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PTC Therapeutics

PTC Therapeutics logo
🇺🇸United States
Ownership
Public
Established
1998-01-01
Employees
1K
Market Cap
$2.7B
Website
http://www.ptcbio.com
Introduction

PTC Therapeutics, Inc. is a biopharmaceutical company, which focuses on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. The company was founded by Allan Steven Jacobson and Stuart Walter Peltz on March 31, 1998 and is headquartered in Warren, NJ.

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

Phase 3
Recruiting
Conditions
Phenylketonuria
Interventions
First Posted Date
2024-03-08
Last Posted Date
2025-04-29
Lead Sponsor
PTC Therapeutics
Target Recruit Count
56
Registration Number
NCT06302348
Locations
🇺🇸

Indiana University, Indianapolis, Indiana, United States

An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

Phase 2
Conditions
Huntington Disease
Interventions
First Posted Date
2024-02-12
Last Posted Date
2025-03-11
Lead Sponsor
PTC Therapeutics
Target Recruit Count
250
Registration Number
NCT06254482
Locations
🇦🇹

Medical University Innsbruck, Innsbruck, Austria

🇦🇺

Westmead Hospital, Westmead, Australia

🇨🇦

The Ottawa Hospital, Parkinson's and Movement Disorders Clinic, Ottawa, Canada

and more 21 locations

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Phase 3
Active, not recruiting
Conditions
Friedreich Ataxia
Interventions
First Posted Date
2022-08-25
Last Posted Date
2025-04-17
Lead Sponsor
PTC Therapeutics
Target Recruit Count
130
Registration Number
NCT05515536
Locations
🇺🇸

University of South Florida, Tampa, Florida, United States

🇺🇸

UCLA, Los Angeles, California, United States

🇺🇸

University of Iowa, Iowa City, Iowa, United States

and more 11 locations

A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Phase 2
Completed
Conditions
Friedreich Ataxia
Interventions
First Posted Date
2022-08-03
Last Posted Date
2024-10-08
Lead Sponsor
PTC Therapeutics
Target Recruit Count
5
Registration Number
NCT05485987
Locations
🇺🇸

The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States

A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

Phase 2
Active, not recruiting
Conditions
Huntington Disease
Interventions
Drug: Placebo
First Posted Date
2022-05-03
Last Posted Date
2025-02-20
Lead Sponsor
PTC Therapeutics
Target Recruit Count
252
Registration Number
NCT05358717
Locations
🇩🇪

Charite University Medicine Berlin, Berlin, Germany

🇺🇸

University of California, San Diego, La Jolla, California, United States

🇺🇸

Vanderbilt University Medical Center, Nashville, Tennessee, United States

and more 25 locations

Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

Phase 2
Terminated
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Drug: Placebo
First Posted Date
2022-04-27
Last Posted Date
2025-03-26
Lead Sponsor
PTC Therapeutics
Target Recruit Count
307
Registration Number
NCT05349721
Locations
🇪🇸

Unidad Neuromuscular. Servicio de Neurologia Hospital Universitari Vall d'Hebron, Barcelona, Spain

🇮🇹

Maggiore della Carita University Hospital, Neurology department, ALS center, Novara, Italy

🇮🇹

AOU Citta Della Salute e Scienza, Torino, Italy

and more 52 locations

A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)

Phase 2
Terminated
Conditions
Leiomyosarcoma
Interventions
First Posted Date
2022-03-08
Last Posted Date
2025-03-19
Lead Sponsor
PTC Therapeutics
Target Recruit Count
360
Registration Number
NCT05269355
Locations
🇺🇸

University of California, Los Angeles (UCLA) - Jonsson Comprehensive Cancer Center, Los Angeles, California, United States

🇺🇸

Northwestern Memorial Hospital, Chicago, Illinois, United States

🇺🇸

The Ohio State University (OSU), Columbus, Ohio, United States

and more 51 locations

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Phase 3
Active, not recruiting
Conditions
Inherited Mitochondrial Disease
Interventions
First Posted Date
2022-02-01
Last Posted Date
2025-03-04
Lead Sponsor
PTC Therapeutics
Target Recruit Count
102
Registration Number
NCT05218655
Locations
🇺🇸

The Johns Hopkins University, Baltimore, Maryland, United States

🇺🇸

Akron Children's Hospital, Akron, Ohio, United States

🇺🇸

Children's Hospital of Philadelphia - CHOP, Philadelphia, Pennsylvania, United States

and more 23 locations

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Phase 3
Recruiting
Conditions
Phenylketonuria
Interventions
First Posted Date
2021-12-21
Last Posted Date
2025-05-08
Lead Sponsor
PTC Therapeutics
Target Recruit Count
200
Registration Number
NCT05166161
Locations
🇺🇸

Children's Hospital Colorado, Aurora, Colorado, United States

🇺🇸

Indiana University School of Medicine Department of Medical and Molecular Genetics, Indianapolis, Indiana, United States

🇺🇸

University of Utah, Salt Lake City, Utah, United States

and more 42 locations

A Study of PTC923 in Participants With Phenylketonuria

Phase 3
Completed
Conditions
Phenylketonuria
Interventions
Drug: Placebo
First Posted Date
2021-10-29
Last Posted Date
2024-01-10
Lead Sponsor
PTC Therapeutics
Target Recruit Count
157
Registration Number
NCT05099640
Locations
🇫🇷

Bretonneau Hospital - CHRU de Tours, Tours, Centre-Val De Loire, France

🇺🇸

Boston Children's Hospital, Boston, Massachusetts, United States

🇺🇸

Icahn School of Medicine at Mount Sinai (ISMMS), New York, New York, United States

and more 40 locations
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