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Phase 1b Safety Study of IMSB301 in Type 1 Interferonopathies

ConditionType 1 Interferonopathies
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age12+
SponsorImmuneSensor Therapeutics Inc.

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

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

Disqualifiers

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.

Evidence of active infection during screening or on Day 1

Trial design

Design model

Single group

Treatments tested in this trial

  • IMSB301

    Drug

    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.

Treatment groups

6 Participants
are divided into 1 treatment group
Group A: IMSB301 TreatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Drug-related safety

Incidence of subjects with Adverse Events

Time frame
57 days

Secondary outcomes

1

Target engagement as measured by Pharmacokinetics

Peak plasma concentration (Cmax)

Time frame
29 Days
2

Target engagement as measured by Pharmacokinetics

Trough plasma concentration (Cmin)

Time frame
29 Days
3

Target engagement as measured by Pharmacokinetics

Area under the plasma concentration versus time curve (AUC)

Time frame
29 Days
4

Target engagement as measured by Pharmacodynamics

Changes to interferon score based on 6 interferon signature gene (ISG) expression levels in whole blood

Time frame
43 Days

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

ImmuneSensor Therapeutics Inc.

Lead sponsor

Source ClinicalTrials.gov
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