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Leukodystrophy, Globoid Cell clinical trials

View clinical trials related to Leukodystrophy, Globoid Cell.

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NCT ID: NCT06308718 Not yet recruiting - Krabbe Disease Clinical Trials

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Start date: June 2024
Phase:
Study type: Observational

This is an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101.

NCT ID: NCT05739643 Recruiting - Krabbe Disease Clinical Trials

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

REKLAIM
Start date: February 3, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

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.

NCT ID: NCT04771416 Suspended - Clinical trials for Leukodystrophy, Globoid Cell

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

GALax-C
Start date: February 24, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

PBKR03 is a gene therapy for Krabbe Disease (Globoid cell leukodystrophy) intended 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.

NCT ID: NCT04693598 Active, not recruiting - Krabbe Disease Clinical Trials

Gene Transfer Clinical Trial for Krabbe Disease

RESKUE
Start date: November 5, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

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.

NCT ID: NCT03655223 Enrolling by invitation - Diabetes Mellitus Clinical Trials

Early Check: Expanded Screening in Newborns

Start date: October 15, 2018
Phase:
Study type: Observational

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health conditions in addition to providing important data on the implementation of this model program. Early diagnosis may result in health and development benefits for the newborns. Infants who have newborn screening in North Carolina will be eligible to participate, equating to over 120,000 eligible infants a year. Over 95% of participants are expected to screen negative. Newborns who screen positive and their parents are invited to additional research activities and services. Parents can enroll eligible newborns on the Early Check electronic Research Portal. Screening tests are conducted on residual blood from existing newborn screening dried blood spots. Confirmatory testing is provided free-of-charge for infants who screen positive, and carrier testing is provided to mothers of infants with fragile X. Affected newborns have a physical and developmental evaluation. Their parents have genetic counseling and are invited to participate in surveys and interviews. Ongoing evaluation of the program includes additional parent interviews.

NCT ID: NCT03333200 Recruiting - Gaucher Disease Clinical Trials

Longitudinal Study of Neurodegenerative Disorders

Start date: January 11, 2012
Phase:
Study type: Observational

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

NCT ID: NCT03047369 Recruiting - Clinical trials for Adrenoleukodystrophy

The Myelin Disorders Biorepository Project

MDBP
Start date: December 8, 2016
Phase:
Study type: Observational [Patient Registry]

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

NCT ID: NCT02993796 Recruiting - Krabbe Disease Clinical Trials

Krabbe Disease Global Patient Registry

Start date: September 2014
Phase:
Study type: Observational [Patient Registry]

The purpose of this study is to develop a clinical database of individuals diagnosed with Krabbe disease in order to determine which symptoms herald the onset of clinical disease in the various phenotypes of Krabbe disease; to determine whether level of GALC enzyme activity, or a specific genetic mutation predict the clinical course; and to determine which neurodiagnostic tests predict onset and/or severity of the disease.

NCT ID: NCT02699190 Active, not recruiting - Clinical trials for Adrenoleukodystrophy

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Start date: January 6, 2017
Phase:
Study type: Observational

Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes. While recent work has demonstrated that whole genome sequencing (WGS), has the potential to dramatically increase diagnostic efficiency, significant questions remain around the impact on downstream clinical management approaches versus standard diagnostic approaches.

NCT ID: NCT02254863 Recruiting - Clinical trials for Mucopolysaccharidosis II

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

DUOC-01
Start date: September 2014
Phase: Phase 1
Study type: Interventional

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.