臨床研究等提出・公開システム

A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED,PARALLEL STUDY TO ASSESS THE EFFICACY,SAFETY, TOLERABILITY, PK, AND BIOMARKER EFFECTS OF PTC857 IN ADULT SUBJECTS WITH AMYOTROPHIC LATERAL SCLEROSIS (CARDINALS)

CARDINALS

Tansy Aaron

PTC Therapeutics, Inc.

100 Corporate Court South Plainfield, NJ 07080 US

atansy@ptcbio.com

Kim Min

Worldwide Clinical Trials Japan K.K.

2-15-1, Konan, Minato-ku, Tokyo Shinagawa Intercity, Tower A, Level 28

+81-3-6717-4360

min.kim@worldwide.com

Not Recruiting

Oct. 12, 2023

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

other

1. Males or females aged between 18 and 80 years with a body mass index between 18
and 35 kg/m2 at the time of the initial Screening Visit
2. Onset of the first symptom leading to the diagnosis of ALS is within 24 months at the time of the initial Screening Visit
3. Revised El Escorial criteria of either:
(i) Clinically definite ALS
(ii) Clinically probable ALS
4. A total ALSFRS-R score of at least 34 at the start of the Screening Period

1.History of allergies or adverse reactions to any of the excipients in the study drug formulation
2. Liver failure (except in Gilbert syndrome, defined as AST and/or ALT 3 times the upper limit of normal [ULN] or liver function test [LFT] with bilirubin 1.5 times the ULN))
3. Moderate to severe renal failure (defined as estimated glomerular filtration rate [eGFR] less than 60 mL/min)
4. Currently participating in a clinical trial of another investigational drug, or have participated 30 days prior to the start of the screening period or 5 half-lives from the most recent dose of the investigational drug, whichever is longer.
5. History of alcohol or drug abuse within 6 months prior to the start of the screening period
6. Patient has received combination therapy with edaravone and sodium phenylbutyrate/taurursodiol within 30 days prior to the start of the screening period.
7. History of breast cancer (regardless of remission status) or history of breast cancer in a first-degree relative

amyotrophic lateral sclerosis

Subjects will receive oral BID of either placebo or active drug during the 24-week treatment period.
For the optional long-term treatment period of 28 weeks, all subjects will receive 250 mg of PTC857 oral BID. For subjects who do not proceed to the long-term study, treatment will be discontinued and a follow-up telephone consultation will be conducted 4 weeks (Allowance 3 days) after the last dose of study drug.

amyotrophic lateral sclerosis ALS

The primary endpoint is the change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) in the Intent-To-Treat 1 (ITT1) Analysis Set after 24 weeks of treatment.

1. Safety and tolerability of PTC857
2. Subject's respiratory function
3. Subject's motor/limb function and medullary function
4. Neuropsychological functioning of subjects
5. Subject survival period
6. Subject's quality of life
7. Pharmacokinetics of PTC857

July. 18, 2023

No

Argentina/Australia/Belgium/Brazil/Canada/Czech Republic/France/Germany/Italy/Ireland/Mexico/Netherlands/Norway/Poland/Spain/Sweden/US