In breve
Clinical trials are investigating "RNA, (UM-SP-(2′-DEOXY-2′-FLUORO)A-SP-GM-AM-AM-(2′-DEOXY-2′-FLUORO)A-CM-UM-CM-AM-UM-AM-GM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)A-UM-UM-GM-CM-AM-SP-CM-SP-AM), COMPLEX WITH RNA (UM-SP-GM-SP-CM-AM-AM-UM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)C-(2′-DEOXY-2′-FLUORO)U-(2′-DEOXY-2′-FLUORO)A-UM-GM-AM-GM-UM-UM-UM-CM-UM-AM) 3′-[[(2S,4R)-1-[29-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-14,14-BIS[[3-[[3-[[5-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]PROPYL]AMINO]-3-OXOPROPOXY]METHYL]-1,12,19,25-TETRAOXO-16-OXA-13,20,24-TRIAZANONACOS-1-YL]-4-HYDROXY-2-PYRROLIDINYL]METHYL HYDROGEN PHOSPHATE] (1:1)" in adults with hereditary hemorrhagic telangiectasia (HHT). The study is looking at safety and tolerability, and it includes healthy adult volunteers and adult patients with HHT.
Punti chiave
- One clinical trial is studying "RNA, (UM-SP-(2′-DEOXY-2′-FLUORO)A-SP-GM-AM-AM-(2′-DEOXY-2′-FLUORO)A-CM-UM-CM-AM-UM-AM-GM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)A-UM-UM-GM-CM-AM-SP-CM-SP-AM), COMPLEX WITH RNA (UM-SP-GM-SP-CM-AM-AM-UM-(2′-DEOXY-2′-FLUORO)C-GM-(2′-DEOXY-2′-FLUORO)C-(2′-DEOXY-2′-FLUORO)U-(2′-DEOXY-2′-FLUORO)A-UM-GM-AM-GM-UM-UM-UM-CM-UM-AM) 3′-[[(2S,4R)-1-[29-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-14,14-BIS[[3-[[3-[[5-[[2-(ACETYLAMINO)-2-DEOXY-Β-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]PROPYL]AMINO]-3-OXOPROPOXY]METHYL]-1,12,19,25-TETRAOXO-16-OXA-13,20,24-TRIAZANONACOS-1-YL]-4-HYDROXY-2-PYRROLIDINYL]METHYL HYDROGEN PHOSPHATE] (1:1)" in adults with hereditary hemorrhagic telangiectasia, also called HHT. This is a Phase 1 interventional study with 48 participants and an authorised status. The trial also includes adult healthy volunteers, so researchers can compare results between people with HHT and people without the disease. The main focus is safety and tolerability, especially the frequency of adverse events. Safety checks also include vital signs, ECGs, and clinical laboratory assessments.
Overview of the trial
The available trial record describes an interventional study, which means participants receive a study treatment or a comparison treatment. The study is about adults with Hereditary Hemorrhagic Telangiectasia (HHT) and also includes adult healthy volunteers.
The trial is authorised and has the identification number NCT06659640.
The study title says it is a Phase 1/2 study, but the trial record also lists the phase as Phase 1.
Who can participate
The trial includes adult healthy volunteers and adult patients with HHT.
The source data does not provide more detailed eligibility rules, such as exact age limits, lab cut-offs, or disease severity requirements.
Phase and endpoints
The trial is listed as Phase 1, which usually means early testing in people.
The primary outcome is the frequency of adverse events (AEs), meaning unwanted medical events that happen during the study.
Safety is also checked with vital signs, ECGs (heart rhythm tests), and clinical laboratory assessments.
The brief summary says the study aims to evaluate the safety and tolerability of multiple doses in adult patients with HHT.
Trial summary
This trial is focused on understanding how the study treatment performs in people with HHT and in healthy adults.
It is not presented as a study of long-term disease control in the available record; instead, the main goal is early evaluation of safety-related measures.
Because the source data is limited, the most reliable details are the study phase, target population, trial status, enrollment, and the safety endpoints listed above.