patients showing HIV positive results will be excluded from the study.
- Active bacterial or fungal infection documented the preceding 7 days.
- Presence of any contraindication for MRI or lumbar puncture (LP)
- Use of any investigational product prior to study enrollment or current enrollment in another study that involves clinical interventions
- Immunizations with live viruses in the 30 days prior to immune suppression
- Active acute Graft Versus Host Disease (GvHD) Grade II or higher according to modified Glucksberg criteria (Przepiorka et al., 1995) or active, moderate or severe, chronic GvHD according to revised NIH criteria (Jagasia et al., 2015)
- Any other medical condition, serious intercurrent illness, other genetic condition or extenuating circumstance that, in the opinion of the PI, would preclude participation in the study
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109
Duke University Medical Center
Durham, North Carolina, 27705
Children's Hospital of Orange County (CHOC)
Orange, California, 92868
About this study
Official title: A Phase 1/2 Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM)
This is a non-blinded, non-randomized dose escalation study of intravenous FBX-101 in which subjects will receive a single infusion of an adeno-associated virus gene therapy product, after more than 21 days of the HSCT (UCBT preferred HSCT source). Data from previously transplanted patients with infantile and late infantile Krabbe disease will be used as a comparator group.
Read the detailed description
The FBX-101-REKLAIM study has been modified on Q4 2023 to allow a broader patient recruitment of infantile and late infantile Krabbe patients. The updated REKLAIM study merges the recruitment populations of the previous FBX-101-RESKUE clinical trial (NCT04693598) and the FBX-101-REKLAIM clinical trial (NCT05739643).
- Conditions
- Krabbe Disease
- Being tested
- FBX-101
- Keywords
- Leukodystrophy, Globoid Cell, Hereditary Central Nervous System Demyelinating Diseases, Brain Diseases, Metabolic, Inborn, Brain Diseases, Metabolic, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Sphingolipidoses, Lysosomal Storage Diseases, Nervous System, Leukoencephalopathies, Demyelinating Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Lipidoses, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Metabolic Diseases, Lipid Metabolism Disorders
- Medical subject headings
- Leukodystrophy, Globoid Cell, Hereditary Central Nervous System Demyelinating Diseases, Brain Diseases, Metabolic, Inborn, Brain Diseases, Metabolic, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Sphingolipidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FBX-101Biological
Also called AAVrh.10-hGALC
A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (GALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC) | Experimental | N=3 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the low dose Receives: Biological: FBX-101 |
| Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC) | Experimental | N=3-6 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the high dose Receives: Biological: FBX-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as assessed by incidence and severity of adverse events and serious adverse events that are attributed to FBX-101
Measured: 24 months
Secondary outcomes (1)
Efficacy as assessed by improvement of gross motor function as measured longitudinally by PDMS-2, BOT-3, or by GMFM-88, depending on the age, compared to patients receiving HSCT only
Measured: 12 months and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2023
- Study start3 Feb 2023
- Primary completionNov 2026 (estimated)
- Study completionNov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (12)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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Dose escalation study from a low dose to a high dose following safety review
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Forge Biologics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05739643
- Sponsor's protocol ID
- FBX-101-REKLAIM
- Acronym
- REKLAIM
- Status last verified
- Nov 2025
- First submitted
- 13 Feb 2023
- Last updated
- 20 Nov 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 15901860Escolar ML, West T, Dallavecchia A, Poe MD, LaPoint K. Clinical management of Krabbe disease. J Neurosci Res. 2016 Nov;94(11):1118-25. doi: 10.1002/jnr.23891.
PubMed 27638597Rafi MA, Luzi P, Wenger DA. Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease. Bioimpacts. 2020;10(2):105-115. doi: 10.34172/bi.2020.13. Epub 2020 Mar 24.
PubMed 32363154Yoon 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 25844309Escolar ML, Poe MD, Smith JK, Gilmore JH, Kurtzberg J, Lin W, Styner M. Diffusion tensor imaging detects abnormalities in the corticospinal tracts of neonates with infantile Krabbe disease. AJNR Am J Neuroradiol. 2009 May;30(5):1017-21. doi: 10.3174/ajnr.A1476. Epub 2009 Apr 22.
PubMed 19386732Bradbury 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 37952085Greco MR, Lopez MA, Beltran-Quintero ML, Tuc Bengur E, Poe MD, Escolar ML. Infantile Krabbe disease (0-12 months), progression, and recommended endpoints for clinical trials. Ann Clin Transl Neurol. 2024 Dec;11(12):3064-3080. doi: 10.1002/acn3.52114. Epub 2024 Nov 5.
PubMed 39499628Heller G, Bradbury AM, Sands MS, Bongarzone ER. Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases. Mol Ther. 2023 Jan 4;31(1):7-23. doi: 10.1016/j.ymthe.2022.09.017. Epub 2022 Oct 4.
PubMed 36196048
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM)
- ClinicalTrials.gov ID
- NCT05739643
- Acronym / sponsor ID
- REKLAIM / FBX-101-REKLAIM
- Conditions
- Krabbe Disease
- Keywords
- Leukodystrophy, Globoid Cell, Hereditary Central Nervous System Demyelinating Diseases, Brain Diseases, Metabolic, Inborn, Brain Diseases, Metabolic, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Sphingolipidoses
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Forge Biologics, IncIndustry (pharma, biotech, devices)
- Study start
- 3 Feb 2023 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Nov 2026 (estimated)
- First posted
- 22 Feb 2023 (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 non-blinded, non-randomized dose escalation study of intravenous FBX-101 in which subjects will receive a single infusion of an adeno-associated virus gene therapy product, after more than 21 days of the HSCT (UCBT preferred HSCT source). Data from previously transplanted patients with infantile and late infantile Krabbe disease will be used as a comparator group.
Detailed description
The FBX-101-REKLAIM study has been modified on Q4 2023 to allow a broader patient recruitment of infantile and late infantile Krabbe patients. The updated REKLAIM study merges the recruitment populations of the previous FBX-101-RESKUE clinical trial (NCT04693598) and the FBX-101-REKLAIM clinical trial (NCT05739643).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Dose escalation study from a low dose to a high dose following safety review
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC) | Experimental | Biological: FBX-101N=3 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the low dose |
| Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC) | Experimental | Biological: FBX-101N=3-6 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the high dose |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- Group Infantile Krabbe: Subjects who are going to be transplanted or have already been transplanted for asymptomatic infantile onset Krabbe disease with initial diagnosis based on:
- Group Late Infantile Krabbe: Subjects who are going to be transplanted or have already been transplanted for symptomatic late infantile onset Krabbe with initial diagnosis based on:
- Participants must be considered candidates for HSCT or have received HSCT at least 21 days prior to dosing date
- For patients already transplanted and followed for more than 3 months chimerism should reflect at least 30% of myeloid cells from the donor by month 3 post-transplant, from 30 to 10% between 3 months and one year post-transplant or 10% by one year post-transplant.
- Participant must have adequate organ function at time of screening or evaluation as measured by:
Key exclusion criteria (14 listed)
- Immunoassay with total anti-AAV10 antibody titers of >1:100. This criterion will not apply to children screened before they have received HSCT or for children who sign the inform consent within 6 months from HSCT. In children who test positive to anti-AAV10 antibody with titers of >1:100 under this exception, the ISR regime proposed by the PI and approved/modified by the ISR committee may include immunosuppressive drugs that prevent the potential development of a secondary immune response to AAVrh10 after FBX-101 administration.
- History of prior treatment with a gene therapy product
- Motor function evaluated by age with PDMS-II by a study physical therapist:
- In patients that sign the informed consent before HSCT or up to 90 days post-HSCT, abnormalities in white count, hemoglobin and platelets found from conditioning regime to Day -1 (the day before FBX-101 administration) will be evaluated by the PI (with referral to the DSMB if indicated). If abnormal, they will not be considered an exclusion criteria if the PI considers they are consistent with expected consequences of the HSCT (and related management) and upon confirmation they were not present before commencement of the conditioning regime.
- Grade 2 or higher abnormalities in LFTs, bilirubin, creatinine, white count, hemoglobin, platelets, PT/INR and PTT according to latest version of CTCAE
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-101Also: AAVrh.10-hGALCA replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (GALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein. | Biological | Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC), Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC) |
Primary outcome
Safety as assessed by incidence and severity of adverse events and serious adverse events that are attributed to FBX-101
Time frame: 24 months
1 secondary outcome
Efficacy as assessed by improvement of gross motor function as measured longitudinally by PDMS-2, BOT-3, or by GMFM-88, depending on the age, compared to patients receiving HSCT only
Time frame: 12 months and 24 months
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 20 Nov 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 27638597Escolar ML, West T, Dallavecchia A, Poe MD, LaPoint K. Clinical management of Krabbe disease. J Neurosci Res. 2016 Nov;94(11):1118-25. doi: 10.1002/jnr.23891.
- Background: PubMed 32363154Rafi MA, Luzi P, Wenger DA. Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease. Bioimpacts. 2020;10(2):105-115. doi: 10.34172/bi.2020.13. Epub 2020 Mar 24.
- 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 19386732Escolar ML, Poe MD, Smith JK, Gilmore JH, Kurtzberg J, Lin W, Styner M. Diffusion tensor imaging detects abnormalities in the corticospinal tracts of neonates with infantile Krabbe disease. AJNR Am J Neuroradiol. 2009 May;30(5):1017-21. doi: 10.3174/ajnr.A1476. Epub 2009 Apr 22.
- 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.
- Results publication: PubMed 39499628Greco MR, Lopez MA, Beltran-Quintero ML, Tuc Bengur E, Poe MD, Escolar ML. Infantile Krabbe disease (0-12 months), progression, and recommended endpoints for clinical trials. Ann Clin Transl Neurol. 2024 Dec;11(12):3064-3080. doi: 10.1002/acn3.52114. Epub 2024 Nov 5.
- Derived from this study: PubMed 36196048Heller G, Bradbury AM, Sands MS, Bongarzone ER. Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases. Mol Ther. 2023 Jan 4;31(1):7-23. doi: 10.1016/j.ymthe.2022.09.017. Epub 2022 Oct 4.
- 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.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FBX-101 in people with Krabbe Disease, run by Forge Biologics, Inc at 3 sites 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 19 inclusion and 14 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: under way, not looking for participants, as of the sponsor's last update on 20 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
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?
Safety as assessed by incidence and severity of adverse events and serious adverse events that are attributed to FBX-101 (time frame: 24 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 NCT05739643, which also lists 12 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 20 Nov 2025.