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 trialA clinical trial is research designed to understand the safety and efficacy of a drug, biologic or device. There are 4 phases to most clinical trials from Phase 1 that seeks to answer safety concern to take all the necessary steps voiced by the National Institute of Health before 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 work with your medical team to provide the best care to your loved one diagnosed with Krabbe disease.
Note: Information is listed chronologically with the most recently approved clinical trial. We encourage you to review all the information on this page with your medical provider.
Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCTHematopoietic stem cell transplantation (HSCT), a type of transplantation using multipotent hematopoietic stem cells typically derived from bone marrow, peripheral blood, or that from umbilical cord b (REKLAIM)
A Phase I/II FDA-approved clinical trial for Krabbe disease. 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 therapyA type of therapy that offers hope and promise for a cure for many genetic disorders. A working copy of the gene replaces the non-working copy of the gene. Gene therapy is at the forefront of many). This clinical trial is referenced as ReKlaim and/or FBX-101.
ClinicalTrials.gov Identifier: NCT05739643
Study Start Date: September 2021 (Recruiting)
Eligibility Criteria: For all the details, please review the content here on FBX-101, provided by clinicaltrials.gov.
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 patients 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 transplantTypically this refers to a bone marrow transplant with the goal of replacing non-working cells with healthy working cells. Bone marrow is a rich source of stem cells that have the unique ability to.
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.
Disclaimers:
- Choosing to participate in a clinical trial is a personal decision and one that should be discussed thoroughly with your specific care team.
- 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.