A Multiple Ascending Dose Trial Investigating Safety, Tolerability and Pharmacokinetics of NNC0361-0041

Completed · Phase 1 · Has a placebo group

Conditions studied: Type I Diabetes

In brief

The trial is a placebo-controlled, double-blinded within cohorts, randomized, multiple ascending dose trial with a sequential trial design. The primary outcome is to investigate the safety and tolerability of ascending subcutaneous weekly doses of NNC0361-0041 plasmid in patients with T1D.

Key facts

Study ID

NCT04279613

Run by

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

People needed

47

Starts

2020-11-23

Expected to finish

2024-04-24

Last updated by the study team

2025-05-20

Who can join

Age: 18 and older, up to 45. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Willing to provide Informed Consent
• Participants must live in a location with rapid access to emergency medical services
• Age 18-45 years (both inclusive) at the time of signing informed consent
• Must have a diagnosis of T1D for less than 48 months at randomization
• Must have at least one diabetes-related autoantibody present (GAD65A; mIAA, if obtained within 10 days of the onset of insulin therapy; IA-2A; ICA; or ZnT8A)
• Must have stimulated C-peptide levels greater than or equal to 0.2 pmol/ml measured during an MMTT conducted at least 21 days from diagnosis of diabetes and within one month (37 days) of randomization
• Be willing to comply with intensive diabetes management
• HbA1c ≤8.5% at screening
• Subjects who are CMV and/or EBV seronegative at screening must be CMV and/or EBV PCR negative within 37 days of randomization and may not have had signs or symptoms of a CMV and/or EBV compatible illness lasting longer than 7 days within 37 days of randomization
• Be up to date on recommended immunizations
• Be at least 6 weeks from last live immunization
• Be at least 4 weeks from killed vaccine other than flu vaccine
• Participants are required to receive killed influenza vaccination at least 2 weeks prior to randomization when vaccine for the current or upcoming flu season is available
• Be willing and medically acceptable to postpone live vaccines during the treatment period and for 3 months following last dose of study drug
• If participant is female with reproductive potential, she must have a negative pregnancy test at screening and be willing to avoid pregnancy using a highly effective contraceptive method for the 12 months of the study
• Males of reproductive age must use adequate contraceptive method during the treatment phase and for 3 months following last dose of study drug
• Participants are required to receive an authorized non-live COVID-19 vaccination and be fully vaccinated, including eligible boosters as indicated, at least two weeks prior to randomization.

You may not qualify if…

• Potential participants must not meet any of the following exclusion criteria:
• One or more screening laboratory values as stated
• Leukocytes < 3,000/μL
• Neutrophils <1,500 /μL
• Lymphocytes <800 /μL
• Platelets <100,000 /μL
• Haemoglobin <6.2 mmol/L (10.0 g/dL)
• Potassium >5.5 mmol/L or <3.0 mmol/L
• Sodium >150mmol/L or < 130mmol/L
• AST or ALT ≥2.5 times the upper limits of normal
• Bilirubin ≥ 1.5 times upper limit of normal
• Glomerular Filtration Rate (eGFR) value of eGFR < 60 ml/min/1.73 m2 as defined by KDIGO 2012 (43)
• Any other laboratory abnormality that might, in the judgment of the investigator, place the subject at unacceptable risk for participation in this trial
• Current or ongoing use of non-insulin pharmaceuticals that affect glycemic control within prior 7 days of screening
• Use of other immunosuppressive agents including chronic use of systemic steroids. Topical products are acceptable (nasal, conjunctival, skin)
• Have active signs or symptoms of acute infection at the time of randomization
• Have current, confirmed COVID-19 infection
• Chronic active infection other than localized skin infections
• Have evidence of prior or current tuberculosis infection as assessed by PPD, interferon gamma release assay or by history
• Have evidence of current or past HIV, Hepatitis B infection
• Have evidence of active Hepatitis C infection
• Vaccination with a live virus within the last 6 weeks and killed vaccine within 4 weeks (except 2 weeks for flu vaccine)
• Be currently pregnant or lactating, or anticipate getting pregnant within the one-year study period.
• Have severe obesity: adults BMI ≥ 40
• Have a history of malignancies

Where it is running

• Children's Hospital of Orange County — Orange, California, United States
• University of California - San Francisco — San Francisco, California, United States
• Stanford University — Stanford, California, United States
• Barbara Davis Center at University of Colorado Anschutz Medical Campus — Aurora, Colorado, United States
• Yale University School of Medicine — New Haven, Connecticut, United States
• University of Florida — Gainesville, Florida, United States
• Emory Children's Center — Atlanta, Georgia, United States
• Indiana University - Riley Hospital for Children — Indianapolis, Indiana, United States
• Joslin Diabetes Center — Boston, Massachusetts, United States
• Regents of the University of Minnesota — Minneapolis, Minnesota, United States
• The Children's Mercy Hospital — Kansas City, Missouri, United States
• The Naomi Berrie Diabetes Center at Columbia University Medical Center — New York, New York, United States
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Vanderbilt Eskind Diabetes Center — Nashville, Tennessee, United States
• University of Texas Southwestern Medical Center — Dallas, Texas, United States
• Benaroya Research Institute — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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