In short
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, with checks such as vital signs, ECGs, and lab tests.
Key points
- Clinical trials are 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 (HHT). The available trial is a Phase 1 study and is authorised. It includes adult healthy volunteers and adult patients with HHT. The main goal is to assess safety and tolerability of multiple doses. Researchers are also measuring adverse events, vital signs, ECGs, and clinical laboratory tests. The study has an enrollment of 48 participants.
Overview of the clinical trial
The available study is an interventional trial, which means researchers are giving a study treatment and then measuring what happens. It is studying adults with hereditary hemorrhagic telangiectasia (HHT).
The trial title says it is a Phase 1/2 study, but the trial record provided here lists the phase as Phase 1. The brief summary says the study is designed to evaluate the safety and tolerability of multiple doses of the study drug in adult patients with HHT.
Who the trial includes
The trial record says the study includes adult healthy volunteers and adult patients with HHT. The condition listed is HHT, so the main patient group is adults who have this disease.
The enrollment listed for the study is 48 participants.
Trial phase and design
This study is in Phase 1, which is an early stage of clinical research. Early phase trials mainly focus on learning whether a treatment can be given safely and how people tolerate it.
The trial status is Authorised. The intervention list includes the study drug given as a subcutaneous injection, which means an injection under the skin, and phosphate-buffered saline for subcutaneous administration.
What the researchers are measuring
The primary outcome is the frequency of adverse events (AEs). Adverse events are medical problems that happen during a study, whether or not they are caused by the study treatment.
Safety is also being checked with vital signs, ECGs, and clinical laboratory assessments. Vital signs are basic health checks such as blood pressure and pulse, ECG means a heart tracing test, and laboratory assessments are tests on blood or other samples.
What this means for patients
For patients, this trial is mainly about learning whether the study treatment can be given safely in adults with HHT. The study is not described here as testing long-term benefit, so the focus is on early safety information first.
Because the trial is early stage, the most important results will be about side effects seen during the study, along with changes in heart tests, lab tests, and other safety checks.