Clinical trial NCT04488601: Participatory Evaluation (of) Aging (With) Rapamycin (for) Longevity Study
Official title: Participatory Evaluation (of) Aging (With) Rapamycin (for) Longevity Study (PEARL): A Prospective, Double-Blind, Placebo-Controlled Trial for Rapamycin in Healthy Individuals Assessing Safety and Efficacy in Reducing Aging Effects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Aging
Keywords: rapamycin
aging
longevity
sirolimus
healthy aging
age-related conditions
Lead sponsor: AgelessRx
Sponsor class: INDUSTRY
Collaborators: name: University of California, Los Angeles
First posted: 2020-07-28
Enrollment: 129
Sex: ALL
Minimum age (years): 50
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Prospective, Double-Blind, Placebo-Controlled Trial
Primary purpose: PREVENTION

Brief summary: This is a randomized, placebo-controlled trial into the safety and efficacy in reducing clinical measures of aging in an older adult population.

Detailed description: A randomized, double-blind, placebo-controlled trial assessing the effects of low and high doses of intermittent Rapamycin on a weekly schedule. The researchers aim to establish a long-term safety profile, determine the long-term efficacy of Rapamycin in reducing clinical aging measures, and biochemical and physiological endpoints associated with declining health and aging in healthy older adults.

Eligibility criteria: Inclusion Criteria:

* Age 50-85
* Any sex
* Any ethnicity
* Interest in taking Rapamycin off-label
* Willing to undergo tests
* Relatively good health with only well-managed chronic diseases (hypertension, coronary artery disease, type II diabetes, etc.) clinically stable
* Adequate cognitive function to be able to give informed consent
* Technologically competent to complete web forms and perform video calls with the PI

Exclusion Criteria:

* Anemia - Hg \< 9.0 g/dl, Leukopenia - white blood cells (WBC) \< 3,500/mm3 , Neutropenia - absolute neutrophil count \< 2,000/mm3 , or Platelet count - platelet count \< 125,000/mm3
* Premenopausal females (due to menstruation-induced anemia, etc.)
* Patients scheduled to undergo major surgery in the next 12 months
* Patients undergoing or scheduled to undergo chemotherapy or any other treatment for malignancy
* Patients scheduled for immunosuppressant therapy for transplant
* Patients with impaired wound healing or history of a chronic open wound
* Untreated dyslipidemia with LDL-c \> 190 and family history of dyslipidemia, Total cholesterol \> 350 mg/dl, or triglycerides \> 880 mg/dl.
* Impaired hepatic function, including elevated alkaline Phosphatase levels, aspartate aminotransferase (AST), alanine aminotransferase (ALT), Albumin, or T. Bili.
* HIV/AIDS, chronic Lyme, Babesia, Ehrlichiosis, Anaplasmosis, or other chronic infections that require ongoing treatment or monitoring
* Allergy to Rapamycin
* Any form of clinically relevant primary or secondary immune dysfunction or deficiency (e.g. X-linked agammaglobulinemia (XLA), common variable immunodeficiency (CVID))
* Chronic oral corticosteroid or immunosuppressive medication use (e.g. Enbrel, Humira, methotrexate).
* Fibromyalgia or Chronic Fatigue Syndrome/Myalgic Encephalomyelitis, Breast Implant Illness,
* Congestive heart failure: self-assessed functional status of heart failure New York Heart Association (NYHA) classification III or IV
* Impaired renal function, as defined as glomerular filtration rate (GFR) \< 30
* Poorly controlled diabetes, as defined as HbA1c \> 7%
* Type I Diabetes, or Insulin-dependent Type II diabetes
* Substance abuse disorder either untreated or if treated within the last 5 years
* PTSD, Bipolar disorder, Schizophrenia, or any other untreated or poorly controlled mental health or mood disorder, or history of hospitalization due to mental health condition
* Those who have taken metformin, rapamycin, or rapalogs in the past 6 months

  * (volunteers who were on metformin for aging can participate, provided they agree to stop taking metformin before and during the trial)

Primary outcomes:
measure: Changes in visceral fat as measured by dual-energy x-ray absorptiometry (DXA) scan
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
Secondary outcomes:
measure: bone density
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: lean body mass
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: adverse events
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: complete blood count (CBC)
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change of blood electrolytes（serum potassium, sodium, chloride, and carbon dioxide in mmol/L）measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change of liver function as measured by serum globulin in g/dL, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change of liver function as measured by serum bilirubin in mg/dL, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change of liver function as measured by serum alkaline phosphatase, AST, and ALT in IU/L, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change of renal function as measured by serum albumin in g/dL, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change of renal function as measured by serum creatinine and uric acid in mg/dL, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change in lipids (serum total cholesterol, triglycerides, HDL, and LDL cholesterol in mg/dL) measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change in fasting serum glucose in mg/dL, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change in insulin in uIU/mL, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change in fasting serum IGF-1 in ng/mL, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
measure: Change in serum Hemoglobin A1c in % of total hemoglobin, measured at baseline and after 6 and 12 months.
timeFrame: 6 month interim analysis of the data, 12 month safety profile will be established
Other outcomes:
Locations:
city: Chicago
country: United States
facility: AgelessRx
state: Illinois
Study officials:
affiliation: University of California, Los Angeles
name: James Watson, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: AgelessRx
name: Sajad Zalzala, MD
role: STUDY_DIRECTOR
References: