| Eligibility |
Inclusion Criteria:
1. Subjects must be at least = 18 years of age.
2. Subjects should have a confirmed diagnosis of typical Werner syndrome by genetic
analysis.
3. Subjects should be osteopenic (T-score between -1.0 and -2.5) or have confirmed
osteoporosis (Tscore = -2.5) at screening/baseline. Subjects may continue to receive
bone supplements (e.g., bisphosphonates, calcium supplements, Vitamin D supplements,
etc.) as appropriate per standard of care throughout the study.
4. Male or non-pregnant, non-lactating female.
5. Subjects screening blood chemistry inclusive of metabolic panel, hematology, and urine
analysis results should be acceptable to the investigator.
6. Negative pregnancy test for female subjects as described below. Women of child bearing
potential (WOCBP) and Women not of child-bearing potential are eligible to
participate. Both women of childbearing potential and women of non-child-bearing
potential should use an approved method of birth control and agrees to continue to use
this method for the duration of the study and for 90 days after last treatment.
Acceptable methods of contraception include abstinence, female subject/partner's use
of hormonal contraceptive (oral, implanted, or injected) in conjunction with a barrier
method (WOCBP only), female subject/partner's use of an intrauterine device (IUD), or
if the female subject/partner is surgically sterile or two years post-menopausal. All
male subjects/partners must agree to use a In addition, subjects may not donate sperm
for the duration of the study and for 90 days after last treatment.
Females who are less than two (2) years post-menopausal, those with tubal ligations
and those using contraception must have a negative serum pregnancy test at baseline
within the one (1) week prior to the first study medication administration. Every six
weeks, and at study termination a pregnancy test should be performed, either serum or
urine stick test. However, if the urine result is positive, a serum pregnancy test
will be performed.
7. Provide signed written informed consent and willingness, ability to comply with study
requirements.
8. Subject must have a projected life expectancy of = 12 months in the opinion of the
Investigator.
Exclusion Criteria:
1. Subject with clinical signs seen at screening are at the final stage of Werner
syndrome progression, and completion of the study is difficult to be assessed,
including:
- Subjects who received continuous or intermittent home oxygen therapy for 6 months
before obtaining consent
- Subjects who received at least 2 hospitalizations for pneumonia during the 12
months prior to obtaining consent
- Subjects who have at least 10% net weight loss and have not recovered. This
includes significant net weight loss over the last six months.
2. Subjects with significant dehydration as judged by the principal investigator.
3. Subjects with pericardial fluid, ascites and pleural effusion.
4. Therapy with investigational drugs within 30 days of beginning study medication.
5. History of prior malignancy, except for adequately treated in situ cancer, basal cell,
squamous cell skin cancer, or other cancers (e.g., breast, prostate) for which the
subject has been disease-free for at least 3 years.
6. Any serious medical condition, laboratory abnormality, psychiatric illness, or
comorbidity that, in the judgment of the Investigator, would make the subject
inappropriate for the study.
7. Serious systemic fungal, bacterial, viral, or other infection that is not controlled
or requires intravenous (IV) treatment for infection(s).
8. Subjects with known history of having Acquired Immunodeficiency Syndrome (AIDS) or
with a history known to be infected with Human Immunodeficiency Virus (HIV).
9. Subjects with a history of serious drug hypersensitivity or allergic reaction such as
anaphylaxis or any component of the formulation.
10. Clinically significant bleeding within 2 weeks prior to baseline (e.g.,
gastrointestinal [GI] bleeding, intracranial hemorrhage).
11. Medical or recreational use of marijuana or THC-containing compounds within 3 months
of screening visit and for the duration of the trial.
12. Pregnant or lactating women.
13. Major surgery, defined as any surgical procedure that involves general anesthesia and
a significant incision (i.e., larger than what is required for placement of central
venous access, percutaneous feeding tube, or biopsy), within 28 days prior to baseline
or anticipated surgery during the study period.
14. Inability to return for scheduled treatment and assessments.
15. Subjects that require concomitant use of any medications or substances that are strong
inhibitors or inducers of CYP2D6 or CYP2C8
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