NCT04625595

Multiple Ascending Dose (MAD) Study of IMT-002 in HLA-DQ8-positive Type 1 Diabetes

A Phase 1b, Randomized, Single-blind, Placebo-controlled, Multiple Ascending Dose (MAD) Study to Assess the Steady-State Pharmacokinetics and DQ8 Blocking Efficacy of Orally Administered IMT-002 in Patients With Type 1 Diabetes and HLA-DQ8

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Immunomolecular Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years – 45 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to characterize the safety, steady-state pharmacokinetics (PK) of IMT-002, and will serve as a dose range identification for the pharmacodynamic effect of blocking self-antigen presentation in adults with type 1 diabetes (T1D) having the human leukocyte antigen (HLA)-DQ8 gene.

Detailed description

This is a randomized, single-blind, placebo-controlled study that will include 4 ascending dose cohorts: 350 mg twice daily (BID), 1050 mg once daily (QD), 700 mg BID, and 1050 mg BID. Subjects will undergo prescreening for genetic typing and then screening procedures up to 28 days prior to the first dose to determine eligibility. T1D adults between 18 and 45 years of age, inclusive, who are positive for at least one gene encoding for HLA-DQ8 (DQA1\*0301, DQB1\*0302) will be enrolled. Each cohort will include 6 subjects on active drug, and the study will include 6 subjects total on placebo. Each cohort will participate in a 2-week dosing period. Enrollment of the cohorts will be sequentially staggered such that initial safety data after the first four subjects assigned to active treatment complete one week of treatment in each cohort will be reviewed before the next ascending dose cohort is enrolled. The safety reviews will include cumulative safety data for all subjects to that point. Subjects will have 5 scheduled clinic visits: screening, first day of dosing, 1 week after dosing begins, 2 weeks after dosing begins (end of treatment), and 1 week following the final dose. Subjects will self-administer study drug on non-clinic treatment days. Safety assessments will be conducted at all study visits. Insulin use (dose and frequency) will be monitored. Pharmacokinetic (PK) assessments will be evaluated at every visit during the treatment period to characterize the following: single dose PK, trough PK and steady PK. Pharmacodynamic (PD) and immunological assessments will be evaluated before, during and after treatment.

Conditions

• Type1 Diabetes

Interventions

Type	Name	Description
DRUG	350mg BID (700mg total daily) IMT-002, D-methyldopa, active formulation in capsule or Placebo, microcrystalline cellulose in capsule	Oral drug or placebo self-administered by subject as a capsule by mouth once-daily or twice-daily
DRUG	700mg BID (1400mg total daily) IMT-002, D-methyldopa, active formulation in capsule or Placebo, microcrystalline cellulose in capsule	Oral drug or placebo self-administered by subject as a capsule by mouth once-daily or twice-daily
DRUG	1050mg QD (1050mg total daily) IMT-002, D-methyldopa, active formulation in capsule or Placebo, microcrystalline cellulose in capsule	Oral drug or placebo self-administered by subject as a capsule by mouth once-daily or twice-daily
DRUG	1050mg BID (2100mg total daily) IMT-002, D-methyldopa, active formulation in capsule or Placebo, microcrystalline cellulose in capsule	Oral drug or placebo self-administered by subject as a capsule by mouth once-daily or twice-daily

Timeline

• Start date: 2020-11-09
• Primary completion: 2021-05-11
• Completion: 2021-07-31
• First posted: 2020-11-12
• Last updated: 2021-08-25

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study