Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy

April 13, 2021 updated by: Eiger BioPharmaceuticals

A Treatment IND (Investigational New Drug) Protocol for EAP (Expanded Access Program) for the Use of Lonafarnib in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) or Progeroid Laminopathy

This treatment IND protocol will allow patients with HGPS and progeroid laminopathies access to lonafarnib, the only compound shown to have an effect on the HGPS disease process resulting in improved outcomes (Gordon et al, 2018). There are no approved treatments for HGPS and progeroid laminopathies.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  • Clinical diagnosis of HGPS or progeroid laminopathy by qualified medical doctor (based on common phenotype as described in Gordon 2015 and Merideth 2008). Confirmation with genetic testing is preferred but not required.
  • Adequate hepatic function as defined by SGPT (ALT) and SGOT (AST) ≤ 5 times upper limit of normal range for age

Exclusion Criteria:

  • Taking medications or foods that are known to be moderate or strong inducers or inhibitors of CYP3A4 or sensitive CYP3A substrates; or if a patient is taking one of these drugs and cannot safely discontinue or take an alternative drug, the dose of the inhibitor/inducer must be adjusted per the treating physician
  • Taking digoxin, a P-gp substrate with a narrow therapeutic window.
  • Severe renal impairment (GFR < 30 mL/min/1.73m2).
  • Uncontrolled infection.
  • Presence of any active clinically relevant medical condition that in the opinion of the treating physician would preclude patient from safely participating in the program.
  • Pregnant or breast-feeding or plan to become pregnant while on therapy.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Eiger BioPharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

March 27, 2019

First Submitted That Met QC Criteria

March 27, 2019

First Posted (Actual)

March 29, 2019

Study Record Updates

Last Update Posted (Actual)

April 15, 2021

Last Update Submitted That Met QC Criteria

April 13, 2021

Last Verified

April 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EIG-EAP-LNF-001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Progeria

Clinical Trials on Lonafarnib

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Lebanon

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe