Long Term Study of Livmarli

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Mirum Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Cardiovascular Diseases [C14], Diseases [C] - Digestive System Diseases [C06]

Trial duration

25 Sep 2025 → ongoing

Decision date (initial)

2025-11-18

Transition trial

No

Low-intervention

Yes

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Mirum Pharmaceuticals, Inc.

External identifiers

EU CT number

2024-516804-40-00

ClinicalTrials.gov

NCT07290257

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Efficacy, Safety

The key objectives of this low-intervention clinical study in participants with ALGS who are prescribed Livmarli are:
1. To evaluate tolerability and long-term safety (including possible liver toxicity)
2. To evaluate long-term efficacy
The key objectives of this low-intervention clinical study in participants with PFIC who are prescribed Livmarli are:
1. To evaluate tolerability and long-term safety (including possible liver toxicity)
2. To evaluate the impact of Livmarli on long-term clinical outcome events (surgical biliary diversion [SBD],liver transplant [LT], portal hypertension, complications of liver cirrhosis, liver carcinoma, disease progression, liver decompensation, liver-related mortality, and all-cause mortality)
3. To evaluate LT waitlist status
4. To evaluate growth and development
5. To obtain biomarkers of cholestasis, liver fibrosis (APRI and PELD), and liver function
6. To monitor pruritus severity over time
7. To evaluate long-term safety of chronic exposure to propylene glycol (PG)

Conditions and MedDRA coding

Progressive Familial Intrahepatic Cholestasis

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Understand and execute an Informed consent and assent (as applicable)
2. A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis (Saleh et al. 2016) or a clinically and/or genetically confirmed PFIC diagnosis (Hassan and Hertel 2022)
3. For the ALGS primary cohort: Initiation of Livmarli at the time of study entry
4. For the ALGS supplemental cohort: Actively using Livmarli prior to study entry
5. For participants with ALGS, ≥2 months of age at Day 1
6. For participants with PFIC, ≥3 months of age at Day 1
7. For participants with PFIC: Prescribed Livmarli at the time of study entry or prior to study entry

Exclusion criteria 6

1. History of LT
2. Any Livmarli contraindications (as per SmPC)
3. Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study
4. Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded access programs is acceptable)
5. Baseline data before start of treatment of Livmarli are unavailable for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameters
6. Received another IBAT inhibitor within 7 days before the first dose of Livmarli

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. Safety • AEs • The number and proportion of participants who experience an AE • Change in liver function tests from Baseline • Change in FSV levels and INR (as an estimate of vitamin K) from Baseline • For participants with PFIC PG toxicity will be monitored
2. Efficacy • Change in pruritus severity from Baseline as measured by Clinician Scratch Scale (CSS) score • Change in serum bile acids (sBA) levels from Baseline • Frequency in the use of concomitant medications for the treatment of underlying liver disease, treatment of pruritus, or treatment of cholestasis • Frequency and timing of long-term clinical outcomes
3. Tolerability: Tolerability of starting dose and dose escalation will be assessed by summarizing AEs and/or any other safety information (as available) on starting dose and during dose escalation phase. The cases of dose reduction, treatment interruption, and treatment discontinuation due to AEs/poor tolerability will be also summarized over the whole treatment period.
4. Growth: Growth, including height/length and weight, will be assessed as an endpoint. The endpoint will be assessed as change from Baseline in height/length and weight (absolute values and z-scores).
5. Overdose, Dosing Errors, Misuse, and Abuse Frequencies of overdose and dosing errors/medication errors will be calculated.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Mirum Pharmaceuticals Inc.

Sponsor organisation

Mirum Pharmaceuticals Inc.

Address

989 East Hillsdale Boulevard

City

Foster City

Postcode

94404-2113

Country

United States

Scientific contact point

Organisation

Mirum Pharmaceuticals Inc.

Contact name

Mirum Clinical Lead

Public contact point

Organisation

Mirum Pharmaceuticals Inc.

Contact name

Mirum Clinical Lead

Third parties 5

Locations

6 EU/EEA countries · 14 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 238 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

6 submissions — initial application plus substantial / non-substantial modifications