Amicus Therapeutics

🇺🇸United States

Country: 🇺🇸United States

Ownership: Public

Established: 2002-01-01

Employees: 517

Market Cap: $3.3B

Website: http://www.amicusrx.com

Introduction: Amicus Therapeutics, Inc. operates as a biotechnology company. The firm focuses on discovering, developing and delivering medicines for people living with metabolic diseases. Its product portfolio includes first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants, a novel, clinical-stage, treatment paradigm for Pompe disease and a rare disease gene therapy portfolio. The company was founded by John Francis Crowley on February 4, 2002 and is headquartered in Princeton, NJ.

Clinical Trials

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

Phase 3

Recruiting

Conditions: Fabry Disease

Interventions: Drug: migalastat HCl 150 mg

First Posted Date: 2019-07-15

Last Posted Date: 2024-12-10

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 14

Registration Number: NCT04020055

Locations: 🇺🇸
Emory University, Atlanta, Georgia, United States
🇺🇸
The Cleveland Clinic, Cleveland, Ohio, United States
🇺🇸
Renal Disease Research Institute, Dallas, Texas, United States

and more 10 locations

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Phase 3

Recruiting

Conditions: Pompe Disease (Late-onset)

Interventions: Biological: Cipaglucosidase Alfa
Drug: Miglustat

First Posted Date: 2019-04-11

Last Posted Date: 2024-07-01

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 22

Registration Number: NCT03911505

Locations: 🇺🇸
Woodruff Memorial Research Building, Atlanta, Georgia, United States
🇺🇸
UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇺🇸
Arkansas Children's Hospital, Little Rock, Arkansas, United States

and more 20 locations

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Conditions: Pompe Disease

First Posted Date: 2019-03-07

Last Posted Date: 2024-08-16

Lead Sponsor: Amicus Therapeutics

Registration Number: NCT03865836

Gene Therapy for Children With CLN3 Batten Disease

Phase 1

Active, not recruiting

Conditions: CLN3
Batten Disease

Interventions: Genetic: High dose AT-GTX-502
Genetic: Low dose AT-GTX-502

First Posted Date: 2018-12-10

Last Posted Date: 2023-08-29

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 7

Registration Number: NCT03770572

Locations: 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Phase 3

Completed

Conditions: Pompe Disease (Late-onset)

Interventions: Biological: Cipaglucosidase Alfa
Biological: Alglucosidase Alfa
Drug: Miglustat
Drug: Placebo

First Posted Date: 2018-11-02

Last Posted Date: 2023-09-11

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 125

Registration Number: NCT03729362

Locations: 🇺🇸
The Ohio State University Wexner Medical Center, Columbus, Ohio, United States
🇫🇷
Hôpital Salengro, Lille, France
🇫🇷
Hôpital Pasteur, Nice, France

and more 70 locations

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

Phase 3

Completed

Conditions: Fabry Disease

Interventions: Drug: Migalastat HCl 150 mg

First Posted Date: 2018-04-17

Last Posted Date: 2021-11-30

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 22

Registration Number: NCT03500094

Locations: 🇬🇧
Clinical Study Site, London, United Kingdom

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Terminated

Conditions: Late-onset Pompe Disease

First Posted Date: 2017-11-20

Last Posted Date: 2019-02-08

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 12

Registration Number: NCT03347253

Locations: 🇺🇸
Hackensack University Medical Center, Hackensack, New Jersey, United States
🇺🇸
NYU Neurogenetics, New York, New York, United States
🇺🇸
University of California, Irvine, Irvine, California, United States

and more 18 locations

Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa

Completed

Conditions: Epidermolysis Bullosa

First Posted Date: 2017-05-18

Last Posted Date: 2017-08-24

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 150

Registration Number: NCT03158662

Locations: 🇺🇸
Amicus Therapeutics, Cranbury, New Jersey, United States

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

Phase 1

Completed

Conditions: Variant Late-Infantile Neuronal Ceroid Lipofuscinosis

Interventions: Genetic: AT-GTX-501

First Posted Date: 2016-04-01

Last Posted Date: 2023-03-08

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 13

Registration Number: NCT02725580

Locations: 🇺🇸
Nationwide Children's Hosptial, Columbus, Ohio, United States

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Phase 1

Completed

Conditions: Pompe Disease

Interventions: Drug: ATB200
Drug: AT2221

First Posted Date: 2016-02-05

Last Posted Date: 2024-08-23

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 32

Registration Number: NCT02675465

Locations: 🇺🇸
Lysosomal & Rare Disorders Research & Treatment Center (LDRTC), Fairfax, Virginia, United States
🇺🇸
Emory University Division of Medical Genetics, Decatur, Georgia, United States
🇬🇧
University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center, Birmingham, United Kingdom

and more 16 locations

Home Terms & Conditions Privacy Policy