Clinical Trial to Investigate CT38 in the Treatment of Myalgic Encephalomyelitis / Chronic Fatigue Syndrome
Condition: Myalgic Encephalomyelitis; Chronic Fatigue Syndrome
MeSH terms: Encephalomyelitis; Fatigue Syndrome, Chronic; Myalgia; Syndrome; Fatigue
Study type: Interventional
Phase: Phase 1/Phase 2

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.
    

Intervention name(s): CT38
Intervention type(s): Drug
Intervention description(s): Infusion
Allocation: Non-Randomized
Intervention model: Sequential Assignment
Intervention model description: 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).
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: myalgic encephalomyelitis; chronic fatigue syndrome; corticotropin releasing-factor receptor subtype 2 (CRF2); Alternative CRF2 names: CRFR2, CRH2, CRHR2