Study Purpose:
This study will assess the effects and safety of PTC857 treatment in participants diagnosed with ALS.Study Status:
Recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
PTC857, Placebo
Placebo:
Yes
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
N/A
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
Coordinating Center Contact Information
Patient Advocacy / email hidden; JavaScript is required / 1-866-562-4620
Full Study Summary:
Study Sponsor:
PTC Therapeutics
Estimated Enrollment:
258
Estimated Study Start Date:
05 / 15 / 2022
Estimated Study Completion Date:
02 / 28 / 2025
Posting Last Modified Date:
05 / 17 / 2023
Date Study Added to neals.org:
04 / 27 / 2022
Minimum Age:
18 Years
Maximum Age:
80 Years
Key Inclusion Criteria:- Males or females aged between 18 and 80 years with a body mass index between 18 and 35 kilograms/meters squared
- ALS with preserved function, defined as:
1. Onset of the first symptom leading to the diagnosis of ALS ≤24 months at the time of the initial Screening Visit
2. Revised EL Escorial criteria of either:
(i) Clinically definite ALS (ii) Clinically probable ALS
- A total ALSFRS-R score of at least 34 at the start of the Screening Period
- No significant respiratory compromise as evidenced by slow vital capacity ≥60% at the start of the Screening Period
- All concomitant medications (both prescription and over the counter), including standard-of-care therapy riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, and non-pharmacologic therapy regimens should be stable and unchanged from 30 days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study
- Female participants must have a negative breast cancer imaging screening status (not considered clinically abnormal and/or requiring further evaluation/treatment) within 6 months prior to the Screening Visit, or during the Screening Period.
Key Exclusion Criteria:
- Females who are pregnant or nursing or plan to become pregnant during the study
- Participants with clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular/ischemic disease or any other condition that, in the opinion of the investigator would jeopardize the safety of the participant or impact the validity of the study results
- Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the investigator or the medical monitor, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant
- Current participation in any other investigational study with an investigational product or participation within 30 days prior to the start of the Screening Period or 5 half-lives of the previously taken investigational drug, whichever is longer
- Participant has previously received PTC857
- Participant is receiving a combination of edaravone and sodium phenylbutyrate/taurursodiol treatment, where applicable, within 30 days prior to the start of the Screening Period
- For female participants, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer
UC Irvine Health ALS and Neuromuscular Center | Recruiting
Orange, California
92868
United States
Forbes Norris MDA/ALS Research Center at California Pacific Medical Center | Recruiting
San Francisco, California
94109
United States
Phil Smith Neuroscience Institute, Holy Cross Hospital | Recruiting
Fort Lauderdale, Florida
33308
United States
Intercoastal Medical Group, Inc. | Recruiting
Sarasota, Florida
34239
United States
University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare | Recruiting
Tampa, Florida
33612
United States
Augusta University | Recruiting
Augusta, Georgia
30912
United States
University of Kansas Medical Center (KUMC) - Landon Center on Aging | Recruiting
Kansas City, Kansas
66205
United States
Henry Ford Health System Department of Neurology | Recruiting
Detroit, Michigan
48202
United States
Neurology Associates, P.C. / Somnos Clinical Research | Recruiting
Lincoln, Nebraska
68506
United States
University of Nebraska Medical Center | Recruiting
Omaha, Nebraska
68198-8440
United States
Lewis Katz School of Medicine at Temple Universtiy | Recruiting
Philadelphia, Pennsylvania
19140
United States
National Neuromuscular Research Institute | Recruiting
Austin, Texas
78759
United States
Nerve and Muscle Center of Texas | Recruiting
Houston, Texas
77030
United States
Royal Brisbane and Women's Hospital | Recruiting
Brisbane, Queensland
4029
Australia
Austin Health | Recruiting
Heidelberg, Victoria
3084
Australia
CHU de Bordeaux | Recruiting
Bordeaux Cedex 33076
France
Hôpital Neurologique Pierre Wertheimer | Recruiting
Bron Cedex 69677
France
CHRU Lille - Hôpital Roger Salengro | Recruiting
Lille Cedex 59037
France
CHU Dupuytren 1 Limoges | Recruiting
Limoges 87000
France
Centrum Medyczne Neuro Protect | Recruiting
Warszawa 01-684
Poland
Hospital Universitario Vall d'Hebron ALS Unit. | Recruiting
Barcelona 08035
Spain
Hospital Universitario y Politecnico La Fe | Recruiting
Valencia 46026
Spain