AuthorisedHuman Pharmacology (Phase I)- OtherInterventional

Safety and Preliminary Efficacy of CTX112 in Adult Participants with Relapsed/Refractory Hematologic Autoimmune Disease

This study is called CTX112 and is for adults who have specific autoimmune diseases that affect their blood. In these conditions, the body's immune system mistakenly attacks healthy blood cells. The people joining this study have tried standard treatments, but these haven't worked well enough or their condition has come back. This research is a very early-stage study, meaning it's the first time this new treatment, CTX112, is being given to humans. The main goals are to carefully check if the treatment is safe to use and to see if there are any early signs that it might be effective in improving the condition. It's a key first step in understanding if CTX112 could be a helpful option in the future.

At a glance

Status

Authorised

Phase

Human Pharmacology (Phase I)- Other

Sponsor

CRISPR Therapeutics AG

Enrolment target

Start

12 Mar 2026

Relapsed/Refractory Hematologic Autoimmune Disease

What is this study about?

You might have heard of autoimmune diseases, where your body's defence system (your immune system) gets confused and starts attacking its own healthy cells. In this particular study, we're focusing on a group of these conditions that affect your blood. These are often called 'hematologic autoimmune diseases'. For people with these conditions, their bodies might be attacking their red blood cells, white blood cells, or platelets, which are all vital parts of your blood.

Sometimes, standard treatments like medications don't work as well as we'd hope, or the disease might come back after treatment. When this happens, it's called 'relapsed' or 'refractory', meaning it hasn't responded to the usual ways of helping. This is where clinical trials like this one come in. They aim to find new and potentially better ways to treat these difficult conditions.

This study, which involves a new treatment called CTX112, is in its very early stages. This means it's the first time researchers are giving CTX112 to people. The main purpose at this point is to see if the treatment is safe for people to take and to get an idea of whether it shows any promising signs of helping with the disease. It's a careful and monitored step to learn more about CTX112 and its potential for the future.

Key takeaways

It's an early-stage study for a new treatment (CTX112).
It's for adults with specific autoimmune blood diseases that haven't responded to other treatments.
The main goals are to check safety and look for early signs of effectiveness.
Participation involves regular hospital visits, tests, and closely monitored treatment.
You can stop participating at any time.

Who may be eligible?

To be able to join this study, you need to be an adult, aged 18 years or older. There is no upper age limit, so people of all adult ages can be considered.

This study is open to both men and women. The most important thing is that you have one of the specific blood-related autoimmune diseases that the researchers are focusing on. Also, these diseases must not have responded well to previous treatments, or they must have come back after treatment.

The research team will have a more detailed list of things they look for to make sure the study is right and safe for you. This often includes checking your general health and specific aspects of your condition. They will talk you through all of these points clearly.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with a blood-related (hematologic) autoimmune disease?
Has your condition not improved with standard treatments, or has it returned?
Are you willing to attend regular hospital appointments and tests?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve regular visits to the hospital or clinic. During these visits, you would have various tests, such as blood tests, and check-ups with the study doctors and nurses, to monitor your health and how you’re responding to the treatment. You would receive the study medication, CTX112, as instructed by the medical team. The study team would explain the exact schedule of treatment and assessments, which might change depending on how you're doing. After receiving the main treatment, there will be follow-up appointments to track your long-term health and the effects of the treatment. The total time you would be involved in the study would be explained in detail by the research team.

Potential risks and benefits

As with any new treatment, there are potential benefits and potential risks. The potential benefit of taking part is that you might have access to a new treatment that could help your condition, especially if other treatments haven't worked well for you. However, because this is an early-stage study, we don't know for certain if it will be effective, and there could be side effects. These side effects would be closely monitored and managed by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Spain
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Unverified
Germany

Common questions

What does 'relapsed/refractory' mean?

It means your disease has come back after treatment, or it hasn't responded well to standard treatments.

Is this a new type of treatment?

Yes, CTX112 is a new treatment being tested in people for the first time in this study.

Will I definitely get the active drug?

In this early phase study, it's common for all participants to receive the active study treatment, but the research team will confirm this with you.

What makes an autoimmune disease 'hematologic'?

It means the autoimmune disease specifically affects your blood cells – like red blood cells, white blood cells, or platelets.

Can I continue my regular medications during the study?

The study team will review all your current medications and advise you on which ones you can continue or if any need to be paused.