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

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

Sponsors

Lead Sponsor: Eiger BioPharmaceuticals

Source Eiger BioPharmaceuticals
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.

Overall Status Approved for marketing
Study Type Expanded Access
Condition
Intervention

Intervention Type: Drug

Intervention Name: Lonafarnib

Description: Farnesyl transferase inhibitor

Other Name: FTI

Eligibility

Criteria:

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.

Gender:

All

Minimum Age:

12 Months

Maximum Age:

N/A

Verification Date

2021-04-01

Responsible Party

Type: Sponsor

Has Expanded Access No
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