Philadelphia, PANCT06193928Now EnrollingIRB Ready

Alagille Syndrome Clinical Trial in Philadelphia, PA

Access cutting-edge alagille syndrome treatment through this clinical trial at a research site in Philadelphia. Study-provided care at no cost to qualified participants.

Sponsored by Mirum Pharmaceuticals, Inc.

Quick Self-Assessment

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Expert Care in Philadelphia

Access alagille syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related alagille syndrome treatment provided free

Apply for This Philadelphia Location

Check if you qualify for this alagille syndrome clinical trial in Philadelphia, PA

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Philadelphia

    Convenient for PA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Philadelphia site if eligible
  4. 4Begin participation

About This Alagille Syndrome Study in Philadelphia

The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.

Sponsor: Mirum Pharmaceuticals, Inc.

Who Can Participate

Inclusion Criteria

A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
Participant prescribed Livmarli

Exclusion Criteria

Refusal to provide informed consent/assent (if required by the local IRB)
Previously or currently on Livmarli through participation in a clinical study or expanded access program
Participants who have previously received an SBD or LT
Any condition or abnormalities that, in the opinion of the investigator, may interfere with the participant participating in or completing the study
Participants who have received an investigational drug within 30 days of the first dose of Livmarli

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Philadelphia?

Yes, this clinical trial (NCT06193928) has an active research site in Philadelphia, PA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Alagille Syndrome Treatment Options in Philadelphia, PA

If you're searching for alagille syndrome treatment options in Philadelphia, PA, this clinical trial (NCT06193928) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Philadelphia research site is actively enrolling participants for this clinical trial. You'll receive care from experienced alagille syndrome specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all alagille syndrome clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Philadelphia, PA