Clinical Trial to Investigate CT38 in the Treatment of Myalgic Encephalomyelitis / Chronic Fatigue Syndrome

NCT03613129 | Completed Phase 1 Results FDA Drug

• 1 other identifier: ME-101p
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

Brief Summary

This study seeks to investigate the safety, tolerability and efficacy of CT38, an experimental peptide administered by subcutaneous infusion, in the treatment of ME/CFS patients.

Conditions

Myalgic Encephalomyelitis; Chronic Fatigue Syndrome

Keywords

myalgic encephalomyelitis; chronic fatigue syndrome; corticotropin releasing-factor receptor subtype 2 (CRF2); Alternative CRF2 names: CRFR2, CRH2, CRHR2

Outcome Measures

Primary Outcomes (1)

• Total Daily Symptom Score (TDSS)

  Pre-/post-treatment difference in TDSS (0-65 scale, 0=no symptoms; 65=maximum of 5 for each of 13 specific patient-reported symptoms). The TDSS sums the patient-reported daily symptom score for each of 13 specific symptoms (including fatigue, muscle/joint pain, sleep problems, cognitive problems, orthostatic intolerance, body temperature perceptions, flu-like symptoms, headaches or sensitivities, shortness of breath, gastrointestinal problems, urogenital problems, anxiety and depression), each assessed on a 0-5 scale (0=no symptom, 1=very mild, 2=mild, 3=moderate, 4=severe, 5=severe)

  28 days preceding Visit 3 (pre-treatment) and 28 days preceding Visit 6 (post-treatment)

Secondary Outcomes (2)

• SF-36, PCS

  Visit 3 before treatment (pre-treatment) and at Visit 6 (post-treatment)

• SF-36, MCS

  Visit 3 before treatment (pre-treatment) and at Visit 6 (post-treatment)

Study Arms (4)

D0.20

ACTIVE COMPARATOR

Subcutaneous infusion of CT38 at 0.20 μg/kg/hour, for 3 hours on each of 2 days

Drug: CT38

D0.03

ACTIVE COMPARATOR

Subcutaneous infusion of CT38 at 0.03 μg/kg/hour, for 3.5 hours on each of 3 days

Drug: CT38

D0.06

ACTIVE COMPARATOR

Subcutaneous infusion of CT38 at 0.06 μg/kg/hour, for 3.5 hours on each of 3 days

Drug: CT38

D0.01

ACTIVE COMPARATOR

Subcutaneous infusion of CT38 at 0.01 μg/kg/hour, for 3.5 hours on each of 3 days

Drug: CT38

Interventions

CT38 DRUG

Infusion

D0.01; D0.03; D0.06; D0.20

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Provision of signed and dated informed consent form
• Ability to read, understand and speak English
• Living at an altitude between 3,500 and 5,500 feet above sea level for the past 1 year
• Willing to perform an exercise test
• Diagnosed with ME/CFS and meet the following 3 case definitions: Fukuda Research Case Definition for CFS (1994), Revised Canadian Consensus Criteria for ME/CFS (2010) and the Institute of Medicine (IOM) Clinical Diagnostic Criteria for ME/CFS (2015)
• Relatively stable state of illness for the individual patient over the past 3 months
• Male or female, between the ages of 18 and 60 years old
• Males or females of reproductive potential agree to remain abstinent or use (or have their partner use) 2 acceptable methods of contraception, starting from the time of informed consent through 28 days after the last dose of study drug. Acceptable methods of birth control during the study are intrauterine device, diaphragm with spermicide, contraceptive sponge, condom or vasectomy. Oral contraceptive pills may not be used as the sole method of contraception because the effect of CT38 on the efficacy of oral contraceptive pills has not yet been established
• Stated willingness to comply with all study procedures and remain available for the study duration
• Have mobile (smart) phone and access to the internet

You may not qualify if:

• Alternate medical or psychiatric illness that could explain the ME/CFS symptoms
• Unwilling or unable to perform an exercise test
• Pregnancy, or while breast feeding. Women should not be enrolled within 6 months of giving birth and within 3 months of cessation of breast feeding
• A Body Mass Index \> 35
• Cigarette smoker or former smoker who has smoked within 6 months of the start of the study
• Living at an altitude that is more than 1,000 feet (lower or higher) from the study site (which is 4,500 feet above sea level)
• History of:
• Major depression with psychotic or melancholic features before the diagnosis of ME/CFS, or active depression (major depression with psychotic or melancholic features) as determined by self-report
• Untreated endocrine diagnoses including hypothyroidism (Hashimoto's, etc.), Grave's disease, adrenal insufficiency, hypogonadism (testosterone deficiency), diabetes mellitus or insipidus
• Acute infection within the past 30 days
• Within the last 3 years, any significant head injury, e.g., concussion with loss of consciousness, brain surgery, an automobile accident with head/neck injury, other traumatic brain injury
• A supra-ventricular tachycardia or ventricular tachycardia, e.g., atrial fibrillation or flutter, paroxysmal atrial fibrillation, junctional tachycardia, ventricular tachycardia
• Severe baseline hypotension defined as rested sitting systolic BP \< 100 mmHg or rested sitting diastolic BP \< 60 mmHg
• Renal impairment based upon the local lab normal estimated glomerular filtration rate (eGFR) (drug is cleared by passive renal filtration)
• Known hypersensitivity or clinically significant allergies to tromethamine or Tween 80 (both excipients in the drug product)

Sponsors & Collaborators

• LUCINDA BATEMAN, MD: lead

Study Sites (1)

Bateman Horne Center

Salt Lake City, Utah, 84102, United States

Location

Related Publications (1)

• Pereira G, Gillies H, Chanda S, Corbett M, Vernon SD, Milani T, Bateman L. Acute Corticotropin-Releasing Factor Receptor Type 2 Agonism Results in Sustained Symptom Improvement in Myalgic Encephalomyelitis/Chronic Fatigue Syndrome. Front Syst Neurosci. 2021 Sep 1;15:698240. doi: 10.3389/fnsys.2021.698240. eCollection 2021.

  PMID: 34539356 DERIVED

MeSH Terms

Conditions

Fatigue Syndrome, Chronic

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

Limitations and Caveats

InTiME was based on a novel theory of ME/CFS and an unprecedented drug mechanism. Predicted doses had to be adjusted, limiting statistical inferences, but providing preliminary support for the hypothesis and treatment approach.

Results Point of Contact

• Title: Lucinda Bateman, MD
• Organization: Bateman Horne Center

LBateman@batemanhornecenter.org

(801) 359-7400

Study Officials

• Lucinda Bateman, MD

  Bateman Horne Center

  PRINCIPAL INVESTIGATOR

• Suzanne D Vernon, PhD

  Bateman Horne Center

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Founder & Medical Director

Model Details: The study is comprised of a recruitment and screening period, enrollment (Visit 1), a 4-week (at least) pre-treatment assessment period, a 1-week interventional treatment period with drug infused at Visits 3, 4 and 4b, a 4-week (at least) post-treatment assessment period, and a close-out (Visit 6).

Study Record Dates

• First Submitted: July 13, 2018
• First Posted: August 2, 2018
• Study Start: July 23, 2018
• Primary Completion: April 30, 2019
• Study Completion: April 30, 2019
• Last Updated: May 12, 2020
• Results First Posted: April 30, 2020

Record last verified: 2020-04

Locations

US (1)