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Clinical Trial Details — Status: Approved for marketing

Administrative data

NCT number NCT03895528
Other study ID # EIG-EAP-LNF-001
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information

Verified date April 2021
Source Eiger BioPharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial 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.


Recruitment information / eligibility

Status Approved for marketing
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers
Gender All
Age group 12 Months and older
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Lonafarnib
Farnesyl transferase inhibitor

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Eiger BioPharmaceuticals

References & Publications (1)

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. — View Citation

See also
  Status Clinical Trial Phase
Completed NCT00094393 - Clinical Studies of Progeria
Completed NCT00425607 - Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria Phase 2
Completed NCT00879034 - A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria Phase 2
Enrolling by invitation NCT02579044 - Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria Phase 1/Phase 2
Active, not recruiting NCT00916747 - Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria Phase 2