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Also known as Maralixibat chloride, Maralixibat, Livmarli Oral Solution
TAK-625 · 2 trials · 2 indications
Change in fasting sBA levels from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22- observed value at Week 18.
The ItchRO (Obs) scale measures severity of pruritus. The score on ItchRO (Obs) scale ranged from 0 to 4, where 0=None observed or reported, 1=Mild, 2=Moderate, 3=Severe, 4=Very severe. A higher score indicated more severe pruritus. Average baseline morning ItchRO (Obs) scores were calculated as sum of the morning scores divided by number of morning scores for the 4-week (28 days) time periods (that is \[i.e.\], Day -28 to Day -1). Average morning ItchRO (Obs) scores Week 15 through Week 26 were calculated as the sum of the morning scores divided by the number of morning scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1).
| Arm | Type | Description |
|---|---|---|
| TAK-625 | EXPERIMENTAL | TAK-625 200 mcg per kilogram, orally, once daily for 1 week. After that, TAK-625 400 mcg per kilogram, orally, once daily after Week 1. |
| TAK-625, Primary cohort | EXPERIMENTAL | TAK-625 orally, twice daily (BID) for 4 weeks as Dose Escalation Period. The dose in Dose Escalation Period will be increased weekly, 150 mcg/kilograms (kg), 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg. After Dose Escalation Period, TAK-625 600 mcg/kg (or maximum tolerated dose \[MTD\]), orally, BID up to study completion. |
| TAK-625, Supplemental cohort | EXPERIMENTAL | TAK-625 orally, twice daily (BID) for 4 weeks as Dose Escalation Period. The dose in Dose Escalation Period will be increased weekly, 150 mcg/kilograms (kg), 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg. After Dose Escalation Period, TAK-625 600 mcg/kg (or maximum tolerated dose \[MTD\]), orally, BID up to study completion. |
| Name | Type | Description |
|---|---|---|
| TAK-625 | DRUG | TAK-625 200 mcg or 400 mcg per kilogram, orally, once daily |
Inclusion Criteria: 1. The participant is Japanese male or female with a body weight \>=3.0 kilograms (kg) and who is \>=1 month of age at the time of informed consent. 2. The participant is diagnosed with ALGS. 3. The participant has one or more of the following evidences of cholestasis: 1. To...
TAK-625 is an investigational small molecule being developed for the treatment of Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC), both rare genetic liver diseases. It is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.
TAK-625 is being developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company listed on the New York Stock Exchange under the ticker symbol TAK. The company is conducting clinical trials for this investigational drug in Japan.
TAK-625 is in Phase 3 clinical development for both Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). Two Phase 3 trials have been completed, but the drug remains investigational and has not received regulatory approval.
TAK-625 has been studied in two completed Phase 3 trials: NCT05543174 for Alagille Syndrome (ALGS) with 7 participants, and NCT05543187 for Progressive Familial Intrahepatic Cholestasis (PFIC) with 5 participants. Both trials were conducted in Japan and enrolled patients as young as 1 month old.
No, TAK-625 is not the same as maralixibat. TAK-625 is a distinct investigational small molecule developed by Takeda for Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis. Maralixibat is a different drug with its own mechanism and developer.