Magnetic Resonance Imaging Study of JM-4 in Multiple Sclerosis/Clinically Patients

Cook, Stuart, MD

Status and phase: Unknown; Early Phase 1

Conditions: Multiple Sclerosis

Treatments: Drug: JM-4

Study type: Interventional

Funder types: Other

Identifiers

NCT03887065

JM-4-001

Details and patient eligibility

About

This is a Phase 0/1 study of MS patients to determine the safety and potential efficacy of a novel, small human peptide designated as JM-4. The study will involve treatment for 5-7 days with JM-4 to determine the effects of Gadolinium(+) lesion number and volume in the brains of patients.

Full description

This study is the first study of JM-4 in patients with Multiple Sclerosis and is intended to show safety and potential efficacy in changing the size and/or number of GAD(+) lesions in the brain. The initial dose level of 1 mg/kg/ will establish safety of JM-4 treatment after 5-7 days of treatment via intravenous infusion over 30 minutes daily in3-5 patients with Multiple Sclerosis. MRI examinations will be conducted prior to treatment with JM-4 and 8 days after the initiation of treatment for the purpose of quantitating GAD(+) brain lesions. Once initial safety is established, the next group of 3-5 patients will receive 4 mg/kg/ of JM-4 daily for 5-7 days via 30 minute intravenous infusions, with MRI scans conducted prior to treatment and 8 days after the initial dose of JM-4. Once safety is established in this cohort of patients, a third group of patients may receive 9 mg/kg/ of JM-4 daily for 5-7 days via 30 minute infusions, with MRI scans conducted prior to the initial treatment and 8 days after the initial treatment.

Enrollment: 15 estimated patients

Sex: All

Ages: 18 to 55 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Definite MS (McDonald criteria) or CIS
• GAD(+) MRI brain lesion on screening exam, with or without clinical activity followed by a baseline MRI
• EDSS of 0-5.5 inclusive
• Weight of 40-115 kg
• Females must be post-menopausal or surgically sterilized or use a hormonal contraceptive, intra-uterine device or diaphragm with spermicide during the study
• Not be pregnant or breast feeding
• Males must be willing to use contraception during each day of the study
• Be willing to comply with study procedures and protocols for the duration of the study
• Voluntarily provide informed consent
• Be wiling and physically able to attend the study center as required for all study screening and procedures

Exclusion criteria

• Taking Tysabri, Gilenya, Tecfidera, Aubagio, Ocrevus or other immunosuppressive drugs within the prior 3 months
• Received Mitoxantrone or Lemtrada at any time
• Consumption of corticosteroids within the past 30 days
• Current or less than 5 years prior malignancy (excluding basal cell or squamous cell skin cancer)
• Serious systemic disorder which might, in the opinion of the investigators, interfere with safety, compliance, treatment or evaluation of efficacy. Conditions would include but not be limited to significant cardiac, liver, kidney, lung or cerebrovascular disease, HIV, serious infections, serous psychiatric disease or poorly controlled diabetes mellitus
• aversion, intolerance or allergy to repeated MRI with gadolinium administration

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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15 participants in 3 patient groups

Starting dose

Experimental group

Description:

Three to five patients will receive a daily dose of 1 mg/kg JM-4 (in normal saline) delivered via intravenous infusion for no more than 30 minutes for up to 7 consecutive days.

Treatment:

Drug: JM-4

Intermediate dose of JM-4

Experimental group

Description:

Three to five patients will receive a daily dose of 4 mg/kg of JM-4 (in normal saline) via intravenous infusion for no more than 30 minutes for up to 7 consecutive days.

Treatment:

Drug: JM-4

High dose of JM-4

Experimental group

Description:

Three to five patients will receive a daily dose of 9 mg/kg of JM-4 (in normal saline) via intravenous infusion for no more than 30 minutes for up to 7 consecutive days.

Treatment:

Drug: JM-4