A Phase 2 study of PTC857 in patients with Amyotrophic Lateral Sclerosis

Phase 1

• Conditions: Amyotrophic Lateral Sclerosis (ALS); MedDRA version: 21.1Level: PTClassification code 10002026Term: Amyotrophic lateral sclerosisSystem Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2021-006511-29-SE
• Lead Sponsor: PTC Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-11-11
• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 258

Inclusion Criteria

1. Males or females aged between 18 and 80 years at the time of the initial Screening visit

2. ALS with preserved function, defined as:
a. Onset of the first symptom leading to the diagnosis of ALS =24 months at the time of the initial Screening Visit
b. Revised El Escorial criteria of either:
(i) Clinically definite ALS
(ii) Clinically probable ALS

3. A total ALSFRS-R score of at least 34 at the start of the Screening Period

4. No significant respiratory compromise as evidenced by slow vital capacity =60% at the start of the Screening Period

5. All chronic concomitant medications (both prescription and over the counter [OTC]) and non pharmacologic therapy regiments, excluding standard-of-care therapy riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, and non pharmacologic therapy regimens should be stable and unchanged from at least 14 days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study

6. Female subjects 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.

7. Standard-of-care therapy for the treatment of ALS (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol) should be stable and unchanged from 30 (-3) days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 248
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

1. Females who are pregnant or nursing or plan to become pregnant during the study
2. Subjects 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 subject or impact the validity of the study results
3. 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 subject’s ability to participate in the study or increase the risk of participation for that subject
4. 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
5. Subject has previously received PTC857
6. Subject is receiving a combination of edaravone and sodium phenylbutyrate/taurursodiol treatment, where applicable, within 30 (-3) days prior to the start of the Screening Period
7. For female subjects, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified