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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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12 December 2020 |
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Main ID: |
NCT00425607 |
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Date of registration:
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22/01/2007 |
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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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Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria
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Scientific title:
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An Open Label Dose Adjusted Phase II Trial of the Oral Farnesyltransferase Inhibitor (FTI) Lonafarnib (SCH66336) for Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) and Progeroid Laminopathies |
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Date of first enrolment:
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May 2007 |
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Target sample size:
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29 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00425607 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Mark W Kieran, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Dana-Farber Cancer Institute, Children's Hospital Boston |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- All patients must have confirmatory mutational analysis showing G608G mutation in the
lamin A gene.
- Patients with progeroid laminopathies, showing clinical signs of Progeria but with
other confirmed mutations in LMNA will be eligible for therapy. This population will
be analyzed separately from those with the classical mutations.
- Patients must be willing and able to come to Boston for appropriate studies and
examinations approximately once every 4 months.
- Patients must have a minimum of one year of weight data available, with five data
points or more, each separated by one month or more over a one year period and
approval by the study team.
- APC (ANC + bands + monocytes = APC) > 1,000/ml, Platelets > 75,000/ml (transfusion
independent); Hemoglobin >9g/dl.
- creatinine less than or equal to 1.5 times normal for age or GFR > 70 ml/min/1.73m2.
- bilirubin less than or equal to 1.5 x upper limit of normal for age; SGPT (ALT) < and
SGOT (AST) < 5 x normal range for age.
- PT/PTT < 120% upper limit of normal OR PI approval.
- No overt renal, hepatic, pulmonary disease or immune dysfunction.
- Patients taking growth hormone when entering the study must have pretreatment weight
measures while on growth hormone which are specified above. In addition, patients must
remain on growth hormone treatment for the duration of the present clinical trial.
Patients entering the trial not on growth hormone must remain off of growth hormone
for the duration of their participation.
- Signed informed consent according to institutional guidelines must be obtained and
patient must begin therapy within twenty eight (28) days.
Exclusion Criteria:
- Patient must not be receiving any other experimental drug therapy.
- Patients must not be taking medications that significantly affect the metabolism of
lonafarnib.
- Subjects who have known or suspected hypersensitivity to any of the excipients
included in the formulation should not be treated.
- Patients must not be pregnant or breast-feeding. Female patients of childbearing
potential must have negative serum or urine pregnancy test. Male and female patients
of reproductive potential must agree to use a medically accepted form of birth control
while on study and up to 10 weeks after treatment. It is permissible for female
patients to take oral contraceptives or other hormonal methods while receiving
treatment with lonafarnib.
Age minimum:
1 Year
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Hutchinson-Gilford Syndrome
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Progeria
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Intervention(s)
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Drug: Lonafarnib
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Primary Outcome(s)
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Proportion of Participants With Successful Rate of Weight Gain
[Time Frame: Assessed at weeks 16, 32, 52, 68, 84 and 104]
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Secondary ID(s)
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07-01-007
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P05009
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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