The ALTO study version 1.0

  • Research type

    Research Study

  • Full title

    ARTHRITIS PREVENTION IN THE PRE-CLINICAL PHASE OF RA WITH ABATACEPT (APIPPRA) LONG TERM OUTCOME STUDY: THE ALTO STUDY

  • IRAS ID

    270373

  • Contact name

    Andrew Cope

  • Contact email

    andrew.cope@kcl.ac.uk

  • Sponsor organisation

    King's College London

  • Eudract number

    2020-000108-12

  • ISRCTN Number

    ISRCTN12680338

  • Duration of Study in the UK

    2 years, 6 months, 31 days

  • Research summary

    In 2014, the first participant was consented into the Arthritis Prevention in the Preclinical Phase of RA with Abatacept (APIPPRA) clinical trial. This study set out to investigate whether rheumatoid arthritis can be prevented if targeted immunotherapy is given to subjects in whom autoantibody screening, together with symptoms of joint pain (e.g. arthralgia), indicated high risk of developing disease. In this double-blind, placebo controlled, parallel group trial, subjects were randomised to receive weekly subcutaneous injections of the costimulatory blocker CTLA4-Ig (abatacept, OrenciaTM), a biologic drug already licensed for the treatment of established RA, or placebo injections.

    After 52 weeks of study drug subjects were monitored for a further 52 weeks. The primary endpoint of the APIPPRA study was the time to development of clinical synovitis or RA.
    The purpose of the ALTO study is to capture long term clinical outcomes of all eligible APIPPRA study subjects to establish if abatacept prevents or merely delays the onset of rheumatoid arthritis, and whether treatment of at risk subjects is safe. The study duration is designed in such a way that all those enrolled will have had at least 48 months of treatment and follow up from the start of APIPPRA to the end of the ALTO study.

    Lay summary of study results: What was this study about?
    This study looked at whether a therapy already licensed for treating established rheumatoid arthritis, called abatacept, could delay or prevent rheumatoid arthritis (RA) in people deemed to be at high risk of developing the disease.
    Rheumatoid arthritis (RA) is a long-term condition where the immune system (the body’s defence system) mistakenly attacks the joints, causing pain, swelling, and damage. The study is called a randomised, double-blind, placebo-controlled trial:
     Randomised = people were put into groups by chance
     Double-blind = neither the participants nor the researchers knew who received the treatment
     Placebo-controlled = one group received a “dummy” treatment with no active drug
    The study is known as the ALTO study and the full results can be found https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Ftrack.pstmrk.it%2F3ts%2Fwww.thelancet.com%252Fjournals%252Flanrhe%252Farticle%252FPIIS2665-9913&data=05%7C02%7Clondonsoutheast.rec%40hra.nhs.uk%7Ce3e516cdc9344088f37708ded386e01d%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C639180773411293316%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=v5GOX8clX0mEQl89HxYtnChKYMEbWJ%2BwJS4RSMNREac%3D&reserved=0(25)00371-6%2Ffulltext/NBTI/CUzGAQ/AQ/58cabd05-1b54-440c-9917-2b7539798c17/1/q7rSRSWiPJ

    What did the researchers do?
    They recruited people who did not yet have rheumatoid arthritis but were at high risk of developing it. Participants were split into two groups. One group received abatacept and the other group received a placebo (inactive treatment). They followed participants over time to see how many people developed rheumatoid arthritis and how long it took for the disease to develop.

    What is abatacept?
    Abatacept is a type of medicine that affects the immune system. It works by reducing the activity of immune cells that can cause inflammation (swelling and damage in joints).

    What were the main results?
    1. Abatacept delayed the onset of rheumatoid arthritis People who received abatacept were less likely to develop rheumatoid arthritis during treatment. If they did develop the disease, it tended to happen later than in the placebo group. Abatacept reduced the symptom burden associated with the at-risk state, including reductions in joint pain and fatigue.

    2. The benefit reduced after stopping treatment After the treatment was stopped, the difference between the two groups became smaller over time. Some people who had been protected during treatment later developed rheumatoid arthritis. This means that the effect of abatacept may not be permanent. Continued or repeated treatment might be needed (this is not yet confirmed).

    3. Not everyone was prevented from getting the disease Even with treatment, some people still developed rheumatoid arthritis. The drug does not completely prevent the disease but may delay it.

    4. Safety findings
    The treatment was generally safe and well tolerated. Side effects were similar to what is already known about abatacept.

    What do these results mean?
    Key message
    Abatacept can delay the development of rheumatoid arthritis in people at high risk. However, it does not fully prevent the disease, especially after treatment stops.

    Why is this important?
    Currently, treatment for rheumatoid arthritis usually starts after symptoms appear. This study suggests it may be possible to treat people earlier, before the disease fully develops and potentially reduce long-term joint damage.

    What are the limitations?
    The protective effect reduced over time after treatment stopped. It is unclear how long treatment should continue or which patients benefit most. More research is needed before this approach becomes routine care

    Overall conclusion
    This study shows that abatacept can delay the onset of rheumatoid arthritis in people at high risk, but it does not provide a permanent cure or complete prevention. Further studies are needed to understand how best to use this treatment in the future.

  • REC name

    London - South East Research Ethics Committee

  • REC reference

    21/LO/0035

  • Date of REC Opinion

    5 Mar 2021

  • REC opinion

    Further Information Favourable Opinion