Apraglutide Open-Label Phase 3 Extension Trial

JRCT ID: jRCT2031220255

Registered date:08/08/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: short bowel syndrome and intestinal failure (SBS-IF)
• Date of first enrollment: 13/07/2022
• Target sample size: 12
• Countries of recruitment: Argentina,Japan,Belgium,Japan,Czech Republic,Japan,Denmark,Japan,France,Japan,Germany,Japan,Israel,Japan,Italy,Japan,Norway,Japan,Poland,Japan,South Korea,Japan,Spain,Japan,Sweden,Japan,Taiwan,Japan,UK,Japan,USA,Japan
• Study type: Interventional
• Intervention(s): Apraglutide 2.5 mg or 5 mg SC once weekly

Outcome(s)

Primary Outcome

Adverse events (AEs; system organ class, frequency and severity)
Occurrence of clinically relevant AEs of special interest (AESIs)
Clinical chemistry, hematology, hemostasis and urinalysis
Occurrence of clinically relevant changes in vital signs (systolic and diastolic blood pressure, heart rate)
Occurrence of clinically relevant changes in electrocardiogram (ECG; intervals and rhythm)

Secondary Outcome

Change from baseline in PS volume at Week 104
Change from baseline in PS frequency at Week 104
Change from baseline in PS composition at Week 104
Change from baseline in PS infusion time at Week 104
Percentage of subjects reaching enteral autonomy by Week 104
Change from baseline in body weight at Week 104
Change from baseline on the Pittsburgh Sleep Quality Index (PSQI) at Week 104
Change from baseline on the Patient Global Impression of Change (PGIC) at Week 104
Change from baseline on the Patient Global Impression of Severity (PGIS) at Week 104

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Males and females with a diagnosis of SBS-IF secondary to surgical resection of the small intestine, with CIC or stoma, who have completed parent trials TA799-007 or TA799-013 and
a. Did not meet any stopping criteria
b. Received a minimum of 70% of the planned doses in the trial (unless an AE precluded the subject from meeting this percentage; in this case, the Investigator will decide if the subject will benefit from enrolling in the trial)
c. Complete the last two scheduled visits of the parent trial. Subjects who were forced to withdraw from TA799-007 for logistical reasons not related to the efficacy or safety of apraglutide (e.g., hospitalization for a car accident, COVID-19 pandemic, or emergency surgery) which resulted in several consecutive missed doses may be eligible to participate in this trial upon approval by the Medical Monitor
2. Able to give informed consent and agree to follow the details of participation as outlined in this protocol
3. Women of childbearing potential must agree to use a highly effective method of contraception during the trial and for 4 weeks after the EOT visit. Such methods include combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable); intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner. To be considered sterilized or infertile, females must have undergone surgical sterilization (bilateral tubectomy, hysterectomy or bilateral ovariectomy) or be post-menopausal (defined as at least 12 months amenorrhea without an alternative medical cause, may be confirmed with follicle-stimulating hormone [FSH] test in case of doubt). Women who do not engage in heterosexual intercourse will be allowed to join the trial without contraception following a thorough discussion with the Investigator to determine if this is feasible for the subject. The following methods are not considered acceptable methods of contraception: calendar, ovulation, symptothermal, post-ovulation methods, withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method
4.Male subjects with a female partner of childbearing potential must commit to practice methods of contraception and abstain from sperm donation during the trial and for 2 weeks after the EOT Visit. Nevertheless, if their partners are women of childbearing potential, they must agree to practice contraception and use a highly effective method of contraception during the trial and for 4 weeks after the EOT visit. Such methods include combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable); intrauterine device; intrauterine hormone-releasing system, bilateral tubal occlusion

Exclude criteria

1. Subject not capable of understanding or not willing to adhere to the trial visit schedules and other protocol requirements
2. Any other reason judged not eligible by the Investigator

Related Information

• Primary Sponsor: Akinori Kashiyama
• Secondary ID(s): NCT05018286,2020-005513-41