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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Assess the Efficacy, Safety, Tolerability, PK, and Biomarker Effects of PTC857 in Adult Subjects With Amyotrophic Lateral Sclerosis (CARDINALS)
EU CTIS ID: 2023-510317-26-00
What this study is testing
To evaluate the efficacy of PTC857 in reducing disease progression in subjects with amyotrophic lateral sclerosis (ALS)
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Males or females aged between 18 and 80 years at the time of the initial Screening Visit
- Females must refrain from ova (egg cell) donation from the start of the Screening Period through 90 days after the last dose of study drug
- Males, if not surgically sterilized, with female partners of childbearing potential must agree to use barrier contraceptive (ie, condom) and their female partners must use a highly effective method of contraception from the start of the Screening Period through 90 days after the last dose of study drug
- Males must refrain from sperm donations from the start of the Screening Period through 90 days after the last dose of the study drug
- Willing and able to comply with all protocol procedures
- 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
You likely can't join if
- Females who are pregnant or nursing or plan to become pregnant during the study
- For female subjects, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer
- History of allergies or adverse reactions to any of the excipients in the study drug formulation
- Hepatic insufficiency, defined as liver function tests (LFTs) (ie, AST and/or ALT) ≥3× the upper limit of normal (ULN), or bilirubin ≥1.5× the ULN (except in the case of Gilbert’s disease)
- Subject is taking a non-approved form of sodium phenylbutyrate and/or taurursodiol for the treatment of ALS
- Moderate or worse renal insufficiency, defined as an estimated glomerular filtration rate (eGFR) of <60 mL/min
See the full eligibility criteria
Who can join
- Males or females aged between 18 and 80 years at the time of the initial Screening Visit
- Females must refrain from ova (egg cell) donation from the start of the Screening Period through 90 days after the last dose of study drug
- Males, if not surgically sterilized, with female partners of childbearing potential must agree to use barrier contraceptive (ie, condom) and their female partners must use a highly effective method of contraception from the start of the Screening Period through 90 days after the last dose of study drug
- Males must refrain from sperm donations from the start of the Screening Period through 90 days after the last dose of the study drug
- Willing and able to comply with all protocol procedures
- 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
- 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 (refer to the laboratory manual for specific requirements)
- All chronic concomitant medications (both prescription and over the counter [OTC]) and non-pharmacologic therapy regimens, excluding standard-of-care therapy riluzole, edaravone, or sodium phenylbutyrate/taurursodiol (refer to inclusion criterion 13) should be stable and unchanged from 14 days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study
- 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
- 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. Note: should a subject be discontinued from standard-of-care therapy due to removal of the therapy from the market, this will not be considered a protocol deviation.
- Subjects or their designee (ie, legal authorized representative or caregiver) must understand the nature of the study and must provide signed and dated written informed consent prior to conducting any study-related procedures
- Females must be either postmenopausal for ≥1 year (cessation of menses for 12 consecutive months) or surgically sterile (having undergone tubal ligation, hysterectomy, or bilateral oophorectomy) for at least 6 months or, if of childbearing potential and not abstinent, willing to use a highly effective method of contraception from the start of the Screening Period through 90 days after the last dose of study drug. Females who are abstinent will not be required to use a contraceptive method unless they become sexually active
What rules you out
- Females who are pregnant or nursing or plan to become pregnant during the study
- For female subjects, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer
- History of allergies or adverse reactions to any of the excipients in the study drug formulation
- Hepatic insufficiency, defined as liver function tests (LFTs) (ie, AST and/or ALT) ≥3× the upper limit of normal (ULN), or bilirubin ≥1.5× the ULN (except in the case of Gilbert’s disease)
- Subject is taking a non-approved form of sodium phenylbutyrate and/or taurursodiol for the treatment of ALS
- Moderate or worse renal insufficiency, defined as an estimated glomerular filtration rate (eGFR) of <60 mL/min
- History of alcohol or drug abuse within the last 6 months prior to the start of the Screening Period or current evidence of substance dependence
- Any surgery within 30 days prior to the start of the Screening Period that may affect the subject’s ability to complete all study procedures
- 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
- 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
- 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 longe
- Subject has previously received PTC857
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Australia
- Argentina
- Japan
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia; Argentina; Japan; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.