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An Open Label Phase II Trial of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome(HGPS) and Progeroid Laminopathies


Phase 2
N/A
N/A
Open (Enrolling)
Both
Progeria

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Trial Information

An Open Label Phase II Trial of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome(HGPS) and Progeroid Laminopathies


Inclusion Criteria:



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

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

- Travel: Patients must be willing and able to come to Boston for appropriate studies
and examinations at initiation of study and at months 6, 12, 18 and 24 on study.

- Patient must have adequate organ and marrow function as defined by the following
parameters:

- Blood: APC (ANC + bands + monocytes = APC) > 1,000/microliters, Platelets >
75,000/microliters (transfusion independent); Hemoglobin >9g/dl.

- Renal: creatinine Less than or equal 1.5 times normal for age or GFR > 70
ml/min/1.73m2.

- Hepatic: 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: PT/PTT < 120% upper limit of normal OR PI approval

- No overt renal, hepatic, pulmonary disease or immune dysfunction.

- 25-hydroxyvitamin D ≥ 20 ng/ml within 4 weeks of bisphosphonate infusion.

- Signed informed consent according to institutional guidelines must be obtained and
patient must begin therapy within twenty eight (28) days.

Exclusion Criteria:

- Other than the drugs used in this protocol, 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 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.

Type of Study:

Interventional

Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To evaluate the therapeutic effects of the combination of zoledronic acid, pravastatin and lonafarnib in patients with HGPS.

Outcome Time Frame:

2 years

Safety Issue:

No

Principal Investigator

Mark Kieran, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Hospital Boston/ Dana-Farber Cancer Instittue

Authority:

United States: Food and Drug Administration

Study ID:

Progeria Efficacy

NCT ID:

NCT00916747

Start Date:

August 2009

Completion Date:

December 2014

Related Keywords:

  • Progeria
  • Progeria
  • Progeria

Name

Location

Children's Hospital BostonBoston, Massachusetts  02115