KrabbeConnect works to arm those impacted by Krabbe disease with valuable information to ensure families have all the information to enhance disease management all in one spot-HERE. Information is power yet can be overwhelming to understand and grasp. For this reason, it is imperative for families and patients considering participation in any clinical trial to take all the necessary steps voiced by the National Institute of Health prior to enrollment in a clinical trial. This will allow you and your loved ones the opportunity to fully understand the risks and benefits of your participation. We hope you find this page helpful as you and your medical team to provide the best care to your loved one diagnosed with Krabbe disease.
Note: Information listed chronologically with the most recently approved clinical trial. We encourage you to review all the information on this page with your medical provider.
Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects with Early Infantile Krabbe Disease (GALax-C)
Passage Bio has initiated a Phase I/II FDA approved clinical trial for Krabbe disease using gene therapy to deliver a functional copy of the GALC gene to the brain and peripheral tissues. This study will evaluate the safety, tolerability, and efficacy of this treatment by first evaluating two different doses in two different age groups, then confirming the optimal dose to be used for confirmation of safety and efficacy.
ClinicalTrials.gov Identifier: NCT04771416
Study Start Date: September 2021 (Recruiting)
Eligibility Criteria: For all the details, please review the content here on PBKR03, provided by clinicaltrials.gov.
To learn more about the GALax-C trial click here.
To learn more about the GALax-C trial click here.
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)
Dr. Joanne Kurtzberg and the Marcus Foundation initiated a Phase I FDA approved clinical trial for patient with metabolic diseases. Krabbe disease is one of many metabolic diseases. This clinical trial works to improve the long-term outcomes of those diagnosed with Krabbe disease by utilizing DUCOC-01 to expedite and improve engraftment of the hematopoietic stem cell transplant.
ClinicalTrials.gov Identifier: NCT01372228
Study Start Date: September 2014
Eligibility Criteria: For all the details, please review the content here on DUOC-01, provided by clinicaltrials.gov.
Gene Transfer Clinical Trial for Krabbe Disease (RESKUE)
Forge Biologics has initiated a Phase I/II FDA approved clinical trial for Krabbe disease called FBX-101. This clinical trial works to improve the long-term outcomes of those diagnosed with Krabbe disease by utilizing a combination therapy, HSCT and AAVrh10 (gene therapy).
ClinicalTrials.gov Identifier: NCT04693598
Study Start Date: September 2021 (Recruiting)
Eligibility Criteria: For all the details, please review the content here on FBX-101, provided by clinicaltrials.gov.
Clinical Trial Video:
Neurogene Clinical Trial for Krabbe disease
More information coming soon.
Neurogene commits to Krabbe disease. More information about clinical trials will be forthcoming as it becomes available.
Please take a moment to hear from Neurogene Founder and COO, Rachel McMin below:
Disclaimer:
1. Choosing to participate in a clinical trial is a personal decision and one that should be discussed thoroughly with your specific care team.
2. KrabbeConnect Clinical Trials webpage is strictly a resource to inform patients and caregivers of the current opportunities available. The information should never be interpreted as medical advice.