A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00879034
• Date of registration: 08/04/2009
• Prospective Registration: No
• Primary sponsor: Boston Children's Hospital
• Public title: A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
• Scientific title: A Phase II Pilot Study of Zoledronic Acid, Pravastatin, and Lonafarnib (SCH66336) for Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) and Progeroid Laminopathies
• Date of first enrolment: March 2009
• Target sample size: 5
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00879034
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Mark W Kieran, MD, PhD
• Affiliation: Dana-Farber Cancer Institute; Boston Children's Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

- Genetic Diagnosis: All patients must have confirmatory mutational analysis showing
mutation in the lamin A gene.

- Patients must display clinical signs of progeria as per the clinical trial team.

- Patients must be willing and able to come to Boston for appropriate studies and
examinations at initiation of study and at week 4 of study.

- Patient must have adequate organ and marrow function as defined by study parameters

Exclusion Criteria:

- Other than the drugs used in this protocol, other drugs targeted to treat Progeria are
excluded. Drugs to treat symptoms of Progeria are permitted.

- Patients must not be taking medications that significantly affect the metabolism of
lonafarnib at the time they start lonafarnib.

- Patient must have no uncontrolled infection.

- Subjects who have known or suspected hypersensitivity to any of the excipients
included in the formulation should not be treated.

- Patients must not be pregnancy of 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: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Hutchinson-Gilford Syndrome

Progeria

Intervention(s)

Drug: Lonafarnib

Drug: Zoledronic Acid

Drug: Pravastatin

Primary Outcome(s)

The Primary Objective of This Study is to Evaluate the Feasibility of Administering Intravenous Zoledronic Acid, Oral Pravastatin and Oral Lonafarnib, to Patients With Progeria for a Minimum of 4 Weeks [Time Frame: 4 weeks]

Secondary Outcome(s)

To Describe Any Acute and Chronic Toxicities Associated With Treating Progeria Patients With the Combination of Zoledronic Acid, Pravastatin and Lonafarnib [Time Frame: 4 weeks]

To Investigate Which Clinical and Laboratory Studies Are Needed to Monitor or Alter Therapy to Prevent Unacceptable Toxicity [Time Frame: 4 weeks]

To Assess the Pharmacokinetics of Lonafarnib in Patients With Progeria. [Time Frame: 4 weeks]

To Assay for the Inhibition of HDJ-2 Farnesylation in Peripheral Blood Leukocytes (PBL) [Time Frame: 4 weeks]

To Obtain Baseline Clinical and Laboratory Data so That Longer-term Measures of Efficacy Will be Achievable if Treatment Continues Beyond the 4-week Feasibility Study Period. [Time Frame: 4 weeks]

Secondary ID(s)

09-02-0074

P06087

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Brigham and Women's Hospital

Schering-Plough

Dana-Farber Cancer Institute

Results

• Results available: Yes
• Date Posted: 31/07/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00879034