Mucopolysaccharidosis Disorders

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Mucopolysaccharidosis Disorders trials you may qualify for

100

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 30 trials

RecruitingNot Applicable

NCT05858606

Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients

This trial aims to evaluate the prevalence of idiopathic short stature among children whose growth is above -2,5SD (AFPA- CRESS/Inserm -CompuGroup Medical 2018…

France200 participantsStarted 2026-03-16

Treatment: Evaluation of the prevalence of truly (authentified) idiopathic short stature after multidisciplinary clinico-radiological evaluation, analysis in a multidisciplinary consultation meeting via a secure platform (ShareConfrère) for evaluation by multidisciplinary team

RecruitingPhase 4

NCT04152551

Effects of Bisphosphonates on OI-Related Hearing Loss

Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, wit…

United States100 participantsStarted 2019-11-02

Treatment: Risedronate Oral Tablet

RecruitingNot Applicable

NCT06749366

Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing

Background: Ollier disease (OD) and Maffucci syndrome (MS) are rare disorders that increase the risk of cancers in cartilage tissue. These tumors can lead to s…

United States100 participantsStarted 2025-01-27

RecruitingPhase 2/3

NCT02716246

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

United States, Australia, Spain36 participantsStarted 2016-04-25

Treatment: UX111, Prophylactic Immunomodulatory (IM) Therapy

RecruitingNot Applicable

NCT03604835

Mucopolysaccharidosis VII Disease Monitoring Program

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersens…

United States50 participantsStarted 2018-01-29

Treatment: No Intervention

RecruitingNot Applicable

NCT05619900

Registry of Patients Diagnosed With Lysosomal Storage Diseases

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…

United States250 participantsStarted 2022-05-31

Treatment: There is no intervention

All recent trials (100 shown)

CompletedNot Applicable

NCT00004738

Genetic Analysis of the Chiari I Malformation

The purpose of this study is to better understand the genetic factors related to the Chiari I malformation. In people with this abnormality, the lower part of t…

United States, Russia152 participantsStarted 2001-06-04

By InvitationNot Applicable

NCT06103487

Long Term Follow-Up for RGX-111

RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therap…

United States, Brazil21 participantsStarted 2023-07-24

Treatment: No Intervention

Mucopolysaccharidosis Disorders

Trial phase breakdown

Trial status

Actively recruiting — 30 trials

Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients

Effects of Bisphosphonates on OI-Related Hearing Loss

Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Mucopolysaccharidosis VII Disease Monitoring Program

Registry of Patients Diagnosed With Lysosomal Storage Diseases

All recent trials (100 shown)

Genetic Analysis of the Chiari I Malformation

Long Term Follow-Up for RGX-111

Trial phase breakdown

Trial status

Actively recruiting — 30 trials

Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients

Effects of Bisphosphonates on OI-Related Hearing Loss

Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Mucopolysaccharidosis VII Disease Monitoring Program

Registry of Patients Diagnosed With Lysosomal Storage Diseases

All recent trials (100 shown)

Genetic Analysis of the Chiari I Malformation

Long Term Follow-Up for RGX-111

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Radicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and…

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants Wit…

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

Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy Participants

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Expanded Access to Mepsevii

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

RGX-111 Gene Therapy in Patients With MPS I

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

Physical Activity Intervention for MPS

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

Mucopolysaccharidosis I (MPS I) Registry

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia