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)

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.

Table of contents

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.[1]

The trial is authorised and has the identification number NCT06659640.[1]

The study title says it is a Phase 1/2 study, but the trial record also lists the phase as Phase 1.[1]

Who can participate

The trial includes adult healthy volunteers and adult patients with HHT.[1]

The source data does not provide more detailed eligibility rules, such as exact age limits, lab cut-offs, or disease severity requirements.[1]

Phase and endpoints

The trial is listed as Phase 1, which usually means early testing in people.[1]

The primary outcome is the frequency of adverse events (AEs), meaning unwanted medical events that happen during the study.[1]

Safety is also checked with vital signs, ECGs (heart rhythm tests), and clinical laboratory assessments.[1]

The brief summary says the study aims to evaluate the safety and tolerability of multiple doses in adult patients with HHT.[1]

Trial summary

This trial is focused on understanding how the study treatment performs in people with HHT and in healthy adults.[1]

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.[1]

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.[1]

Trial IDPhaseCondition studiedStatusEnrollment
NCT06659640Phase 1Hereditary Hemorrhagic Telangiectasia (HHT)Authorised48

Sperimentazioni cliniche in corso su 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)

Glossario

  • Hereditary Hemorrhagic Telangiectasia (HHT): The condition being studied in this trial. It is the disease named in the trial record.
  • Healthy volunteers: Adults without the disease who join a study so researchers can compare results.
  • Adult patients: People aged 18 years or older who have the condition being studied.
  • Phase 1: An early stage of clinical research. It is usually used to first check safety in people.
  • Interventional study: A study where participants receive a treatment or comparison intervention.
  • Safety: How well a treatment can be used without causing unacceptable problems.
  • Tolerability: How well people can handle a treatment, based on how it affects them.
  • Adverse events (AEs): Unwanted medical events that happen during a study, whether or not they are caused by the study treatment.
  • Vital signs: Basic body measurements such as pulse, blood pressure, and temperature.
  • ECG: An electrocardiogram, a test that records the heart’s electrical activity.
  • Clinical laboratory assessments: Blood or other lab tests used to help check health and safety during a study.