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

NCT03895528 | Unknown

• 1 other identifier: EIG-EAP-LNF-001
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Progeria; HGPS

Interventions

Lonafarnib DRUG

Farnesyl transferase inhibitor

Also known as: FTI

Eligibility Criteria

• Age: 12 Months+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Eiger BioPharmaceuticals: lead

Related Publications (1)

• Gordon LB, Shappell H, Massaro J, D'Agostino RB Sr, Brazier J, Campbell SE, Kleinman ME, Kieran MW. Association of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome. JAMA. 2018 Apr 24;319(16):1687-1695. doi: 10.1001/jama.2018.3264.

  PMID: 29710166 BACKGROUND

MeSH Terms

Conditions

Progeria

Interventions

lonafarnib

Condition Hierarchy (Ancestors)

Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 27, 2019
• First Posted: March 29, 2019
• Last Updated: April 15, 2021

Record last verified: 2021-04