Low Dose Rapamycin in ME/CFS, Long-COVID, and Other Infection Associated Chronic Conditions

NCT06257420 | Enrolling By Invitation FDA Drug

• 1 other identifier: SRI-RP-2023-1
• Study Type: observational
• Target: 150
• Locations: 1 country
• Sites: 5

Brief Summary

The goal of this observational study is to assess the clinical response and the effect of autophagy function in patients before, during and throughout oral low dose sirolimus (rapamycin) therapy. The main questions this study aims to answer are:

• Does rapamycin reduce the overall symptom burden in this patient population and does it improve the quality of life?
• Does rapamycin change mTOR driven autophagy deficits observed in a subset of patients? Participants will be asked to complete a series of questionnaires and quality of life instruments before starting rapamycin therapy prescribed by their physician and throughout their course of treatment. Study blood samples will be collected before starting therapy and throughout the course of treatment to assess serological markers of autophagy function.

Conditions

Myalgic Encephalomyelitis; Long-COVID

Keywords

ME/CFS; Long-COVID; Infection Associated Chronic Conditions

Outcome Measures

Primary Outcomes (3)

• The Short Form (36) Health Survey

  The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability i.e., a score of zero is equivalent to maximum disability and a score of 100 is equivalent to no disability.

  1 year

• Multidemensional Fatigue Inventory (MFI)

  The MFI is a 20-item scale designed to evaluate five dimensions of fatigue: general fatigue, physical fatigue, reduced motivation, reduced activity, and mental fatigue.

  1 year

• The Bell Activity Scale

  The Bell CFIDS Disability Scale is described in the book, The Doctor's Guide to Chronic Fatigue Syndrome: Understanding, Treating and Living with CFIDS (Bell 1995). This scale assesses functional ability in adult ME/CFS patients. Eleven statements describe patient status such as level of symptoms at rest, level of symptoms with exercise, activity level, and ability to perform work, travel and self care.

  1 year

Secondary Outcomes (1)

• Change in mTOR activation panel and blood markers involved in autophagy function.

  1.5 year

Study Arms (2)

ME/CFS and Long-COVID patients with serological evidence of autophagy disruption

ME/CFS and Long-COVID patients with serological evidence of autophagy disruption at baseline will be prescribed once weekly rapamycin at a max dose of 6mg per week.

Drug: Rapamycin

ME/CFS and Long-COVID patients without serological evidence of autophagy disruption

ME/CFS and Long-COVID patients without serological evidence of autophagy disruption at baseline will be prescribed once weekly rapamycin at a max dose of 6mg per week.

Drug: Rapamycin

Interventions

Rapamycin DRUG

Once weekly oral rapamycin

Also known as: Sirolimus, Rapamune

ME/CFS and Long-COVID patients with serological evidence of autophagy disruption; ME/CFS and Long-COVID patients without serological evidence of autophagy disruption

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients are recruited by study clinicians who are Principal Investigators in this trial.

You may qualify if:

• Diagnosis of ME/CFS that meets the Institute of Medicine (IOM) criteria
• Diagnosis of Long-COVID (PASC clinical criteria)

You may not qualify if:

• No diagnosis of ME/CFS or Long-COVID

Sponsors & Collaborators

• Simmaron Research Inc.: lead
• Center For Complex Diseases-Seattle, WA: collaborator
• Sierra Internal Medicine, Incline Village, NV: collaborator
• Center for Complex Diseases- Palo Alto, CA: collaborator
• Simmaron Research Inc. R&D Lab, University of Wisconsin, Milwaukee (UWM): collaborator
• Bateman Horne Center, Salt Lake City, UT: collaborator
• Mayo Clinic: collaborator

Study Sites (5)

Center For Complex Diseases

Palo Alto, California, 94040, United States

Location

The Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Sierra Internal Medicine

Incline Village, Nevada, 89451, United States

Location

Bateman Horne Center

Salt Lake City, Utah, 84102, United States

Location

Center For Complex Diseases

Seattle, Washington, 98109, United States

Location

Related Publications (2)

• Gottschalk G, Peterson D, Knox K, Maynard M, Whelan RJ, Roy A. Elevated ATG13 in serum of patients with ME/CFS stimulates oxidative stress response in microglial cells via activation of receptor for advanced glycation end products (RAGE). Mol Cell Neurosci. 2022 May;120:103731. doi: 10.1016/j.mcn.2022.103731. Epub 2022 Apr 26.

  PMID: 35487443 BACKGROUND

• Gottschalk CG, Whelan R, Peterson D, Roy A. Detection of Elevated Level of Tetrahydrobiopterin in Serum Samples of ME/CFS Patients with Orthostatic Intolerance: A Pilot Study. Int J Mol Sci. 2023 May 13;24(10):8713. doi: 10.3390/ijms24108713.

  PMID: 37240059 BACKGROUND

Related Links

• Simmaron Website

Biospecimen

Retention: SAMPLES WITH DNA

Whole blood will be collected to isolate plasma, PBMC, serum and RNA.

MeSH Terms

Conditions

Fatigue Syndrome, Chronic; Post-Acute COVID-19 Syndrome

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Encephalomyelitis; Neuroinflammatory Diseases; Nervous System Diseases; Neuromuscular Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; COVID-19; Pneumonia, Viral; Pneumonia; Respiratory Tract Infections; Infections; Virus Diseases; Coronavirus Infections; Coronaviridae Infections; Nidovirales Infections; RNA Virus Infections; Lung Diseases; Respiratory Tract Diseases; Post-Infectious Disorders

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Gunnar Gottschalk, PhD

  Simmaron Research Inc.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 17, 2024
• First Posted: February 14, 2024
• Study Start: December 11, 2023
• Primary Completion: December 11, 2025
• Study Completion (Estimated): June 11, 2026
• Last Updated: November 5, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)