Recruiting studies: Enzyme Deficiency

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI
Phase 2 · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT06887777 Dose Individualization of Chemotherapy in Patients With Gastrointestinal Cancers Lacking a Specific Liver Enzyme
Phase 2 · 41 location(s) · UNICANCER · NCT06475352 RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Phase N/A · 1 location(s) · Hospital Universitari Vall d'Hebron Research Institute · NCT06213402 Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Phase N/A · 1 location(s) · Duke University · NCT01665326 A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked
Phase 2 · 1 location(s) · National Cancer Institute (NCI) · NCT05027945 A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compar
Phase 3 · 22 location(s) · Grifols Therapeutics LLC · NCT07555483 SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)
Phase N/A · 1 location(s) · University of California, San Francisco · NCT04885179 Effect OF Lactase Enzyme Supplements ON Intolerance IN Preterm Neonates
NA · 1 location(s) · Alexandria University · NCT07088302 A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset P
Phase 1 · 3 location(s) · Denali Therapeutics Inc. · NCT07354724

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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.