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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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26 April 2021 |
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Main ID: |
NCT03895528 |
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Date of registration:
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27/03/2019 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy
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Scientific title:
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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 |
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Date of first enrolment:
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June 8, 2020 |
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Target sample size:
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Recruitment status: |
Approved for marketing |
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URL:
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https://clinicaltrials.gov/show/NCT03895528 |
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Study type:
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Expanded Access |
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Study design:
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Phase:
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Key inclusion & exclusion criteria
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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.
Age minimum:
12 Months
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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HGPS
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Progeria
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Intervention(s)
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Drug: Lonafarnib
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Secondary ID(s)
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EIG-EAP-LNF-001
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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