Hunter Syndrome: Clinical Trials

6 completed, 2 active not recruiting. 8 Phase 2, 4 Phase 3, 2 Phase 1.

By phase and status

Phase	Trials	Status
Phase 2	8	Completed 6, Active not recruiting 2
Phase 3	4	Completed 3, Active not recruiting 1
Phase 1	2	Completed 2

Follow Hunter Syndrome: new trials by email Literature Review of Hunter Syndrome trials Research Report on Hunter Syndrome

Trials (filtered) newest first · largest first

All 8 trials

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment Idursulfase-IT, Elaprase - Hunter Syndrome	Phase 2 / Phase 3	Active not recruiting	Takeda	6	2024-03-05	2025-11-06
Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 Idursulfase-IT, Elaprase - Hunter Syndrome	Phase 2 / Phase 3	Completed	Shire	56	2015-04-14	2025-06-19
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome	Phase 2	Active not recruiting	Masonic Cancer Center, University of Minnesota	149	2014-07-10	2026-11-03
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment idursulfase-IT, No IT treatment - Hunter Syndrome	Phase 2 / Phase 3	Completed	Shire	58	2014-03-24	2021-06-11
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 Idursulfase-IT, Elaprase - Hunter Syndrome	Phase 1 / Phase 2	Completed	Takeda	15	2010-08-01	2025-08-06
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome	Phase 2	Completed	Masonic Cancer Center, University of Minnesota	46	2009-12-01	2018-02-05
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® Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg) - Hunter Syndrome	Phase 1 / Phase 2	Completed	Shire	16	2009-11-18	2021-06-28
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase Idursulfase - Hunter Syndrome, Mucopolysaccharidosis II (MPS II)	Phase 2 / Phase 3	Completed	Shire	94	2004-09-13	2021-06-10

Narrower conditions

Mucopolysaccharidosis II (8)  ·  Lysosomal Storage Diseases (4)  ·  Sudden Infant Death (4)  ·  Aspartylglucosaminuria (2)  ·  Fucosidosis (2)  ·  Leukodystrophy, Globoid Cell (2)  ·  Leukodystrophy, Metachromatic (2)  ·  Mucopolysaccharidoses (2)  ·  Mucopolysaccharidosis I (2)  ·  Mucopolysaccharidosis VI (2)  ·  Mucopolysaccharidosis VII (2)  ·  Peroxisomal Disorders (2)  ·  Sphingolipidoses (2)  ·  alpha-Mannosidosis (2)  ·  Adrenoleukodystrophy (1)

Broader conditions

Carbohydrate Metabolism, Inborn Errors (8)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8)  ·  Connective Tissue Diseases (8)  ·  Genetic Diseases, Inborn (8)  ·  Genetic Diseases, X-Linked (8)  ·  Heredodegenerative Disorders, Nervous System (8)  ·  Intellectual Disability (8)  ·  Metabolic Diseases (8)  ·  Metabolism, Inborn Errors (8)  ·  Mucinoses (8)  ·  Nervous System Diseases (8)  ·  Neurobehavioral Manifestations (8)  ·  Neurologic Manifestations (8)  ·  Nutritional and Metabolic Diseases (8)  ·  Skin and Connective Tissue Diseases (8)

Interventions

idursulfase (4)  ·  Stem Cell Transplantation (2)  ·  Alemtuzumab (1)  ·  Busulfan (1)  ·  Cyclophosphamide (1)  ·  Cyclosporine (1)  ·  Iduronate Sulfatase (1)  ·  Mycophenolic Acid (1)  ·  Regimen B (1)

Sponsors

Shire (4)  ·  Masonic Cancer Center, University of Minnesota (2)  ·  Takeda (2)

Countries

United States (7)  ·  Canada (5)  ·  United Kingdom (5)  ·  France (4)  ·  Spain (3)  ·  Australia (2)  ·  Mexico (2)  ·  Brazil (1)  ·  Germany (1)  ·  Italy (1)  ·  Romania (1)  ·  Sweden (1)

Top investigators

Paul Orchard, M.D. (1)  ·  Paul Orchard, MD (1)

Results publications

PMID 35961250, PMID 37915038, PMID 21150784, PMID 22929184, PMID 35588317, PMID 36027721

NIH funding

2004 (1), 2014 (1), 2023 (1)