HGPS, Progeria
Conditions
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.
Interventions
DRUGLonafarnib
Farnesyl transferase inhibitor
Sponsors
Eiger BioPharmaceuticals
Eligibility
Sex/Gender
ALL
Age
12 Months to No maximum
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.
Outcome results
None listed