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3 clinical studies listed.
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Tundra lists 3 Aspartylglucosaminuria clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT02171104
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Gender: All
Ages: Any - 55 Years
Updated: 2026-11-03
1 state
NCT07903402
AGU Registry and NHS
The Aspartylglucosaminuria (AGU) Registry and Natural History Study (AGU Registry) aims to gain a more comprehensive understanding of Aspartylglucosaminuria (AGU) disease through an in-depth understanding of the disease progression in global patient population and burden of the disease on the family and caretakers.
Gender: All
Updated: 2026-10-29
1 state
NCT07530796
Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)
The goal of this clinical trial is to learn if the treatment is a safe, tolerable, and efficacious treatment for adults and children with Aspartylglucosaminuria (AGU).
Gender: All
Ages: 4 Years - 45 Years
Updated: 2026-10-27
1 state