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):
Subha Krishnan, PTC Therapeutics
Clinicaltrials.gov ID:
Neals Affiliated?
No
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:
11 / 18 / 2022
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
California Pacific Medical Center Research Institute, Sutter Health | Recruiting
San Francisco, California
94107
United States
University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare | Recruiting
Tampa, Florida
33612
United States
University of Kansas Medical Center (KUMC) - Landon Center on Aging | Recruiting
Kansas City, Kansas
66103
United States
Austin Neuromuscular Center | Recruiting
Austin, Texas
78759
United States
University of Texas Southwestern Medical Center | Recruiting
Dallas, Texas
75390
United States