What side effects are associated with this type of intervention?

The most common treatments for Krabbe Disease include hematopoietic stem cell transplantation (HSCT) and gene therapy approaches like adeno-associated virus (AAV) gene therapy. For AAV gene therapy, side effects can include immune responses to the viral vector, which may cause inflammation or other immune-related issues. Additionally, there can be risks of insertional mutagenesis, where the integration of the viral vector into the host genome might disrupt normal gene function, potentially leading to malignancies. HSCT can have side effects such as graft-versus-host disease (GVHD), infections, and organ damage due to the conditioning regimen required before transplantation.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved gene therapies for Krabbe Disease. The current study involving FBX-101, an adeno-associated virus gene therapy, represents an investigational approach aimed at treating this condition. Traditional treatments have primarily focused on hematopoietic stem cell transplantation (HSCT), which can help slow disease progression if administered early. The FBX-101 trial is exploring the potential of gene therapy to provide a more targeted and effective treatment option for patients with Krabbe Disease.

What other types of research exist?

Promising alternatives to FBX-101 for Krabbe Disease patients include enzyme replacement therapies and other gene therapies. Specifically, therapies targeting the GALC gene using different viral vectors or CRISPR-based gene editing techniques are under investigation. Additionally, substrate reduction therapies and small molecule chaperones that enhance the function of residual enzyme activity are also being explored.

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Gene Therapy

Gene Therapy for Krabbe Disease (REKLAIM Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Forge Biologics, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a single dose of a virus carrying a healthy gene in patients with severe forms of Krabbe disease. The virus helps by delivering the healthy gene to the patient's cells. Gene therapy has shown promise in extending survival in previous studies.

Who is the study for?

This trial is for children with Krabbe Disease who've had a stem cell transplant at least 90 days before. They must have certain diagnostic criteria met, including specific test results or mutations, and sufficient engraftment of donor cells. Children can't join if they have recent infections, MRI or lumbar puncture contraindications, used investigational products recently, live virus immunizations within the last month, severe organ function issues, neurocognitive deficits not due to Krabbe disease, heart problems without pulmonary hypertension evidence or previous gene therapy treatments.

What is being tested?

The study tests FBX-101 in kids who previously received a stem cell transplant for Krabbe Disease. It's an escalating dose trial where participants get one infusion of this gene therapy product. The effects will be compared to data from untreated and transplanted patients with infantile and late infantile forms of the disease.

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions related to immune response against the viral vector (like fever), complications from underlying conditions exacerbated by treatment or typical risks associated with infusions such as discomfort at injection site.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2 - High Dose FBX-101 (aka AAVrh.10-GALC)Experimental Treatment1 Intervention

N=3-6 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the high dose

Group II: Cohort 1 - Low Dose FBX-101 (aka AAVrh.10-GALC)Experimental Treatment1 Intervention

N=3 patients with Infantile or Late Infantile Krabbe disease will receive a single infusion at the low dose

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Krabbe Disease include hematopoietic stem cell transplantation (HSCT) and gene therapy. HSCT aims to provide the patient with healthy donor cells that can produce the enzyme galactocerebrosidase (GALC), which is deficient in Krabbe Disease. Gene therapy, such as the adeno-associated virus (AAV) gene therapy studied in FBX-101, involves delivering a functional copy of the GALC gene directly to the patient's cells. This approach aims to correct the underlying genetic defect and restore normal enzyme levels. These treatments are crucial for Krabbe Disease patients because they target the root cause of the disease, potentially halting or reversing the progression of neurological damage.