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Bile Acid Sequestrant

LUM001 (Maralixibat) for Progressive Familial Intrahepatic Cholestasis (INDIGO Trial)

Phase 2
Waitlist Available
Research Sponsored by Mirum Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline (day 0) to week 13
Awards & highlights
All Individual Drugs Already Approved
No Placebo-Only Group

Summary

This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.

Eligible Conditions
  • Progressive Familial Intrahepatic Cholestasis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (day 0) to week 13
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 0) to week 13 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)

Side effects data

From 2020 Phase 2 trial • 19 Patients • NCT02047318
58%
Abdominal pain
58%
Diarrhoea
58%
Upper respiratory tract infection
53%
Cough
42%
Pyrexia
42%
Nasopharyngitis
37%
Oropharyngeal pain
32%
Headache
26%
Ear infection
26%
Vomiting
21%
Ear pain
21%
Tonsillitis
21%
Wound haemorrhage
21%
Pain in extremity
21%
Lethargy
16%
Fatigue
16%
Varicella
16%
Viral upper respiratory tract infection
16%
Contusion
16%
Scratch
16%
International normalised ratio increased
16%
Epistaxis
11%
Seasonal allergy
11%
Abdominal pain upper
11%
Constipation
11%
Dental caries
11%
Haematochezia
11%
Chest pain
11%
Vitamin D decreased
11%
Vitamin D deficiency
11%
Arthralgia
11%
Dizziness
11%
Nasal congestion
11%
Pruritus
11%
Rash maculo-papular
11%
Nausea
5%
Somnolence
5%
Post procedural fever
5%
Eczema
5%
Hypotension
5%
Taste disorder
5%
Hypoxia
5%
Thrombosis
5%
Intracardiac pressure increased
5%
Alanine aminotransferase increased
5%
Bradycardia
5%
Dehydration
5%
Pericardial effusion
5%
Right ventricular failure
5%
Ear haemorrhage
5%
Lower respiratory tract infection
5%
Pathological fracture
5%
Forearm fracture
5%
Humerus fracture
5%
Procedural haemorrhage
5%
Medical device change
5%
Anaemia
5%
Lymphadenopathy
5%
Ear pruritus
5%
Abdominal discomfort
5%
Abdominal distension
5%
Abnormal faeces
5%
Chapped lips
5%
Faeces discoloured
5%
Flatulence
5%
Frequent bowel movements
5%
Gingival bleeding
5%
Inguinal hernia
5%
Lip haemorrhage
5%
Malabsorption
5%
Melaena
5%
Mouth haemorrhage
5%
Teething
5%
Feeling abnormal
5%
Influenza like illness
5%
Biliary tract disorder
5%
Hepatic lesion
5%
Jaundice
5%
Gastroenteritis
5%
Gastrointestinal infection
5%
Hepatitis infectious mononucleosis
5%
Oral herpes
5%
Otitis media
5%
Anaemia postoperative
5%
Clavicle fracture
5%
Concussion
5%
Drain site complication
5%
Fall
5%
Hand fracture
5%
Head injury
5%
Limb crushing injury
5%
Limb injury
5%
Procedural pain
5%
Skin abrasion
5%
Stoma site erythema
5%
Stoma site pain
5%
Stress fracture
5%
Bilirubin urine present
5%
Blood bilirubin increased
5%
Blood parathyroid hormone increased
5%
Body temperature increased
5%
Urobilinogen urine increased
5%
Vitamin E decreased
5%
Bone metabolism disorder
5%
Bone pain
5%
Disturbance in attention
5%
Poor quality sleep
5%
Seizure
5%
Device occlusion
5%
Breath holding
5%
Enuresis
5%
Sleep disorder
5%
Pollakiuria
5%
Amenorrhoea
5%
Fibrinous bronchitis
5%
Pleural effusion
5%
Pulmonary hypertension
5%
Rhinorrhoea
5%
Sneezing
5%
Acne
5%
Drug eruption
5%
Dry skin
5%
Rash
5%
Skin discolouration
5%
Skin lesion
5%
Xanthelasma
5%
Hypertension
5%
Dysuria
5%
Urinary incontinence
5%
Gastrointestinal haemorrhage
5%
Post procedural haemorrhage
5%
Gastrointestinal stoma output decreased
5%
Feeling hot
5%
Clostridium difficile infection
5%
Epstein-Barr virus infection
5%
Impetigo
5%
Joint injury
5%
Vaccination complication
5%
Back pain
5%
Restlessness
5%
Skin ulcer
5%
Urticaria
100%
80%
60%
40%
20%
0%
Study treatment Arm
LUM001 MRX Treatment
14 Microgram Per Kilogram Per Day (mcg/kg/Day)
35 Microgram Per Kilogram Per Day (mcg/kg/Day)
70 Microgram Per Kilogram Per Day (mcg/kg/Day)
140 Microgram Per Kilogram Per Day (mcg/kg/Day)
280 Microgram Per Kilogram Per Day (mcg/kg/Day)
560 Microgram Per Kilogram Per Day (mcg/kg/Day)
420 Microgram Per Kilogram Per Day (mcg/kg/Day)

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: LUM001 (Maralixibat)Experimental Treatment1 Intervention
Participants will receive LUM001, also known as Maralixibat (MRX) twice a day (BID).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Maralixibat
FDA approved

Find a Location

Who is running the clinical trial?

Mirum Pharmaceuticals, Inc.Lead Sponsor
31 Previous Clinical Trials
101,704 Total Patients Enrolled
Study DirectorStudy DirectorMirum
1,280 Previous Clinical Trials
500,667 Total Patients Enrolled
~3 spots leftby Dec 2025
Unbiased ResultsWe believe in providing patients with all the options.
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