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
1-732-943-8138
atansy@ptcbio.com
Phillips Robert
Worldwide Clinical Trials Japan K.K.
2-15-1, Konan, Minato-ku, Tokyo Shinagawa Intercity, Tower A, Level 28,
+81-3-6717-4360
robert.phillips@worldwide.com
Recruiting
Sept. 29, 2023
258
Interventional
randomized controlled trial
double blind
placebo control
parallel assignment
treatment purpose
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
18age old over
80age old under
Both
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
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. Change from baseline in ALSFRS-R in the Intent-to-Treat 2(ITT2) Analysis Set after 24 weeks of treatment
2. Safety and tolerability of PTC857 as measured by the severity and number of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events(TESAEs), and change in clinical laboratory tests, physical examination, vital
signs, Columbia-Suicide Severity Rating Scale(C-SSRS), and 12-lead electrocardiograms (ECGs) during the Treatment Period
3. Change from baseline in respiratory function as assessed by pulmonary function tests (PFTs) after 24 weeks of treatment
4. Change from baseline in motor/limb and bulbar function as assessed by the Modified Norris Scale after 24 weeks of treatment
5. Change from baseline in neuropsychological function as assessed by the ALS Cognitive Behavioral Screen (ALS CBS) after 24 weeks of treatment
6. Survival as assessed by rate of and length of time to needing respiratory support/intubation and/or death
7. Survival and functional change as assessed by the Combined Assessment of Function and Survival (CAFS) after 24 weeks of treatment
8. Quality of life as assessed by Amyotrophic Lateral Sclerosis Assessment Questionnaire(ALSAQ-40)after 24 weeks of treatment
9. Plasma PK and cerebrospinal fluid (CSF) exposure of PTC857
PTC Therapeutics, Inc.
Asahikawa Medical Center Institutional Review Board
