Long Term Follow-up Study of AAVAnc80-antiVEGF Gene Therapy

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

NCT06614569 ·Status: ACTIVE_NOT_RECRUITING

A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation

NCT06789913 ·Status: RECRUITING ·Phase: PHASE2

A Long-term, 3-Year, Follow-up Study for Patients Completing the KS-GIG-001-01 Study

NCT05613569 ·Status: ACTIVE_NOT_RECRUITING

Long Term Follow-Up for RGX-111

NCT06103487 ·Status: ENROLLING_BY_INVITATION

Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency

NCT06199531 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

NCT04273243 ·Status: ACTIVE_NOT_RECRUITING

Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201

NCT04999059 ·Status: TERMINATED

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

NCT04655911 ·Status: TERMINATED

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients

NCT05616793 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy

NCT05506254 ·Status: ACTIVE_NOT_RECRUITING

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

NCT04836377 ·Status: TERMINATED

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

NCT05878860 ·Status: RECRUITING ·Phase: PHASE3

Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)

NCT07293546 ·Status: NOT_YET_RECRUITING ·Phase: PHASE1/PHASE2

Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)

NCT06345898 ·Status: RECRUITING ·Phase: EARLY_PHASE1

Long-term Extension of GTX-102 in Angelman Syndrome

NCT06415344 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3

A Pharmacokinetic, Pharmacodynamic, Safety and Tolerability Study of CERC-006 in Adults With Complex Lymphatic Malformations

NCT04994002 ·Status: WITHDRAWN ·Phase: PHASE1

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)

NCT05073133 ·Status: COMPLETED ·Phase: PHASE4

Sirolimus Effect on Hypertrophic Syndromes Related Gene PIK3CA

NCT02443818 ·Status: COMPLETED ·Phase: PHASE2

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

NCT05371613 ·Status: RECRUITING ·Phase: PHASE2/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

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCT03041324 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Long-Term Follow-up of Subjects Who Were Treated With ST-920

NCT05039866 ·Status: ENROLLING_BY_INVITATION

Carisbamate Safety Study in Adult and Pediatric Subjects With Lennox-Gastaut Syndrome

NCT04062981 ·Status: COMPLETED ·Phase: PHASE1

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

NCT03612869 ·Status: UNKNOWN ·Phase: PHASE2/PHASE3

Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors

NCT00973739 ·Status: COMPLETED ·Phase: PHASE2