Phase 3
N=5
A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)
Progressive Familial Intrahepatic Cholestasis (PFIC)
Bottom Line
View on ClinicalTrials.gov: NCT05543187 ↗Enrolled (actual)
5
Serious AEs
0.0%
Results posted
Sep 2024
Primary outcome: Primary: Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26 — -1.514; -0.202 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- TAK-625 (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Takeda
- Primary completion
- Sep 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26 |
-1.514; -0.202 | — |
| SECONDARY Change in the Average Morning ItchRO (Obs) Frequency Score Between Baseline and the Average of Week 15 Through Week 26 |
-0.740; -0.735 | — |
| SECONDARY Change From Baseline in Total Serum Bile Acid (sBA) Levels to Week 26 |
-149.900; -18.250 | — |
| SECONDARY Percentage of Participants (Responders) Who Experienced an sBA Control From Baseline Through Week 26 |
33.3; 0.0 | — |
| SECONDARY Change in the ItchRO (Obs) Weekly Average Severity Between Baseline and the Average of Week 15 Through Week 26 |
-1.629; -0.254 | — |
Summary
The main aim of the study is to check if TAK-625 improves symptoms of Progressive Familial Intrahepatic Cholestasis (PFIC), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time. This will help the study sponsor (Takeda) to work out the best dose to give people in the future.
The participants will be treated with TAK-625 for up to the end of study (about 34 months).
Participants will visit their study clinic 15 times from the start of study. After 15 times visits, participants will visit their study clinic every 12 weeks up to the end of study.
Eligibility Criteria
Inclusion Criteria
- The participant is Japanese male or female with a body weight >=3.0 kg and who is >=1 month of age at the time of informed consent.
- The participant has a cholestasis as manifested by total serum bile acid (sBA) >=3^ upper limit of the normal range (ULN) (applies to the primary cohort only).
- The participant has an average morning ItchRO (Obs) score >=1.5 during 4 consecutive weeks of the screening period, leading to the baseline visit (Week 0/Visit 2). Since it is difficult to evaluate pruritus in infants, participants 6 months*) pruritus in addition to biochemical abnormalities and/or pathological evidence of progressive liver disease. (* = 1.5, and/or albumin 15^ ULN at screening.
- The participant has other liver disease.
- The participant has any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (eg, inflammatory bowel disease), per investigator discretion.
- The participant has a possible malignant liver mass in imaging, including screening ultrasound.
- The participant has received bile acid, lipid binding resins or ileal bile acid transporter (IBAT) inhibitors within 28 days prior to screening and throughout the trial.
- The participant who has received sodium phenylbutyrate for less than 6 months at the initiation of screening.
Data sourced from ClinicalTrials.gov (NCT05543187). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.