University of Texas, Southwestern Medical Centre
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 0 Trials 
8 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
McNutt, Markey
NCT03335488 / 2015-000075-27: Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

Completed
4
16
Europe, US
RAVICTI, Glycerol phenylbutyrate, GPB, HPN-100, NaPBA, Sodium phenylbutyrate
Amgen
Urea Cycle Disorder
07/22
12/22
NCT05099640 / 2021-000474-29: A Study of PTC923 in Participants With Phenylketonuria

Completed
3
157
Europe, Canada, US, RoW
PTC923, Placebo
PTC Therapeutics
Phenylketonuria
04/23
05/23
SYNPHENY-3, NCT05764239: Efficacy and Safety of SYNB1934 in Patients With PKU

Terminated
3
35
Canada, US, RoW
SYNB1934v1, Placebo
Synlogic
Phenylketonuria
03/24
03/24
NCT05166161 / 2021-000497-28: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Recruiting
3
200
Europe, Canada, Japan, US, RoW
PTC923, Sepiapterin
PTC Therapeutics
Phenylketonuria
04/26
04/26
CACN00177-100D, NCT05154890 / 2019-004791-19: A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

Terminated
1/2
13
Europe, US, RoW
Pegtarviliase IV, ACN00177, Pegtarviliase SC
Aeglea Biotherapeutics, Aeglea Biotherapeutics
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
04/23
04/23
NCT06332807: AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

Recruiting
1/2
12
US
NGGT002
NGGT INC.
Phenylketonurias
12/30
12/30
NCT05222178: Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Terminated
1
3
US
HMI-103
Homology Medicines, Inc
Phenylketonurias, PAH Deficiency, Phenylketonuria
09/23
09/23
NCT05489549: Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers

Recruiting
N/A
500
US
University of Texas Southwestern Medical Center, The Cleveland Clinic, Columbia University, The University of Texas at Arlington, National Heart, Lung, and Blood Institute (NHLBI)
Amyloidosis, Hereditary, Amyloidosis Cardiac, Amyloidosis, Familial, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Transthyretin Gene Mutation
06/27
06/27

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