CTX112 for Lupus

Recruiting at 9 trial locations

Overseen By Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: CRISPR Therapeutics

Must not be taking: Anti-CD19 therapy

Disqualifiers: Organ transplant, CNS involvement, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 4 weeks

80 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the safety and early effectiveness of CTX112, an experimental treatment for stubborn autoimmune diseases such as systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM). The treatment involves an IV infusion administered after a specific type of chemotherapy that prepares the body. It targets individuals diagnosed with one of these conditions who experience active symptoms affecting the skin or lungs. Participants should not have received certain prior treatments or have conditions like severe brain involvement or recent cancer. The trial aims to determine if CTX112 can help manage these challenging autoimmune diseases. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that CTX112 is likely to be safe for humans?

Research has shown that CTX112, a potential treatment for autoimmune diseases like lupus, appears safe. Early results suggest most patients tolerate CTX112 well, with side effects typically mild and manageable.

Since CTX112 remains in the early testing stages, scientists continue to study its safety. Reaching this stage indicates promising earlier lab results. Prospective trial participants should know that researchers will closely monitor them to manage any potential side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for lupus?

Unlike the standard treatments for lupus, such as corticosteroids and immunosuppressive drugs, CTX112 is a cutting-edge experimental therapy that uses engineered cells to target the condition. Researchers are excited about CTX112 because it involves a novel approach called CAR-T cell therapy, which has been successful in other autoimmune diseases. This therapy is administered via IV infusion following lymphodepleting chemotherapy, potentially offering a more precise and powerful way to manage lupus symptoms by directly modifying immune cells.

What evidence suggests that CTX112 might be an effective treatment for lupus?

Research has shown that CTX112, the investigational treatment in this trial, could benefit autoimmune diseases. Early data suggest it might be safe and effective for conditions like lupus. In this trial, researchers administer CTX112 following lymphodepleting chemotherapy, which includes cyclophosphamide—a drug that has improved outcomes for lupus nephritis, a kidney condition related to lupus. These early findings offer hope that CTX112 might be effective for systemic lupus erythematosus (SLE) and similar diseases. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

Adults aged 18-70 with certain autoimmune diseases (like lupus, scleroderma, or myositis) that haven't responded to other treatments can join. They must be able to follow the study plan and use birth control. People with recent serious infections, certain past illnesses like cancer within 5 years, or those who've had organ transplants can't participate.

Inclusion Criteria

Subjects must voluntarily sign a written informed consent and be willing and able to comply with all study requirements

My blood, kidney, liver, heart, and lung functions are all within normal ranges.

Subjects must agree to use acceptable methods of contraception

Exclusion Criteria

I have a history of specific infections.

Pregnant or lactating

I have had a severe reaction to anti-phospholipid syndrome.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Lymphodepleting Chemotherapy

Participants receive lymphodepleting chemotherapy prior to CTX112 infusion

1 week

Treatment

CTX112 is administered by IV infusion following lymphodepleting chemotherapy

4 weeks

1 visit (in-person) for infusion

Follow-up

Participants are monitored for safety and effectiveness after CTX112 infusion

60 months

What Are the Treatments Tested in This Trial?

Interventions

• CTX112

Trial Overview The trial is testing CTX112's safety and how well it works for people with tough-to-treat autoimmune diseases. Everyone in the study gets this drug; there's no comparison group. The doses may increase as the study goes on to see what level is safe and effective.

How Is the Trial Designed?

Group I: CTX112 Experimental Treatment 1 Intervention

Administered by IV infusion following lymphodepleting chemotherapy

Who Is Running the Clinical Trial?

CRISPR Therapeutics

Lead Sponsor

Trials

7

Recruited

630+

Published Research Related to This Trial

In a study of 77 lupus nephritis patients, those with the GSTA1*A heterozygous mutation had a higher risk of not achieving remission from cyclophosphamide therapy (44% vs. 20%).

Pharmacokinetic analysis showed that patients with the GSTA1*A variant had lower levels of the active metabolite 4-hydroxycyclophosphamide, which was linked to poorer treatment outcomes, indicating that genetic factors can influence the effectiveness of cyclophosphamide in lupus nephritis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The GSTA1 polymorphism and cyclophosphamide therapy outcomes in lupus nephritis patients. Wang, HN., Zhu, XY., Zhu, Y., et al.[2015]

In a study of 27 patients with active systemic lupus erythematosus (SLE), various treatment outcome measures, including SRI-50 and SLE-DAS, showed comparable accuracy in identifying patient responders based on physician assessments, with overall accuracies ranging from 64.6% to 75.0%.

For patients with lupus nephritis, the SRI-50 and SLE-DAS demonstrated even higher accuracy rates of 82.6%, indicating that these measures are effective tools for evaluating treatment responses in SLE, although no significant differences were found between the different measures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Performance of systemic lupus erythematosus responder index for detecting clinician-rated responders in patients with active systemic lupus erythematosus. Leosuthamas, P., Narongroeknawin, P., Chaiamnuay, S., et al.[2023]

Citations

1. ir.crisprtx.com ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-presents-data-2024-american-society

CRISPR Therapeutics Presents Data at the 2024 American ... These preliminary data demonstrate that CTX112 has the potential to provide meaningful clinical benefit with a well-tolerated safety profile.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06925542

A Safety and Efficacy Study Evaluating CTX112 in Adult ... This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with ...

3. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/593835

A Safety and Efficacy Study Evaluating CTX112 in Adult ... This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with ...

4. withpower.com withpower.com/trial/phase-1-muscular-diseases-2-2025-bba11

CTX112 for Lupus · Recruiting Participants for Phase ... Research shows that cyclophosphamide (CTX), a component of CTX112, has been effective in improving outcomes for lupus nephritis, a kidney condition related to ...

5. crisprmedicinenews.com crisprmedicinenews.com/clinical-trial/refractory-autoimmune-disease-nct06925542/

Refractory Autoimmune Disease, (NCT06925542) This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult ...

6. ir.crisprtx.com ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-provides-business-update-and-reports-13

Press Release In addition, CTX112 is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the product candidate in ...

7. pharmacia.pensoft.net pharmacia.pensoft.net/article/167410/

CAR T-cell therapy in systemic lupus erythematosus Since the discovery of CARs , their structure has been continuously refined based on efficacy and safety data. Currently, five generations ...