O & O Alpan
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 8 Trials 
16 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Goker-Alpan, Ozlem
SWITCH, NCT04718779: A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

Completed
4
4
US
Digital Engagement Application (GD App), No Intervention
Takeda
Gaucher Disease
02/23
02/23
LEAP2MONO, NCT05222906 / 2021-005402-10: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Active, not recruiting
3
43
Europe, Canada, Japan, US, RoW
Venglustat, imiglucerase, Cerezyme®
Sanofi, Sanofi-aventis recherche & développement
Gaucher's Disease Type III
09/25
10/26
NCT06614894: An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

Not yet recruiting
2/3
10
US
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
Ozlem Goker-Alpan, Team Sanfilippo
Sanfilippo Syndrome, MPS3
02/26
02/26
STAAR, NCT04046224 / 2019-000667-24: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

Active, not recruiting
1/2
34
Europe, Canada, US, RoW
ST-920
Sangamo Therapeutics
Fabry Disease
04/25
09/25
NCT06545136: Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

Recruiting
1/2
75
Europe, US, RoW
FLT201
Freeline Therapeutics
Gaucher Disease, Type 1
05/29
05/29
CT-AMT-191-01, NCT06270316: Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Recruiting
1/2
12
US
AMT-191
UniQure Biopharma B.V.
Fabry Disease
04/27
04/27
NCT06858397: A Proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients with Fabry Disease

Not yet recruiting
1/2
18
US, RoW
HM15421/GC1134A
GC Biopharma Corp, Hanmi Pharmaceutical co., ltd.
Fabry Disesase
08/28
08/28
NCT05238324: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Withdrawn
1
9
Canada, US
Genetic HMI-203
Homology Medicines, Inc
Mucopolysaccharidosis II
10/24
01/29
GALILEO-1, NCT05324943 / 2020-005032-30: A Gene Therapy Study in Patients With Gaucher Disease Type 1

Active, not recruiting
1
18
Europe, US, RoW
FLT201
Freeline Therapeutics, Freeline Therapeutics Limited
Gaucher Disease, Type 1
12/24
01/25
NCT02000310: Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Recruiting
N/A
80
US
O & O Alpan LLC
Lysosomal Storage Disorders
12/21
12/22
NCT04552691: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

Approved for marketing
N/A
US
Pegunigalsidase Alfa
Chiesi Farmaceutici S.p.A., Chiesi USA, Inc.
Fabry Disease
 
 
PEDS, NCT04721366: A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

Completed
N/A
11
US
Standard of Care, SOC
Takeda
Gaucher Disease
04/23
04/23
NCT06065605: Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

Recruiting
N/A
40
US
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fabry Disease
07/25
09/25
NCT06116071: Biomarkers Related to Bone in Pediatric Gaucher Disease

Recruiting
N/A
20
US
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Gaucher Disease, Bone Diseases
10/25
12/25
NCT04724083: Inflammatory Pathways and Cardiac Growth Factors Associated With Fabry Disease Cardiomyopathy

Recruiting
N/A
50
US
biomarkers
Lysosomal and Rare Disorders Research and Treatment Center, Inc., Sanofi
Fabry
05/22
08/22
NCT05705674: The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

Recruiting
N/A
6
US
Lysosomal and Rare Disorders Research and Treatment Center, Inc., Team Sanfilippo
Sanfilippo Syndrome, MPS3
12/24
12/24
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Goker-Alpan, Ozlem
SWITCH, NCT04718779: A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

Completed
4
4
US
Digital Engagement Application (GD App), No Intervention
Takeda
Gaucher Disease
02/23
02/23
LEAP2MONO, NCT05222906 / 2021-005402-10: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Active, not recruiting
3
43
Europe, Canada, Japan, US, RoW
Venglustat, imiglucerase, Cerezyme®
Sanofi, Sanofi-aventis recherche & développement
Gaucher's Disease Type III
09/25
10/26
NCT06614894: An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

Not yet recruiting
2/3
10
US
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
Ozlem Goker-Alpan, Team Sanfilippo
Sanfilippo Syndrome, MPS3
02/26
02/26
STAAR, NCT04046224 / 2019-000667-24: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

Active, not recruiting
1/2
34
Europe, Canada, US, RoW
ST-920
Sangamo Therapeutics
Fabry Disease
04/25
09/25
NCT06545136: Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

Recruiting
1/2
75
Europe, US, RoW
FLT201
Freeline Therapeutics
Gaucher Disease, Type 1
05/29
05/29
CT-AMT-191-01, NCT06270316: Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Recruiting
1/2
12
US
AMT-191
UniQure Biopharma B.V.
Fabry Disease
04/27
04/27
NCT06858397: A Proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients with Fabry Disease

Not yet recruiting
1/2
18
US, RoW
HM15421/GC1134A
GC Biopharma Corp, Hanmi Pharmaceutical co., ltd.
Fabry Disesase
08/28
08/28
NCT05238324: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Withdrawn
1
9
Canada, US
Genetic HMI-203
Homology Medicines, Inc
Mucopolysaccharidosis II
10/24
01/29
GALILEO-1, NCT05324943 / 2020-005032-30: A Gene Therapy Study in Patients With Gaucher Disease Type 1

Active, not recruiting
1
18
Europe, US, RoW
FLT201
Freeline Therapeutics, Freeline Therapeutics Limited
Gaucher Disease, Type 1
12/24
01/25
NCT02000310: Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Recruiting
N/A
80
US
O & O Alpan LLC
Lysosomal Storage Disorders
12/21
12/22
NCT04552691: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

Approved for marketing
N/A
US
Pegunigalsidase Alfa
Chiesi Farmaceutici S.p.A., Chiesi USA, Inc.
Fabry Disease
 
 
PEDS, NCT04721366: A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

Completed
N/A
11
US
Standard of Care, SOC
Takeda
Gaucher Disease
04/23
04/23
NCT06065605: Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

Recruiting
N/A
40
US
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fabry Disease
07/25
09/25
NCT06116071: Biomarkers Related to Bone in Pediatric Gaucher Disease

Recruiting
N/A
20
US
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Gaucher Disease, Bone Diseases
10/25
12/25
NCT04724083: Inflammatory Pathways and Cardiac Growth Factors Associated With Fabry Disease Cardiomyopathy

Recruiting
N/A
50
US
biomarkers
Lysosomal and Rare Disorders Research and Treatment Center, Inc., Sanofi
Fabry
05/22
08/22
NCT05705674: The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

Recruiting
N/A
6
US
Lysosomal and Rare Disorders Research and Treatment Center, Inc., Team Sanfilippo
Sanfilippo Syndrome, MPS3
12/24
12/24

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