Alagille SyndromeNCT04530994
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
A study testing maralixibat in people with Alagille Syndrome, run by Mirum Pharmaceuticals, Inc. at 14 sites in United States.
Trial summary
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303. Read more about this study
- Who is it for?
- Ages 12 months and older
- Conditions the study is for
- Alagille Syndrome
- What is being tested?
- maralixibat
- How many people
- Not given
- Where
- 14 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 months and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
- Clinical and/or genetic diagnosis of ALGS
- Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
- Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program
- No other therapeutic options or access to any ongoing ALGS clinical trials
Who cannot join
- Diagnosis with a cholestatic liver disease other than ALGS
- Female who is pregnant or breastfeeding
- Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program
- Past medical history of compensated or decompensated cirrhosis
- Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, 30329
University of Virginia
Charlottesville, Virginia, 22903
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611
Baylor College of Medicine
Houston, Texas, 77030
University of California, Los Angeles
Los Angeles, California, 90095
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota
Minneapolis, Minnesota, 55455
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
The Children's Hospital at Montefiore
New York, New York, 10467
University of Oklahoma
Oklahoma City, Oklahoma, 73104
Advent Health
Orlando, Florida, 32804
Stanford University
Redwood City, California, 94063
University of California San Francisco
San Francisco, California, 94158
Georgetown University
Washington D.C., District of Columbia, 20007
About this study
Official title: A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303.
Read the detailed description
The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.
- Conditions
- Alagille Syndrome
- Being tested
- maralixibat
- Keywords
- Liver Disease, ALGS, Maralixibat, Bile Duct Diseases, Digestive System Diseases, Biliary Tract Diseases, Cholestasis, Pruritis
- Medical subject headings
- Alagille Syndrome, Liver Diseases, Bile Duct Diseases, Digestive System Diseases, Biliary Tract Diseases, Cholestasis, Pruritus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
maralixibatDrug
Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alagille SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Clinigen, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04530994
- Sponsor's protocol ID
- MRX-EAP
- Status last verified
- Oct 2021
- First submitted
- 25 Aug 2020
- Last updated
- 20 Oct 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome
- ClinicalTrials.gov ID
- NCT04530994
- Acronym / sponsor ID
- MRX-EAP
- Conditions
- Alagille Syndrome
- Keywords
- Liver Disease, ALGS, Maralixibat, Bile Duct Diseases, Digestive System Diseases, Biliary Tract Diseases, Cholestasis, Pruritis
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Clinigen, Inc.
- First posted
- 28 Aug 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303.
Detailed description
The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.
Study design
Who may qualify
- Ages
- 12 months and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
- Clinical and/or genetic diagnosis of ALGS
- Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
- Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program
- No other therapeutic options or access to any ongoing ALGS clinical trials
Key exclusion criteria (5 listed)
- Diagnosis with a cholestatic liver disease other than ALGS
- Female who is pregnant or breastfeeding
- Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program
- Past medical history of compensated or decompensated cirrhosis
- Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| maralixibatMaralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2021.
- Genetics Home Reference - Alagille syndromeLink provided by the sponsor.
- US FDA ResourcesLink provided by the sponsor.
- Mirum Pharmaceuticals homepageLink provided by the sponsor.
- Mirum webpage specific to ALGS EAPLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing maralixibat in people with Alagille Syndrome, run by Mirum Pharmaceuticals, Inc. at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2021.