Hunter Syndrome

8
Pipeline Programs
3
Companies
25
Clinical Trials
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
2
2
3
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

Takeda
ELAPRASEApproved
idursulfase
Takeda
Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme [EPC]iv (infusion)2006

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Takeda
TakedaTOKYO, Japan
7 programs
1
2
2
ElaprasePhase 41 trial
Elaprase for intravenousPhase 41 trial
ELAPRASE(Idursulfase)Phase 2/35 trials
Idursulfase-ITPhase 2/31 trial
ControlPhase 1/25 trials
+2 more programs
Active Trials
NCT03582449Completed5Est. May 2020
NCT01449240Completed10Est. Dec 2013
NCT03942263Completed2,000Est. Dec 2022
+12 more trials
GC Biopharma
GC BiopharmaKorea - Yongin
2 programs
2
GC1111Phase 31 trial
HunterasePhase 31 trial
Active Trials
NCT03920540CompletedEst. Feb 2022
NCT01645189CompletedEst. Sep 2013
C
CENTOGENEGermany - Rostock
1 program
Biomarkers for Hunter SyndromeN/A1 trial
Active Trials
NCT01330277Terminated11Est. Dec 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
TakedaElaprase
TakedaElaprase for intravenous
TakedaIdursulfase
GC BiopharmaGC1111
GC BiopharmaHunterase
TakedaControl
TakedaIdursulfase-IT
TakedaIdursulfase
TakedaIdursulfase
TakedaIdursulfase
TakedaControl
TakedaIdursulfase
TakedaControl
TakedaControl
TakedaControl

Showing 15 of 24 trials with date data

Clinical Trials (25)

Total enrollment: 21,621 patients across 25 trials

A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

Start: Apr 2022Est. completion: Apr 20245 patients
Phase 4Completed
NCT02455622TakedaElaprase for intravenous

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

Start: Oct 2015Est. completion: Jul 202521 patients
Phase 4Completed

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

Start: Dec 2007Est. completion: Jul 201128 patients
Phase 4Completed

A Study of GC1111 in Hunter Syndrom Patients

Start: Mar 2017Est. completion: Feb 2022
Phase 3Completed

Safety and Efficacy of Hunterase

Start: Jul 2012Est. completion: Sep 2013
Phase 3Completed

Efficacy and Safety of Lanthanum in Controlling Serum Phosphate Levels in Subjects With End Stage Renal Disease Who Require Treatment for High Levels of Phosphate in Their Blood

Start: Feb 2004Est. completion: Oct 2005460 patients
Phase 3Completed
NCT06031259TakedaIdursulfase-IT

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Start: Mar 2024Est. completion: Jan 20296 patients
Phase 2/3Active Not Recruiting

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Start: Apr 2015Est. completion: Apr 202456 patients
Phase 2/3Completed

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

Start: Mar 2014Est. completion: Sep 201758 patients
Phase 2/3Completed

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

Start: Sep 2004Est. completion: Jan 200894 patients
Phase 2/3Completed

A Phase II Non-Controlled, Open-Label, Efficacy, Safety, Pharmacokinetic, and Pharmacodynamic Study of Pacritinib in Myelofibrosis

Start: Nov 2015Est. completion: Aug 20200
Phase 2Withdrawn

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

Start: Aug 2010Est. completion: Apr 202415 patients
Phase 1/2Completed

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®

Start: Nov 2009Est. completion: Oct 201216 patients
Phase 1/2Completed

Evaluation of Safety and Activity of Celaderm in Healing Venous Leg Ulcers

Start: Jan 2007Est. completion: Apr 200840 patients
Phase 1/2Completed

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

N/AAvailable

A Study to Describe Treatment Patterns and Disease Control in Participants With cHL and sALCL in Routine Clinical Practice in the Russian Federation

Start: May 2019Est. completion: Dec 20222,000 patients
N/ACompleted

A Study to Evaluate Disease Control, Treatment Patterns, Burden of Disease and Quality of Life in Participants With Moderate to Severe Inflammatory Bowel Disease (IBD)

Start: Dec 2018Est. completion: May 2019246 patients
N/ACompleted
NCT01330277CENTOGENEBiomarkers for Hunter Syndrome

Biomarkers for Hunter Syndrome

Start: Aug 2018Est. completion: Dec 202211 patients
N/ATerminated
NCT03582449TakedaIntensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Start: Jan 2018Est. completion: May 20205 patients
N/ACompleted
NCT01449240TakedaNo treatment

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Start: Nov 2012Est. completion: Dec 201310 patients
N/ACompleted

Candesartan Cilexetil Special Drug Use Surveillance 「Challenge - Quality Control」

Start: Jun 2011Est. completion: Apr 201318,113 patients
N/ACompleted

Case Control Study of Pharmacogenomic Factors Associated With Hepatocellular Injury Following Exposure to Lapaquistat Acetate

Start: May 2009Est. completion: May 201066 patients
N/ACompleted

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Start: Oct 2008Est. completion: Feb 201326 patients
N/ACompleted

Long-term Safety and Efficacy of Adipose-derived Stem Cells to Treat Complex Perianal Fistulas in Patients Participating in the FATT-1 Randomized Controlled Trial

Start: Sep 2008Est. completion: May 2010148 patients
N/ACompleted

Use of Expressive Writing in Irritable Bowel Syndrome (IBS)

Start: Sep 2008Est. completion: Nov 2010197 patients
N/ACompleted

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space