Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers

NCT05028764 ·Status: COMPLETED

Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT06941025 ·Status: RECRUITING

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

NCT05358886 ·Status: COMPLETED ·Phase: PHASE3

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

NCT06706388 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Multicenter Study Evaluating the Safety of Proellex® in Premenopausal Women With Uterine Fibroids

NCT00737282 ·Status: TERMINATED ·Phase: PHASE3

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria

NCT04534842 ·Status: COMPLETED ·Phase: PHASE2

Evaluating the Safety and Efficacy of Proellex® (CDB-4124) in Premenopausal Women With Symptomatic Uterine Fibroids

NCT00735553 ·Status: TERMINATED ·Phase: PHASE3

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

NCT03516487 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia

NCT04117880 ·Status: WITHDRAWN ·Phase: PHASE2

First in Human Study to Test the Safety and Preliminary Efficacy of PPSGG in Patients With Anti-MAG Neuropathy

NCT04568174 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

NCT03759639 ·Status: COMPLETED ·Phase: PHASE2

Carisbamate in Adult & Pediatric Subjects With Lennox-Gastaut Syndrome

NCT03731715 ·Status: COMPLETED ·Phase: PHASE1

Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

NCT03810690 ·Status: WITHDRAWN ·Phase: PHASE1/PHASE2

Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)

NCT02386839 ·Status: COMPLETED ·Phase: PHASE2

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

NCT05367960 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

NCT05967351 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3

A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Intravitreal Injections of FCFD4514S in Patients With Geographic Atrophy

NCT00973011 ·Status: COMPLETED ·Phase: PHASE1

An Open-Label Study to Assess the Safety & Efficacy of Leniolisib in Japanese Patients With APDS

NCT06249997 ·Status: RECRUITING ·Phase: PHASE3

Effect of P-glycoprotein Inhibition on Lenalidomide Pharmacokinetics in Healthy Males

NCT01712828 ·Status: COMPLETED ·Phase: PHASE1

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288 ·Status: RECRUITING ·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

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

NCT03675126 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

PASS of Paediatric Patients Initiating Selumetinib

NCT05388370 ·Status: ACTIVE_NOT_RECRUITING

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

NCT05515536 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Disease Progression in Women With X-linked Adrenoleukodystrophy

NCT06178120 ·Status: RECRUITING