Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
LUM001 · 8 trials · 5 indications
This primacy efficacy endpoint is the mean change from MRX baseline to week 48 in fasting sBA levels.
Pruritus was assessed using Itch report outcome measure (ItchRO\[Obs\]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.
The primary efficacy endpoint of this study was the mean change from Week 18 to Week 22 (the RWD period) of fasting sBA levels in participants who had a reduction in sBA ≥50% from baseline to Week 12 or Week 18 (Modified Intent-to-Treat \[MITT\] Population). Five participants in the MRX group and 10 participants in the placebo group met the prespecified sBA reduction criteria.
An Adverse Event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered to be related to the investigational drug product. TEAEs were AEs with a start date on or after the first dose of investigational product and started prior to the last dose of investigational product plus 14 days.
Serum bile acid levels were evaluated using blood samples collected.
The primary endpoint of this study was the mean change from MRX baseline to Week 48 in fasting sBA level.
Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.
Pruritus was assessed using ItchRO measure, administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). (ItchRO) scores ranged from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit.
| Arm | Type | Description |
|---|---|---|
| LUM001 (Maralixibat) | EXPERIMENTAL | Participant will receive LUM001 also known as Maralixibat (MRX) administered orally once per day. |
| LUM001 | EXPERIMENTAL | LUM001 for oral administration |
| Placebo | PLACEBO_COMPARATOR | Placebo administered orally once each day |
| LUM001 and Ursodeoxycholic Acid (UDCA) | EXPERIMENTAL | Administered orally once daily |
| Placebo and Ursodeoxycholic Acid (UDCA) | PLACEBO_COMPARATOR | Administered orally once daily |
| Name | Type | Description |
|---|---|---|
| LUM001 (Maralixibat) | DRUG | Dosing of LUM001 also known as Maralixibat (MRX) with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the participant and up to a maximum daily dose of 280 micrograms per kilogram (mcg/kg). |
| LUM001 | DRUG | LUM001 administered orally |
| Placebo | DRUG | Placebo administered orally |
| Ursodeoxycholic Acid | DRUG | - |
Inclusion Criteria: 1. Male or female, 12 months to 18 years of age. 2. Competent to provide informed consent and assent (per institutional review board/Ethics Committee \[IRB/EC\]), as appropriate. 3. Completed participation in the LUM001-301 protocol. 4. Females of childbearing potential must hav...
LUM001 is an investigational small molecule being developed for cholestatic liver diseases, including Alagille Syndrome, Primary Sclerosing Cholangitis (PSC), Primary Biliary Cholangitis (PBC), and Progressive Familial Intrahepatic Cholestasis (PFIC). It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
LUM001 is being developed by Mirum Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker MIRM. The company is focused on rare disease therapies, and LUM001 is one of its investigational drug candidates in clinical development.
LUM001 is in Phase 2 clinical development. It has completed five clinical trials, all of which are Phase 2 studies. The drug remains investigational and has not received FDA approval or any other regulatory approval for commercial use.
LUM001 has been studied in five completed Phase 2 clinical trials. These include NCT01903460 in Alagille Syndrome, NCT01904058 in Primary Biliary Cirrhosis, NCT02057718 in Progressive Familial Intrahepatic Cholestasis, and NCT02160782 in Alagille Syndrome. The trials enrolled a total of 141 participants across multiple countries.
Yes, LUM001 is also known as maralixibat. In clinical trial records, the drug is referred to as LUM001 (Maralixibat), indicating that these names refer to the same investigational compound being developed by Mirum Pharmaceuticals for cholestatic liver diseases.