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Clinical trials test new treatments โ€” sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

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30 recruiting trials

for Phenylketonuria (PKU)

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU) NCT04404530n/a2 sites๐Ÿ“ Atlanta๐Ÿ“ Atlanta Emory University ยท Tests: Palynziq Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference? NCT05128149n/a1 site๐Ÿ“ Zurich University Children's Hospital, Zurich ยท Tests: no intervention Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria NCT01659749NA1 site๐Ÿ“ Atlanta Emory University ยท Tests: Metabolic camp A Trial to Examine the Interaction of Repinatrabit With Ethinyl Estradiol/Norethindrone, Metformin,Carbamazepine, Rosuvastatin, and Methotrexate When Administered Together NCT07446400Phase 11 site๐Ÿ“ Melbourne Otsuka Pharmaceutical Development & Commercialization, Inc. ยท Tests: Repinatrabit, EE/NE, Metformin, Rosuvastatin, Methotrexate The Impact of Frequency of Home Phenylalanine Measurements on Metabolic Control in a Population of Patients With Classic Phenylketonuria NCT07825883NA1 site๐Ÿ“ Krakow Michaล‚ Kania ยท Tests: DBS Phe monitoring once weekly, DBS Phe monitoring once monthly PheCheckโ„ข Validation Study NCT07551921n/a2 sites๐Ÿ“ Chicago๐Ÿ“ Boston Aptatek BioSciences, Inc ยท Tests: PheCheck, Dried Blood Spot Cards. Immune Modulation During Palynziqยฎ Treatment in Adults (IMPALA) NCT07477691Phase 412 sites๐Ÿ“ Los Angeles๐Ÿ“ Aurora๐Ÿ“ Tampa BioMarin Pharmaceutical ยท Tests: Pegvaliase, Methotrexate A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU) NCT07672756Phase 11 site๐Ÿ“ Hefei Chongqing Peg-Bio Biopharm Co., Ltd. ยท Tests: PL54 Brain Aging in Phenylketonuria NCT06969209n/a1 site๐Ÿ“ Bern Insel Gruppe AG, University Hospital Bern Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience. NCT06289348n/a1 site๐Ÿ“ Paris Assistance Publique - Hรดpitaux de Paris ยท Tests: socio-psychological questionnaire, revised event impact scale (IER-S), Non directive interview, Stern interview, semi-dโ€ฆ A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) NCT05813678n/a26 sites๐Ÿ“ Aurora๐Ÿ“ Atlanta๐Ÿ“ Chicago BioMarin Pharmaceutical ยท Tests: Pegvaliase Walking Program in Fatty Liver Children With Phenylketonuria NCT06718842NA1 site๐Ÿ“ Dokki Cairo University ยท Tests: walking free online supervised program Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers NCT07220265NA1 site๐Ÿ“ Columbia University of Missouri-Columbia ยท Tests: Phenylalanine (Phe), Placebo Phenylalanine-free Diet for Patients With Secondary Hyperphenylalaninemia in ICU NCT04896281NA1 site๐Ÿ“ Keelung Chang Gung Memorial Hospital ยท Tests: Phenylalanine-free milk A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziqยฎ (Pegvaliase) During Pregnancy and Breastfeeding NCT05579548n/a4 sites๐Ÿ“ Wilmington๐Ÿ“ Calgary๐Ÿ“ Hamburg BioMarin Pharmaceutical ยท Tests: Pegvaliase A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria NCT07241234Phase 15 sites๐Ÿ“ Indianapolis๐Ÿ“ Pittsburgh๐Ÿ“ Dallas Agios Pharmaceuticals, Inc. ยท Tests: AG-181 A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function NCT07713758Phase 11 site๐Ÿ“ Miami Otsuka Pharmaceutical Development & Commercialization, Inc. ยท Tests: Repinatrabit A Study of Sepiapterin in Participants With Phenylketonuria (PKU) NCT06302348Phase 314 sites๐Ÿ“ Indianapolis๐Ÿ“ Nedlands๐Ÿ“ North Adelaide PTC Therapeutics ยท Tests: Sepiapterin GMP Powdered Substitutes in PKU and TYR NCT06941532NA1 site๐Ÿ“ Birmingham Nutricia UK Ltd ยท Tests: GMP Intervention Product GenSci144 Tablets Phase I Clinical Trial NCT07685210Phase 11 site๐Ÿ“ Jinan Changchun GeneScience Pharmaceutical Co., Ltd. ยท Tests: GenSci144 tablets, GenSci144 placebo tablets Multiomics Approach in Adult Patients With Phenylketonuria NCT07484945n/a15 sites๐Ÿ“ Angers๐Ÿ“ Bordeaux๐Ÿ“ Brest University Hospital, Tours A Long-Term Study of JNT-517 in Participants With Phenylketonuria NCT06628128Phase 312 sites๐Ÿ“ Gainesville๐Ÿ“ Tampa๐Ÿ“ Portland Otsuka Pharmaceutical Development & Commercialization, Inc. ยท Tests: JNT-517 Eating Disorders in Patients With Phenylketonuria NCT07698743n/a1 site๐Ÿ“ Nancy Central Hospital, Nancy, France A Study of JNT-517 in Participants With Phenylketonuria (PKU) NCT06971731Phase 326 sites๐Ÿ“ Los Angeles๐Ÿ“ Gainesville๐Ÿ“ Tampa Otsuka Pharmaceutical Development & Commercialization, Inc. ยท Tests: JNT-517, JNT-517 Matching Placebo Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria NCT06560736NA1 site๐Ÿ“ Columbia University of Missouri-Columbia ยท Tests: Show Me FIRST coping skills intervention A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) NCT06305234n/a11 sites๐Ÿ“ Aurora๐Ÿ“ Atlanta๐Ÿ“ Chicago BioMarin Pharmaceutical ยท Tests: Pegvaliase Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU NCT07728032n/a1 site๐Ÿ“ Birmingham Birmingham Women's and Children's NHS Foundation Trust ยท Tests: Phenylalanine-restricted diet alone, Sapropterin Dihydrochloride, Sepiapterin Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria NCT06337864NA2 sites๐Ÿ“ Copenhagen๐Ÿ“ Copenhagen Copenhagen University Hospital, Rigshospitalet ยท Tests: PreKUnilยฎ LNAA Medical Food for PKU AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) NCT06332807Phase 1, Phase 25 sites๐Ÿ“ Orange๐Ÿ“ Minneapolis๐Ÿ“ Morristown NGGT INC. ยท Tests: NGGT002 A Study of MZE782 in Adults With PKU NCT07694440Phase 29 sites๐Ÿ“ Oakland๐Ÿ“ Palo Alto๐Ÿ“ Chicago Maze Therapeutics ยท Tests: MZE782, Placebo, MZE782
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