Phase 1 N=11 Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
Hurler Syndrome
Primary: Percentage of Adherence to the Scheduled Weekly Infusion by the Participants — 99 percentage
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Phase 2 N=41 Stem Cell Transplantation for Hurler
Mucopolysaccharidosis I · Mucopolysaccharidosis VI · Mannosidosis
Primary: Mean Percentage of Donor Cells in Study Population (Chimerism). — 85.8; 73.2; 84.6; 81.1 Percentage
Phase 2 N=25 Stem Cell Transplant w/Laronidase for Hurler
Mucopolysaccharidosis I · Hurler Syndrome
Primary: Number of Patients Alive at One Year Post Transplant — 18 Participants
Phase 2 N=12 Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Mucopolysaccharidosis (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
Phase 2 N=21 Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615
Phase 2 N=6 A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
Phase 2 N=6 A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 53; 128; 0; 1 number of events
Phase 3 N=58 Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Hunter Syndrome
Primary: Change From Baseline in the Differential Ability Scales, Second Edition (DAS-II) General Conceptual Ability (GCA) Standard Score at Week 52 — -7.4; -4.4 Score on a scale…
Phase 4 N=28 Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
Hunter Syndrome · Mucopolysaccharidosis II · MPS II
Primary: Safety Evaluation — 28; 0; 0; 16 participants
Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Phase 4 N=5 A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
Hunter Syndrome
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death — 5; 1; 1; 0 Participants
Phase 3 N=94 Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Hunter Syndrome · Mucopolysaccharidosis II (MPS II)
Primary: Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105 — -0.056 percent predicted FVC
N/A N=10 Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
Hunter Syndrome
Primary: Levels of Total Glycosaminoglycan (GAG) in CSF — 816.750 ng/mL
Phase 3 N=96 Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
Mucopolysaccharidosis II
Primary: Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53 — 69.81; 50.86 sum of the ranked scores — p=0.0049
Phase 4 N=66 Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
Hunter Syndrome
Primary: Height Overall — 113.759; 108.143 centimeter (cm) — p== 0.011
Phase 3 N=44 Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher Disease
Primary: Spleen Volume — 2324.0; 2120.0; 778.0; 1707.7 mL
Phase 3 N=3 Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Mucopolysaccharidosis I · Hurler Syndrome · Hurler-Scheie Syndrome
Primary: Safety Evaluation — 3; 0; 0; 1 participants
N/A N=25 A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Sanfilippo Syndrome Type A
Primary: Change From Baseline in Bayley Scales of Infant Development-III/Kaufman Assessment Battery for Children-II (BSID-III/KABC-II) Age-Equivalent Scores — 0.17; 1.19; -1.72…
Phase 3 N=45 Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
Mucopolysaccharidosis I · Hurlers Syndrome · Hurler-Scheie Syndrome
Primary: Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC) — 54.2; 48.4; 51.5; 50.2 Percent predicted FVC
Phase 2 N=11 Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Mucopolysaccharidosis IIIB
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1; 0; 1 Participants
N/A N=33 Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®
Hunter Syndrome
Primary: Number of Participants Who Were Screened For The Follow-On Study With an Investigational Agent — 17 participants
Phase 2 N=15 Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Mucopolysaccharidosis IVA · Morquio A Syndrome · MPS IVA
Primary: To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of…
Phase 4 N=12 Growth and Development Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm
Phase 3 N=177 A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
MPS IV A
Primary: Change From Baseline in Endurance as Measured by the 6-minute Walk Test — 211.9; 205.7; 203.9; 225.4 meters — p=0.0174
Phase 2 N=15 An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
Hunter Syndrome
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 4; 10; 5 Participants
Phase 2 N=16 A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Hunter Syndrome
Primary: Number of Serious Adverse Event (SAE) — 0; 8; 3; 3 events
Phase 2 N=24 Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
Metachromatic Leukodystrophy (MLD)
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity — 6; 6; 6; 6 Participants
Phase 2 N=20 A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Mucopolysaccharidosis I · Hurler Syndrome · Hurler-Scheie Syndrome
Primary: Safety Evaluation — 16; 4; 5; 3 participants
Phase 2 N=18 A Study of JR-171 in Patients With Mucopolysaccharidosis I
Mucopolysaccharidosis I
Primary: Number of Participants With Adverse Events — 3; 6; 7; 0 participants
Phase 2 N=8 Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
Sly Syndrome · MPS VII · Mucopolysaccharidosis
Primary: Percent Change From Baseline in uGAG Excretion (LC-MS/MS-DS) at Week 48 — -58.17 percent change — p=< 0.0001