Actively Recruiting

Phase 1
Age: 12Years +
All Genders
NCT07364513

Phase 1b Safety Study of IMSB301 in Type 1 Interferonopathies

Led by ImmuneSensor Therapeutics Inc. · Updated on 2026-01-23

6

Participants Needed

1

Research Sites

28 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Open-Label Study (Phase Ib) of Type 1 Interferonopathy patients receiving IMSB301 monotherapy.

CONDITIONS

Official Title

Phase 1b Safety Study of IMSB301 in Type 1 Interferonopathies

Who Can Participate

Age: 12Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • First subject must be at least 16 years old and weigh at least 50 kg; remaining subjects must be at least 12 years old and weigh at least 40 kg
  • Molecular diagnosis of one of the following: Aicardi-Gouti�88res Syndrome with specific mutations, monogenic SLE with specific mutations, familial chilblain lupus with specific mutations, neurological syndromes with ATAD3A mutations, unnamed interferonopathy with DNASE2 mutations, or COPA syndrome with COPA mutations
  • Clinical syndrome consistent with Type 1 Interferonopathy based on clinical, CSF, or radiological findings
  • Women of child-bearing potential must have negative serum pregnancy test at screening and negative urine pregnancy test on Day -1
  • Sexually active males and females with reproductive potential must agree to use effective contraception or abstain from heterosexual intercourse during the study and for three months after last dose
  • Subjects and caregivers must be willing and able to comply with visits, assessments, blood sampling, and other trial procedures
  • Ability to provide informed consent or have a legal representative provide consent with assent from minor/vulnerable subjects as appropriate
Not Eligible

You will not qualify if you...

  • Other significant neurological disorders not related to Type 1 Interferonopathy, brain tumor, space-occupying lesion, or severe head injury history
  • Significant concomitant disease posing additional risk or interfering with drug assessment
  • BMI above 33 kg/m2 or body weight over 130 kg
  • Evidence of active infection at screening or Day 1
  • Symptomatic herpes zoster infection within 12 weeks or more than one episode in past two years
  • Positive hepatitis B, hepatitis C, HIV, or TB test at screening
  • Household contact with active TB without documented prophylaxis
  • Serious infection requiring hospitalization or intravenous treatment within six months
  • Resting ECG showing prolonged QTc interval or other abnormalities posing safety risk
  • Prior history of cancer except certain successfully treated types more than five years prior
  • Receipt of investigational drug within 30 days or five half-lives before Day -1
  • Prior treatment with immunomodulators including JAK inhibitors within 14 days
  • Prior treatment with interferon inhibitors within three months
  • Concurrent treatment with nucleoside reverse transcriptase inhibitors or other antivirals
  • Receipt of strong CYP3A4 inhibitors within five half-lives before Day -1
  • Known allergy to IMSB301 or its components
  • Known allergy to shellfish
  • Female subjects breastfeeding or with positive pregnancy test at screening or Day -1
  • Vaccination within three weeks before Day -1

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

The Children's Hospital at Westmead

Sydney, Australia

Actively Recruiting

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How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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