A Study to Assess the Effectiveness and Safety of Canakinumab in Clinical Use in Patients With Schnitzler's Syndrome

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

NCT04573023 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

NCT02663024 ·Status: UNKNOWN ·Phase: PHASE2

The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

NCT02044692 ·Status: UNKNOWN

Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia

NCT02961218 ·Status: COMPLETED ·Phase: PHASE2

Study of the Efficacy and Safety of RPH-104 in Adult Subjects With Schnitzler Syndrome

NCT04213274 ·Status: WITHDRAWN ·Phase: PHASE2

The Safety and Efficacy of Canakinumab in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada

NCT01105507 ·Status: COMPLETED ·Phase: PHASE3

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

NCT00607386 ·Status: COMPLETED ·Phase: PHASE4

Corticosteroids for Immune Reconstitution Inflammatory Syndrome (IRIS)

NCT01211665 ·Status: TERMINATED ·Phase: PHASE4

An Open Label Study of the Effects and Safety of Zanubrutinib in NMOSDs Adult Patients

NCT05356858 ·Status: TERMINATED ·Phase: PHASE2

A Study of Galcanezumab in Healthy Participants

NCT02576951 ·Status: COMPLETED ·Phase: PHASE1

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

NCT02262338 ·Status: COMPLETED ·Phase: PHASE1

Eliglustat on Gaucher Disease Type IIIB

NCT03519646 ·Status: COMPLETED ·Phase: NA

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

NCT05594992 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3

Safety and Efficacy of CC-10004 for Prurigo Nodularis

NCT00869089 ·Status: COMPLETED ·Phase: PHASE2

Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease

NCT00685373 ·Status: COMPLETED ·Phase: PHASE3

A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease

NCT02053896 ·Status: COMPLETED

A Study of SHR-2173 in Participants With Primary IgA Nephropathy

NCT07354932 ·Status: RECRUITING ·Phase: PHASE2

Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease

NCT02770625 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

NCT05422482 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1

Screening Trial for Pain Relief in Schwannomatosis (STARFISH)

NCT05684692 ·Status: RECRUITING ·Phase: PHASE2

Rituximab in the First Episode of Paediatric Nephrotic Syndrome

NCT05850546 ·Status: NOT_YET_RECRUITING ·Phase: PHASE3

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

NCT01830972 ·Status: COMPLETED ·Phase: PHASE2

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

NCT02341807 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Clinical Evaluation of Patients With X-linked Retinoschisis

NCT02331173 ·Status: COMPLETED

Exploratory Study of NS-089/NCNP-02 in DMD

NCT04129294 ·Status: COMPLETED ·Phase: PHASE1/PHASE2