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Trial record 5 of 8 for:    progeria
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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 : Available
First Posted : March 29, 2019
Last Update Posted : July 17, 2019
Sponsor:
Information provided by (Responsible Party):
Eiger BioPharmaceuticals

Study Description
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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

Study Design
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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
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

Resource links provided by the National Library of Medicine


Interventions
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Intervention Details:
  • Drug: Lonafarnib
    Farnesyl transferase inhibitor
    Other Name: FTI
Eligibility Criteria
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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
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03895528


Contacts
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Contact: Senior Director, Clinical Affairs (650) 279-9845 ProgeriaMA@eigerbio.com

Sponsors and Collaborators
Eiger BioPharmaceuticals
More Information
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Publications:

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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: July 17, 2019
Last Verified: July 2019
Additional relevant MeSH terms:
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Progeria
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
 Lonafarnib
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action