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

Primary Purpose

Progeria, HGPS

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
Lonafarnib
Sponsored by
Eiger BioPharmaceuticals
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Progeria

Eligibility Criteria

12 Months - undefined (Child, Adult, Older Adult)All Sexes

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.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    March 27, 2019
    Last Updated
    April 13, 2021
    Sponsor
    Eiger BioPharmaceuticals
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03895528
    Brief Title
    Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy
    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
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    April 2021
    Overall Recruitment Status
    Approved for marketing
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Eiger BioPharmaceuticals

    4. Oversight

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Progeria, HGPS

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Lonafarnib
    Other Intervention Name(s)
    FTI
    Intervention Description
    Farnesyl transferase inhibitor

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    12 Months
    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.

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    29710166
    Citation
    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.
    Results Reference
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    Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy

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