A Single-Dose Study to Assess the Food Effect on the Pharmacokinetics and Tolerability of Ataluren (PTC124) in Healthy Japanese Subjects

JRCT ID: jRCT2071220020

Registered date:24/06/2022

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Nonsense mutation Duchenne muscular dystrophy
• Date of first enrollment: 24/06/2022
• Target sample size: 12
• Study type: Interventional
• Intervention(s): To administrate a oral single dose of PTC124 1375 mg (125 mg 1 packet, 250 mg 1 packet, and 1000 mg 1 packet) on Day 1 and Day 5.

Outcome(s)

• Primary Outcome: PK parameters of ataluren including AUC0-t, AUC0-inf, Cmax, and Tmax and the relative ratios under fasted and fed conditions

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: <= 55age old
• Gender: Male

Include criteria

1. Males aged between 18 and 55 years old and have a body mass index (BMI) between 18.5 and 30.0 kg/m2.
2. Subjects must understand the nature of the study and must provide signed and dated written informed consent before the conduct of any study-related procedures.
3. Males 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 Screening through 30 days after the last dose of study drug. Males must also refrain from sperm donations during this time period. Males who are abstinent will not be required to use a contraceptive method unless they become sexually active. Males who have undergone a vasectomy are not required to use a contraceptive method, if at least 16 weeks after the procedure. Same-sex couples are not required to use contraception.
4. Willing and able to comply with the protocol.
5. Have not used tobacco products of any kind (eg, cigarettes, e-cigarettes, cigars, smokeless tobacco) for at least 2 weeks prior to the Screening Visit and are willing to abstain from these products through end of study.
6. Subject must be Japanese and have 2 Japanese biological parents and 4 Japanese grandparents as confirmed by interview.

Exclude criteria

1. Gastrointestinal disease (such as irritable bowel syndrome, inflammatory bowel disease, chronic gastritis, peptic ulcer disease, etc) that could affect the absorption of study drug.
2. History of gastric surgery, including Roux-en-Y gastric bypass surgery or an antrectomy with vagotomy, or gastrectomy.
3. History of fat malabsorption.
4. Dietary restrictions that preclude participation.
5. Inability to tolerate oral medication.
6. History of allergies or adverse reactions to ataluren or related compounds or to any excipients in the study drug formulation.
7. 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.
8. Alanine aminotransferase or aspartate aminotransferase laboratory values >1.5x the upper limit of normal (ULN).
9. Serum creatinine >_1.5xULN at Screening.
10. Any other clinically significant laboratory abnormality at the Screening Visit or prior to the administration of the first dose of study drug. In general, each laboratory value from Screening and baseline chemistry and hematology panels should fall within the limits of the normal laboratory reference range, unless deemed not clinically significant by the investigator. The investigator may repeat laboratory testing one time in the event of an out-of-range laboratory value to confirm eligibility.
11. Current participation in any other investigational drug study or participation within 30 days prior to Screening.
12. History of alcohol or drug abuse within the last 6 months prior to Screening, current evidence of substance dependence, or self-reported alcoholic intake >10 standard drinks/week.
13. QT with Fridericia's correction (QTcF) >_450 milliseconds.
14. Resting heart rate <_40 or >_100 beats per minute.
15. Resting blood pressure <90/40 mmHg or >150/95 mmHg at Screening.
16. Subject is, in the opinion of the investigator, unwilling or unable to adhere to the requirements of the study.
17. Major surgery within the 90 days prior to Screening.
18. Use of melatonin within 1 week prior to Screening and through discharge from clinic.
19. Positive test for HIV, syphilis, hepatitis B, or hepatitis C.
20. Positive screen for drugs with a high potential for abuse at Screening and check-in.
21. Blood donation of >_450 mL of blood within the 90 days prior to Screening.
22. Subjects taking prescription (IV aminoglycosides/IV Vancomycin) or nonprescription drugs (including vitamins, recreational drugs, and dietary or herbal supplements) within 7 days (or 14 days if the drug is a potential enzyme inducer) or 5 half-lives (whichever is longer) before the start of study intervention until completion of the Follow-Up Visit, unless, in the opinion of the investigator and PTC, the medication will not interfere with the study drug.

Related Information

• Primary Sponsor: Shunya KATO