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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04693598
Other study ID # FBX-101-RESKUE
Secondary ID
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date November 5, 2021
Est. completion date July 2024

Study information

Verified date June 2024
Source Forge Biologics, Inc
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cell transplantation for Krabbe disease, followed by a single infusion of an adeno-associated virus gene therapy product. Extensive natural history subjects will be used to compare as control group.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 6
Est. completion date July 2024
Est. primary completion date July 2024
Accepts healthy volunteers No
Gender All
Age group 1 Day to 12 Months
Eligibility Inclusion Criteria: 1. Diagnosis of infantile Krabbe disease, characterized by the following criteria outlined below: - Galactocerebrosidase (GALC) activity levels in leukocytes compatible with the diagnosis of Krabbe disease; AND AT LEAST ONE OF THE FOLLOWING: - Elevated psychosine levels predictive of infantile disease onset by 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. 2. Age at the time of screening: 1 day to 12 months 3. Participant has been deemed eligible for treatment with HSCT (standard of care) and a fully myeloablative reduced intensity/toxicity conditioning regimen (RIC/RTC) is/has been used 4. Participant's parents or legal guardian consents to participate in the study and provides informed consent according to IRB guidelines prior to any study procedures being performed 5. Parent(s) and/or legal guardian able to comply with the clinical protocol 6. Participant must have adequate organ function at time of screening as measured by: - Creatinine = 1.5x upper limit of age appropriate normal and creatinine clearance = 60 mL/min/1.73 m2 - Hepatic transaminases (ALT/AST) = 2x age related upper limit of normal - 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 - Coagulation tests within 110% of normal ranges for age. (PT/INR and PTT) Exclusion Criteria: 1. History of prior treatment with a gene therapy product 2. Presence of major congenital anomaly or any other condition that affects neurodevelopmental function 3. Presence of any neurocognitive deficit or brain damage not attributable to Krabbe disease 4. Active aspiration 5. Signs of active infection or disease from cytomegalovirus, adenovirus or other viruses 6. HIV positive 7. Uncontrolled and progressive bacterial or fungal infection 8. Presence of any contraindication for MRI 9. Use of any investigational product prior to study enrollment or current enrollment in another study that involves clinical interventions 10. Any other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the PI, would preclude participation in the study 11. Ongoing veno-occlusive disease (VOD) as determined by liver ultrasound (moderate ascites and static or retrograde portal vein flow) the day before FBX-101 infusion.

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
FBX-101
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.

Locations

Country Name City State
United States University of Michigan Hospitals - Michigan Medicine Ann Arbor Michigan

Sponsors (1)

Lead Sponsor Collaborator
Forge Biologics, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety as assessed by incidence and severity of adverse events and serious adverse events that are attributed to FBX-101. 24 months
Primary Safety as assessed by HSCT incident of engraftment. 24 months
Secondary Efficacy as assessed by improvement of probability to achieve independent sitting compared to untreated patients or those receiving HSCT only. 12 months and 24 months
Secondary Efficacy as assessed by improvement of gross motor function as measured by Peabody Developmental Motor Scale 2nd Edition (PDMS-2) above a functional age equivalent of 12 months compared to untreated patients or those receiving HSCT only 24 months
See also
  Status Clinical Trial Phase
Recruiting NCT02993796 - Krabbe Disease Global Patient Registry
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Terminated NCT00668564 - Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Phase 2
Not yet recruiting NCT06308718 - Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients
Active, not recruiting NCT02699190 - LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Completed NCT01938014 - Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Recruiting NCT03333200 - Longitudinal Study of Neurodegenerative Disorders
Recruiting NCT05739643 - Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT Phase 1/Phase 2
Recruiting NCT03047369 - The Myelin Disorders Biorepository Project
Completed NCT01043640 - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Phase 2
Withdrawn NCT01425489 - Biomarker for Krabbe Disease (BioKrabbe)
Active, not recruiting NCT00787865 - Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease