In short
Clinical trials are investigating ATTR-01 in adults with select epithelial solid tumours. These studies aim to evaluate safety, tolerability, and anti-tumour activity, and some sub-protocols also look for the best dose for further development. The main study is a Phase 1/2 interventional trial.
Key points
- ATTR-01 is being studied in a Phase 1/2 clinical trial for adults with select epithelial solid tumours. The study is authorised and is designed as a master protocol with multiple sub-protocols. Researchers are looking at safety, tolerability, and anti-tumour activity, and some parts of the study also aim to find the optimal dose for future development. The main outcomes include side effects, serious side effects, dose limiting toxicities, changes in vital signs, objective response rate, and duration of response. The trial plans to enroll 60 participants.
Trial overview
The available study is titled A Phase 1-2 Master Protocol to study intravenous ATTR-01 in adult participants with select epithelial solid tumours under multiple sub-protocols, also called ATTEST.
This is an interventional study, which means participants receive the study treatment and the research team measures the results.
The trial is currently listed as Authorised.
Who is being studied
The trial is designed for adult participants with select epithelial solid tumours.
“Epithelial” means the cancer starts in cells that line organs or body surfaces, and “solid tumours” means the cancer forms a mass or lump rather than being a blood cancer.
The source data does not give more detailed inclusion or exclusion rules, so the exact patient group is limited to the information above.
What the study measures
The main safety outcomes include the incidence of adverse events, which means how often unwanted medical problems happen during the study.
Researchers also measure serious adverse events, dose limiting toxicities, stopping the investigational product because of toxicity, and clinically significant changes in vital signs or other safety checks.
The study also looks at Objective Response Rate (ORR) from the first scan onward, using RECIST V1.1, which is a standard way to measure whether tumours shrink or disappear on scans.
Another key endpoint is Duration of Response (DoR), which shows how long a tumour response lasts once it begins.
The brief summary says the trial aims to evaluate safety and tolerability in select solid epithelial tumours, and also anti-tumour activity in these participants, although this does not apply to SP A.
Study design and phases
This is a Phase 1/2 trial, which usually means an early study that first focuses on safety and then starts to look at whether the treatment may help the cancer.
The trial uses a master protocol, meaning one main study plan can include several related sub-protocols.
Some sub-protocols include dose escalation, which means the dose may be increased step by step to help determine the optimal dose for further development.
The source data notes that this dose-finding goal applies where relevant for those sub-protocols, but not to every part of the trial.
Trial status and size
The trial has an enrollment target of 60 participants.
This number shows the planned study size, which helps researchers collect enough information on safety and early signs of activity.
Because the study is authorised and still in an early phase, the main focus is on learning how ATTR-01 performs in the target cancer group rather than proving final treatment benefit.