NCT03613129

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

Pilot Phase 1/2, Open-Label, Clinical Trial to Investigate CT38 in the Treatment of Myalgic Encephalomyelitis / Chronic Fatigue Syndrome

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: LUCINDA BATEMAN, MD · Academic / Other
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Myalgic Encephalomyelitis / Chronic Fatigue Syndrome (ME/CFS) is a complex disorder that may be triggered by infection or other stressors (e.g., emotional or physical trauma, immune activation, chemical exposures). Its hallmark is a reduced capacity for physical and mental activity manifest as profound fatigue along with a cascade of debilitating symptoms (including pain, cognitive dysfunction, orthostatic intolerance, sensitivities, and irregularities of the autonomic, immune and metabolic systems) that worsen with activity (referred to as post-exertional malaise or PEM), are not improved by sleep, and can persist for years. Patients are often unable to handle the activities of daily living and experience a loss of career and a very poor quality of life. There are no established diagnostic tests or approved therapeutics for ME/CFS. The cause of ME/CFS is not known. It has been postulated that ME/CFS could arise from the up-regulation of a specific receptor (CRF2) in those parts of the brain that govern the sensitivity of the stress response. This configuration would invoke a major response to a minor stimulus, ultimately leading to neuroendocrine, autonomic, immune and metabolic abnormalities that are commonly observed. There is no animal model of ME/CFS, but overstimulating CRF2 in healthy rats, induces signs and symptoms consistent with the disease in humans; while down-regulating it, via CT38 (an experimental peptide), eliminates the ability to stimulate these signs and symptoms. Hypothesis: Utilize CT38 to down-regulate CRF2 to restore a normal stress response, and potentially eliminate disease signs and symptoms. The study will enroll 18 patients, who meet the Fukuda and Canadian criteria for ME/CFS, and treat them with various doses of CT38. The primary endpoint will be the change in the average total daily symptom score (TDSS), over 28-day periods immediately prior to the first treatment (pre-treatment) and immediately prior to exit from the trial (post-treatment). The TDSS is the sum of 13 individual symptom scores, each recorded daily by the patient on a 6-point scale (0=none, 1=very mild, 2=mild, 3=moderate, 4=severe, 5=very severe). The individual symptoms included fatigue, muscle/joint pain, sleep issues (e.g., un-refreshing sleep, difficulty falling or staying asleep, excessive sleepiness), cognitive issues (e.g., slow information processing, memory difficulties, inability to concentrate/focus, attention deficit), orthostatic intolerance (e.g., dizziness, spatial disorientation, light-headedness, feeling faint), body temperature perceptions, flu-like symptoms (e.g., sore throat, tender lymph nodes, swollen glands, fever, chills, sinus/nasal problems), headaches or sensory sensitivities (to light, sound, smell, touch, taste), shortness of breath, gastrointestinal problems (e.g., nausea, stomach/abdominal pain, diarrhea), urogenital problems (e.g., frequent urination), anxiety and depression. The secondary outcomes will assess general health status (determined by Short-Form 36, or SF-36), as well as safety assessments.

Conditions

• Myalgic Encephalomyelitis
• Chronic Fatigue Syndrome

Interventions

Type	Name	Description
DRUG	CT38	Infusion

Timeline

• Start date: 2018-07-23
• Primary completion: 2019-04-30
• Completion: 2019-04-30
• First posted: 2018-08-02
• Last updated: 2020-05-12
• Results posted: 2020-04-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study