Progeria Clinical Trial
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
NCT number | NCT03895528 |
Other study ID # | EIG-EAP-LNF-001 |
Secondary ID | |
Status | Approved for marketing |
Phase | |
First received | |
Last updated |
Verified date | April 2021 |
Source | Eiger BioPharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Expanded Access |
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.
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. |
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Eiger BioPharmaceuticals |
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
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 |