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Results for “Mucopolysaccharidosis (MPS)”

163 trials

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Showing 20 of 163 results

Post-approval studies (Phase 4)Study completedNCT00299000
What this trial is testing

Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

Who this might be right for
Mucopolysaccharidosis VIMaroteaux-Lamy Syndrome
BioMarin Pharmaceutical 4
Not applicableStudy completedNCT03292887
What this trial is testing

Hunter Outcome Survey (HOS)

Who this might be right for
Hunter Syndrome
Shire 1,443
Not applicableStudy completedNCT01047306
What this trial is testing

Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome Type A
Shire 25
Post-approval studies (Phase 4)Study completedNCT02455622
What this trial is testing

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

Who this might be right for
Hunter Syndrome
Shire 21
Not applicableAvailableNCT07733856
What this trial is testing

Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)

Who this might be right for
Mucopolysaccharidosis Type IIIBMPS IIIB (Sanfilippo B Syndrome)
Spruce Biosciences
Not applicableStudy completedNCT03150069
What this trial is testing

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

Who this might be right for
Morquio DiseaseMPS - Mucopolysaccharidosis
Emory University 18
Testing effectiveness (Phase 2)UnknownNCT03632213
What this trial is testing

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Who this might be right for
Mucopolysaccharidosis IV AMucopolysaccharidosis VIMucopolysaccharidoses+6 more
Hospital de Clinicas de Porto Alegre 10
Post-approval studies (Phase 4)Enrolling By InvitationNCT05492799
What this trial is testing

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

Who this might be right for
MPS III B
Allievex Corporation 15
Not applicableEnded earlyNCT01733615
What this trial is testing

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

Who this might be right for
Mucopolysaccharidosis Type IVA
Emory University 3
Not applicableNot Yet RecruitingNCT07136896
What this trial is testing

Nutritional Assessment in Patient of Mucopolysaccharide "

Who this might be right for
Mucopolysaccharidosis (MPS)Malnutrition (Calorie)Undernutrition
Assiut University 40
Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
Testing effectiveness (Phase 2)Ended earlyNCT02350816
What this trial is testing

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Who this might be right for
Sanfilippo SyndromeMucopolysaccharidosis (MPS)
Shire 17
Post-approval studies (Phase 4)Study completedNCT00144781
What this trial is testing

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 34
Testing effectiveness (Phase 2)Active Not RecruitingNCT03153319
What this trial is testing

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 14
Testing effectiveness (Phase 2)Study completedNCT04571970
What this trial is testing

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis Type II (MPS II)
REGENXBIO Inc. 6
Not applicableStudy completedNCT01387854
What this trial is testing

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

Who this might be right for
MPS VI
BioMarin Pharmaceutical 59
Not applicableUnknownNCT01695161
What this trial is testing

Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.

Who this might be right for
MucopolysaccharidosisFabry Disease
Johannes Gutenberg University Mainz 75
Testing effectiveness (Phase 2)Study completedNCT03423186
What this trial is testing

Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Who this might be right for
Sanfilippo Syndrome Type A (MPS IIIA)
Swedish Orphan Biovitrum 6
Testing effectiveness (Phase 2)Ended earlyNCT04088734
What this trial is testing

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Who this might be right for
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical Inc 5
Not applicableEnded earlyNCT04628871
What this trial is testing

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

Who this might be right for
Hemophilia BMucopolysaccharidosis IMucopolysaccharidosis II
Sangamo Therapeutics 13
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