Type 1 InterferonopathiesPhase 1NCT07364513
Phase 1b Safety Study of IMSB301 in Type 1 Interferonopathies
A Phase 1 study testing IMSB301 in people with Type 1 Interferonopathies, run by ImmuneSensor Therapeutics Inc. at 1 site in Australia.
Trial summary
Open-Label Study (Phase Ib) of Type 1 Interferonopathy patients receiving IMSB301 monotherapy. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Type 1 Interferonopathies
- What is being tested?
- IMSB301
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteAustralia
- Study dates
- 12 Jan 2026to 30 Jul 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- First subject must be at least 16 years of age and weighing at least 50 kg. Remaining subjects must be at least 12 years of age and weighing at least 40 kg.
- Molecular diagnosis of one of the following:
- Aicardi-Goutières Syndrome (AGS) with mutations in TREX1, RNASEH2A, RNASEH2B, RNASEH2C, SAMHD1, LMS11, or RNU7-11, or
- Monogenic SLE with mutations in TREX1, RNASEH2A, RNASEH2B, or RNASEH2C, or
- Familial chilblain lupus (CHBL) with mutations in TREX1 or SAMHD1, or
- Neurological syndromes with mutations in ATAD3A, or
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- An unnamed interferonopathy with mutations in DNASE2, or
- Coatomer Protein Complex subunit alpha (COPA) syndrome with mutations in COPA. The remaining subtypes of AGS and other type 1 interferonopathies are not eligible.
- Clinical syndrome consistent with type 1 interferonopathy diagnosis based on clinical, CSF, or radiological findings.
- Women of child-bearing potential (defined as a female who has experienced menarche and who has not undergone successful surgical sterilization [hysterectomy, bilateral salpingectomy, or bilateral oophorectomy]) or is not postmenopausal (defined as amenorrhea for at least 12 consecutive months with an appropriate clinical profile at the appropriate age, e.g., greater than 45 years) must have a negative serum pregnancy test at screening and a negative urine pregnancy test on Day -1.
- Sexually active male and female subjects with reproductive potential must agree to use highly effective contraception or agree to abstain from heterosexual intercourse throughout the study and for at least 3 months after last study drug dose.
- Subjects and caregiver must be willing and able to comply with scheduled visits, clinical assessments, blood sample collections, and other trial procedures.
- Have the ability to provide informed consent or have legal representative of minor/vulnerable subjects who is willing and able to provide written informed consent, provided that assent is obtained from subjects at an age-appropriate level.
Who cannot join
- Presence of other significant neurological disorder that is not related to type 1 interferonopathy, brain tumor or other space-occupying lesion, or history of severe head injury.
- The presence of significant concomitant disease that would, in the judgement of the Investigator, pose additional risk to the subject, interfere with the assessment of safety and tolerability, or significantly interfere with the metabolic disposition of study drug.
- BMI above 33 kg/m2 or a total body weight in excess of 130 kg.
- Have any of the following infection risks:
- Evidence of active infection during screening or on Day 1
- Symptomatic herpes zoster infection within 12 weeks prior to the screening period, or more than one episode of herpes zoster infection in the preceding two years
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- Positive hepatitis B, hepatitis C, HIV or TB test at screening
- Household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB
- Serious infection, defined as infection requiring admission to hospital or treatment with intravenously administered medication, within the six months.
- Resting 12-lead ECG showing confirmed prolongation of QTc (Fridericia's correction) interval (QTc interval > 470 for females and >450 for males), or other baseline ECG abnormalities that, in the judgement of the Investigator, would pose additional safety risk to the subject.
- A prior history of cancer, other than squamous cell carcinoma, basal cell carcinoma, or cervical intra-epithelial neoplasia that was successfully treated at least five years prior to the screening visit and which has shown no sign of clinical recurrence.
- Receipt of an investigational drug within 30 days or five half-lives of the drug (whichever is longer) prior to Day -1.
- Prior treatment with an immunomodulator including a JAK inhibitor within 14 days of screening.
- Prior treatment with an interferon inhibitor within 3 months of screening.
- Concurrent treatment of a nucleoside reverse transcriptase inhibitor or other antiviral drug.
- Receipt of a strong inhibitor of CYP3A4 (APPENDIX A) within five half-lives prior to Day -1
- Known allergy to IMSB301 or its components.
- Known allergy to shellfish.
- Female subjects who are breastfeeding or who have a positive pregnancy test at screening or Day -1.
- Receipt of a vaccination within 3 weeks prior to Day -1.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Australia1 site, 1 recruiting
- Recruiting
The Children's Hospital at Westmead
Sydney
Dr. Russell Dale, MD, +61 2 7825 0000, russell.dale@health.nsw.gov.au
About this study
Official title: A Phase 1b Open-Label Study of IMSB301 in Subjects With a Type 1 Interferonopathy
Open-Label Study (Phase Ib) of Type 1 Interferonopathy patients receiving IMSB301 monotherapy.
Read the detailed description
This is an open-label, Phase Ib study designed to evaluate the safety of IMSB301 monotherapy in Type 1 Interferonopathy patients. The dose of IMSB301 will be based on the recommended Phase Ib dose from Phase Ia.
The following methodology applies to all patients (unless otherwise indicated):
- On Day 1 the first dose of IMSB301 will be administered.
- Subjects will be observed for a minimum period of 6 hours after the dose, and unless acute safety or tolerability issues emerge, they will be discharged and dispensed with study drug sufficient to last until the next visit (Day 8).
- The morning and evening doses will be separated by ~12 hours, and administered at approximately the same time daily.
- Subjects will be treated as out-patients and will return to the clinic at least once weekly. Subsequent study site visits will be morning of Days 8, 15, 22 and 29.
- The subject will have three further visits during the 28-day follow-up period, on Days 36, 43 and 57 (EOS).
- Continued treatment with IMSB301 beyond Day 28 may be considered on a case-by-case basis for subjects benefiting from treatment in the opinion of the Investigator, as outlined in Appendix B.
- Conditions
- Type 1 Interferonopathies
- Being tested
- IMSB301
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IMSB301Drug
Twice daily administration for 28 days.
Subjects will be treated as out-patient and will return to the clinic at least once weekly on Days 8, 15, 22 and 29.
Continued treatment beyond Day 28 may be considered on a case-by-case basis for subjects benefiting from treatment.
| Study group | Role | What they receive |
|---|---|---|
| IMSB301 Treatment | Experimental | IMSB301 Monotherapy Receives: Drug: IMSB301 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Drug-related safety
Incidence of subjects with Adverse Events
Measured: 57 days
Secondary outcomes (6)
Target engagement as measured by Pharmacokinetics
Peak plasma concentration (Cmax)
Measured: 29 Days
Target engagement as measured by Pharmacokinetics
Trough plasma concentration (Cmin)
Measured: 29 Days
Target engagement as measured by Pharmacokinetics
Area under the plasma concentration versus time curve (AUC)
Measured: 29 Days
Target engagement as measured by Pharmacodynamics
Changes to interferon score based on 6 interferon signature gene (ISG) expression levels in whole blood
Measured: 43 Days
Target engagement as measured by Pharmacodynamics
Determination of interferon alpha (IFNa) protein levels in plasma
Measured: 43 Days
Target engagement as measured by Pharmacodynamics
Changes to Interferon beta (IFNb) expression levels in plasma
Measured: 43 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2026
- Study start12 Jan 2026 (estimated)
- Primary completion30 Jul 2026 (estimated)
- Study completion30 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type 1 InterferonopathiesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ImmuneSensor Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07364513
- Sponsor's protocol ID
- IMSB301-101
- Status last verified
- Jan 2026
- First submitted
- 13 Jan 2026
- Last updated
- 23 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Open-Label Study of IMSB301 in Subjects With a Type 1 Interferonopathy
- ClinicalTrials.gov ID
- NCT07364513
- Acronym / sponsor ID
- IMSB301-101
- Conditions
- Type 1 Interferonopathies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- ImmuneSensor Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Jan 2026 (estimated)
- Primary completion
- 30 Jul 2026 (estimated)
- Study completion
- 30 Jul 2026 (estimated)
- First posted
- 23 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Open-Label Study (Phase Ib) of Type 1 Interferonopathy patients receiving IMSB301 monotherapy.
Detailed description
This is an open-label, Phase Ib study designed to evaluate the safety of IMSB301 monotherapy in Type 1 Interferonopathy patients. The dose of IMSB301 will be based on the recommended Phase Ib dose from Phase Ia.
The following methodology applies to all patients (unless otherwise indicated):
- On Day 1 the first dose of IMSB301 will be administered.
- Subjects will be observed for a minimum period of 6 hours after the dose, and unless acute safety or tolerability issues emerge, they will be discharged and dispensed with study drug sufficient to last until the next visit (Day 8).
- The morning and evening doses will be separated by ~12 hours, and administered at approximately the same time daily.
- Subjects will be treated as out-patients and will return to the clinic at least once weekly. Subsequent study site visits will be morning of Days 8, 15, 22 and 29.
- The subject will have three further visits during the 28-day follow-up period, on Days 36, 43 and 57 (EOS).
- Continued treatment with IMSB301 beyond Day 28 may be considered on a case-by-case basis for subjects benefiting from treatment in the opinion of the Investigator, as outlined in Appendix B.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IMSB301 Treatment | Experimental | Drug: IMSB301IMSB301 Monotherapy |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- First subject must be at least 16 years of age and weighing at least 50 kg. Remaining subjects must be at least 12 years of age and weighing at least 40 kg.
- Molecular diagnosis of one of the following:
- Clinical syndrome consistent with type 1 interferonopathy diagnosis based on clinical, CSF, or radiological findings.
- Women of child-bearing potential (defined as a female who has experienced menarche and who has not undergone successful surgical sterilization [hysterectomy, bilateral salpingectomy, or bilateral oophorectomy]) or is not postmenopausal (defined as amenorrhea for at least 12 consecutive months with an appropriate clinical profile at the appropriate age, e.g., greater than 45 years) must have a negative serum pregnancy test at screening and a negative urine pregnancy test on Day -1.
- Sexually active male and female subjects with reproductive potential must agree to use highly effective contraception or agree to abstain from heterosexual intercourse throughout the study and for at least 3 months after last study drug dose.
Key exclusion criteria (20 listed)
- Presence of other significant neurological disorder that is not related to type 1 interferonopathy, brain tumor or other space-occupying lesion, or history of severe head injury.
- The presence of significant concomitant disease that would, in the judgement of the Investigator, pose additional risk to the subject, interfere with the assessment of safety and tolerability, or significantly interfere with the metabolic disposition of study drug.
- BMI above 33 kg/m2 or a total body weight in excess of 130 kg.
- Have any of the following infection risks:
- Resting 12-lead ECG showing confirmed prolongation of QTc (Fridericia's correction) interval (QTc interval > 470 for females and >450 for males), or other baseline ECG abnormalities that, in the judgement of the Investigator, would pose additional safety risk to the subject.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IMSB301Twice daily administration for 28 days. Subjects will be treated as out-patient and will return to the clinic at least once weekly on Days 8, 15, 22 and 29. Continued treatment beyond Day 28 may be considered on a case-by-case basis for subjects benefiting from treatment. | Drug | IMSB301 Treatment |
Primary outcome
Drug-related safety
Time frame: 57 days
Incidence of subjects with Adverse Events
6 secondary outcomes
Target engagement as measured by Pharmacokinetics
Time frame: 29 Days
Target engagement as measured by Pharmacokinetics
Time frame: 29 Days
Target engagement as measured by Pharmacokinetics
Time frame: 29 Days
Target engagement as measured by Pharmacodynamics
Time frame: 43 Days
Target engagement as measured by Pharmacodynamics
Time frame: 43 Days
Target engagement as measured by Pharmacodynamics
Time frame: 43 Days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing IMSB301 in people with Type 1 Interferonopathies, run by ImmuneSensor Therapeutics Inc. at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Drug-related safety (time frame: 57 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2026.