Efficacy and Safety of ELGN-2112 on Intestinal Malabsorption in Preterm Infants

A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

NCT01422187 ·Status: COMPLETED ·Phase: PHASE3

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

NCT05152914 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia

NCT04902781 ·Status: COMPLETED ·Phase: PHASE2/PHASE3

A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)

NCT05543174 ·Status: COMPLETED ·Phase: PHASE3

Evaluation of Long Term Safety and Efficacy of Glepaglutide in Treatment of SBS

NCT03905707 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

An Observational Study to Collect Data on How Aflibercept (Eylea) Given Using a Paediatric Dosing Device is Used in Preterm Babies With Retinopathy of Prematurity in the United Kingdom (UK)

NCT06315556 ·Status: RECRUITING

Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia

NCT04117711 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760 ·Status: COMPLETED ·Phase: PHASE3

A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

NCT01132690 ·Status: COMPLETED ·Phase: PHASE4

Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

NCT03687333 ·Status: COMPLETED ·Phase: PHASE4

Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation

NCT02428296 ·Status: COMPLETED ·Phase: PHASE2

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

NCT06193304 ·Status: COMPLETED ·Phase: PHASE1

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

NCT02193867 ·Status: TERMINATED ·Phase: PHASE2

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261 ·Status: RECRUITING ·Phase: PHASE3

Evaluation of Long Term Safety and Efficacy of Glepaglutide in Treatment of SBS - Extension Trial

NCT04881825 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Synthetic Human Secretin in Children With Autism and Gastrointestinal Dysfunction

NCT00036231 ·Status: TERMINATED ·Phase: PHASE3

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

NCT04808505 ·Status: RECRUITING ·Phase: PHASE3

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

NCT03500094 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).

NCT04729751 ·Status: COMPLETED ·Phase: PHASE2

To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

NCT01301898 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

NCT00025896 ·Status: COMPLETED ·Phase: PHASE2

A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

NCT01411228 ·Status: COMPLETED ·Phase: PHASE3

A Phase II, Parallel Group, Randomized, Placebo-Controlled Study of the Safety and Efficacy of Thalidomide in Reducing Weight Loss in Adults With HIV Wasting Syndrome

NCT00002127 ·Status: COMPLETED ·Phase: PHASE2

China Post-marketing Surveillance (PMS) Study of Aldurazyme®

NCT05134571 ·Status: COMPLETED ·Phase: PHASE4

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT05270837 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3