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Clinical Trial Search

Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.

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30 closest matches · ranked by relevance

Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors

Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Masonic Cancer Center, University of Minnesota Results Jul 2019 26.3% serious AE View details

Phase 2 N=1 JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease

Kohlmeier Degos Disease With Neurologic Involvement · Kohlmeier-Degos Disease
Primary: Number of Participants With Stable or no Lesions Developed Based on MRI Neurological Involvement of K-D Disease Following Ruloxitinib — 1; 0; 0 Participants
National Heart, Lung, and Blood Institute (NHLBI) Results Feb 2026 100.0% serious AE View details

Phase 4 N=10 Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

Infantile Neronal Ceroid Lipofuscinosis
Primary: Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells. — -0.3317 Average number of GRODs
Eunice Kennedy Shriver National Institute of… Results Jan 2015 0.0% serious AE View details