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Trial record 2 of 718 for:    march

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) (MARCH-PFIC)

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ClinicalTrials.gov Identifier: NCT03905330
Recruitment Status : Recruiting
First Posted : April 5, 2019
Last Update Posted : August 7, 2019
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Brief Summary:
The purpose of this study is to determine if the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).

Condition or disease Intervention/treatment Phase
Progressive Familial Intrahepatic Cholestasis (PFIC) Drug: Maralixibat Other: Placebo Phase 3

Detailed Description:
This study will be conducted at multiple sites in North America, Europe, Asia, and South America.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFIC
Actual Study Start Date : July 9, 2019
Estimated Primary Completion Date : May 2020
Estimated Study Completion Date : May 2021


Arm Intervention/treatment
Experimental: Maralixibat
Participants will be randomized to Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) orally twice daily for 26 weeks.
Drug: Maralixibat
Maralixibat oral solution (up to 600 mcg/kg) orally twice daily for 26 weeks.
Other Name: Formerly LUM001 and SHP625

Placebo Comparator: Placebo
Participants will receive placebo matched to maralixibat oral solution twice daily for 26 weeks.
Other: Placebo
Placebo matching to maralixibat orally twice daily for 26 weeks.




Primary Outcome Measures :
  1. Treatment response as measured by the mean change in pruritus severity as assessed by the Observer rated Itch Reported Outcome (ItchRO [Obs]) [ Time Frame: Between Baseline and Week 15 through 26 ]
    Compare the average before midday (AM) Observer Itch Reported Outcome (ItchRO[Obs]) pruritus severity score between participants on active treatment versus placebo


Secondary Outcome Measures :
  1. Treatment response as measured by the mean change in pruritus frequency as assessed by the Observer rated Itch Reported Outcome (ItchRO [Obs]) [ Time Frame: Between Baseline and Week 15 through 26 ]
    Compare the average before midday (AM) Observer Itch Reported Outcome (ItchRO[Obs]) pruritus frequency score between participants on active treatment versus placebo

  2. Mean change in total serum Bile Acids [ Time Frame: Between Baseline and Week 26 ]
    Compare normalization or reduction in total serum Bile Acids between participants on active treatment versus placebo



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Ages Eligible for Study:   1 Year to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
  • Male or female subjects with a body weight ≥ 5 kg, who are ≥ 12 months and < 18 years of age at time of consent
  • Cholestasis as manifested by total serum Bile Acids ≥ 3× ULN
  • An average morning Itch Observer Reported Outcome (ItchRO[Obs]) score ≥ 1.5 during 4 consecutive weeks of the screening period, leading to the baseline visit (Visit 1)
  • Diagnosis of PFIC based on the following:

    • Chronic cholestasis as manifested by persistent (>6 months) pruritus, biochemical abnormalities or pathological evidence of progressive liver disease and
    • Primary Cohort: Subjects with genetic testing results consistent with biallelic disease-causing variation in ABCB11 (PFIC2), based on standard of care genotyping
    • Supplemental Cohort: i. Subjects with genetic testing results consistent with biallelic disease-causing variation in ATP8B1 (PFIC1), ABCB4 (PFIC3), or TJP2 (PFIC4), based on standard of care genotyping ii. Subjects with PFIC phenotype without a known mutation or with another known mutation not described above iii. Subjects with PFIC after internal or external biliary diversion surgery or for whom internal or external biliary diversion surgery was reversed

Key Exclusion Criteria:

  • Predicted complete absence of bile salt excretion pump (BSEP) function based on the type of ABCB11 mutation (PFIC2), as determined by a standard of care genotyping (applies to primary cohort only)
  • History of surgical disruption of the enterohepatic circulation (applies to primary cohort only)
  • Chronic diarrhea requiring intravenous fluid or nutritional intervention for the diarrhea and/or its sequelae at screening or during the 6 months prior to screening
  • Previous or planned liver transplant
  • Decompensated cirrhosis (international normalized ratio [INR] > 1.5, albumin < 30 g/L, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy)
  • ALT or total serum bilirubin (TSB) > 15× ULN at screening
  • Presence of other liver disease
  • Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per Investigator discretion
  • Liver mass on imaging, including screening ultrasound
  • Known diagnosis of human immunodeficiency virus (HIV) infection
  • Any prior cancer diagnosis (except for in situ carcinoma) within 5 years of the screening visit (Visit 0)
  • Administration of growth hormones at any time before or during the study
  • Previous use of an ileal bile acid transporter inhibitor (IBATi)
  • History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator or Sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the subject, or lead to nonadherence with the study protocol or inability to conduct the study procedures

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03905330


Contacts
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Contact: Clinical Trials Mirum +16506674085 clinicaltrials@mirumpharma.com
Contact: Medinfo Mirum medinfo@mirumpharma.com

Locations
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United States, Michigan
Michigan Medicine (University of Michigan) Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Frank DiPaola, MD         
United States, Pennsylvania
Children'S Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Robert H Squires, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Simon Horslen, MD         
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.

Additional Information:
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Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03905330     History of Changes
Other Study ID Numbers: MRX-502
2019-001211-22 ( EudraCT Number )
First Posted: April 5, 2019    Key Record Dates
Last Update Posted: August 7, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Mirum Pharmaceuticals, Inc.:
Cholestasis
Maralixibat
Mutation
PFIC
PFIC2
Bile Duct Diseases
Liver Diseases
Biliary Tract Diseases
Digestive System Diseases
Pediatric

Additional relevant MeSH terms:
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Cholestasis
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases