TPN-101 in Aicardi-Goutières Syndrome (AGS) (NCT05613868) | Clinical Trial Compass
TerminatedPhase 2
TPN-101 in Aicardi-Goutières Syndrome (AGS)
Stopped: Business decision
France, Italy, United Kingdom4 participantsStarted 2023-03-15
Plain-language summary
A phase 2a multi-center, open-label single dose level study of TPN-101 in Patients with Aicardi-Goutières Syndrome (AGS)
Who can participate
Age range12 Months
SexALL
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Inclusion criteria
✓. Male or female participants of the following ages:
✓. Cohort 1: Adults (≥ 18 years of age)
✓. Cohort 2: Adolescents (12 to 17 years of age)
✓. Cohort 3: Children 5 to 11 years of age
✓. Cohort 4: Children 1 to \< 5 years of age and \>= 10 kg in weight
✓. Molecular diagnosis of AGS due to biallelic mutations in 1 of the following 5 genes: TREX1, RNASEH2A, RNASEH2B, RNASEH2C, or SAMHD1, or due to a recognized dominant mutation in TREX1
✓. IFN score in peripheral blood \> 2 standard deviations above the mean score of healthy controls measured on 3 occasions, approximately 2 weeks apart, during the 6-week Screening Period.
✓. Clinical syndrome consistent with AGS diagnosis based on clinical, CSF, and radiological findings. The following are examples of such findings (none of these are required for inclusion):
Exclusion criteria
✕. Mutation in IFIH1, ADAR1, LSM11, or RNU7-1.
✕. Pre-/perinatal infections, in particular the TORCH complex (toxoplasmosis, rubella, cytomegalovirus, herpes simplex virus)
✕. Presence of other significant neurological disorders; brain tumor or other space-occupying lesion; history of severe head injury
✕
What they're measuring
1
Change in innate immune signaling
Timeframe: 48 weeks
2
Incidence and severity of spontaneously reported treatment-emergent adverse events (TEAEs) of TPN-101