A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

NCT04674761 ·Status: COMPLETED ·Phase: PHASE3

Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)

NCT07290257 ·Status: RECRUITING ·Phase: PHASE4

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

NCT05174039 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Study of rhASB in Patients With Mucopolysaccharidosis VI

NCT00104234 ·Status: COMPLETED ·Phase: PHASE3

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

NCT05543174 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

NCT06188325 ·Status: COMPLETED ·Phase: PHASE1

A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

NCT05677659 ·Status: TERMINATED ·Phase: PHASE2

Managed Access Programs for BYL719, Alpelisib

NCT04085653 ·Status: AVAILABLE

A Phase 1, Study of the Safety and Tolerability of ALV003 in Healthy Adult Volunteers and Subjects With Well-Controlled Celiac Disease

NCT00626184 ·Status: COMPLETED ·Phase: PHASE1

Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis

NCT01687179 ·Status: COMPLETED ·Phase: PHASE1

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

NCT00048711 ·Status: COMPLETED ·Phase: PHASE2

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

NCT00925301 ·Status: COMPLETED ·Phase: PHASE3

Open-Label Phase 3 Long-Term Safety Study of Migalastat

NCT01458119 ·Status: TERMINATED ·Phase: PHASE3

A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

NCT05791864 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

NCT00214773 ·Status: COMPLETED

Testing Miglustat Administration in Subjects With Spastic Paraplegia 11

NCT04768166 ·Status: COMPLETED ·Phase: PHASE2

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

NCT00954460 ·Status: APPROVED_FOR_MARKETING

Long Term Follow-Up for RGX-111

NCT06103487 ·Status: ENROLLING_BY_INVITATION

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

NCT01842841 ·Status: COMPLETED ·Phase: PHASE3

An Intermediate Size Expanded Access Protocol of Elamipretide

NCT04689360 ·Status: AVAILABLE

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

NCT03500094 ·Status: COMPLETED ·Phase: PHASE3

Gene Therapy Study for Children With CLN5 Batten Disease

NCT05228145 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

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

NCT01422187 ·Status: COMPLETED ·Phase: PHASE3

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

AT-007 in Adult Subjects With Classic Galactosemia (CG)

NCT05418829 ·Status: UNKNOWN ·Phase: PHASE3