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

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ClinicalTrials.gov Identifier: NCT03895528
Expanded Access Status : Approved for marketing
First Posted : March 29, 2019
Last Update Posted : April 15, 2021
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Progeria HGPS Drug: Lonafarnib

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: 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

Intervention Details:
  • Drug: Lonafarnib
    Farnesyl transferase inhibitor
    Other Name: FTI

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

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.

No Contacts or Locations Provided
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Responsible Party: Eiger BioPharmaceuticals
ClinicalTrials.gov Identifier: NCT03895528    
Other Study ID Numbers: EIG-EAP-LNF-001
First Posted: March 29, 2019    Key Record Dates
Last Update Posted: April 15, 2021
Last Verified: April 2021
Additional relevant MeSH terms:
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Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action