CTX112 for Lupus

Recruiting at 6 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

Trial Summary

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.

What data supports the effectiveness of the drug CTX112 for treating lupus?

Research shows that cyclophosphamide (CTX), a component of CTX112, has been effective in improving outcomes for lupus nephritis, a kidney condition related to lupus. Additionally, regulatory T cells, which are part of CTX112, have shown promise in maintaining disease remission in lupus-prone mice. ^{1 2 3 4 5}

What is the purpose of this trial?

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 refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).

Eligibility Criteria

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.

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

Pregnant or lactating

Timeline

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Towards a novel clinical outcome assessment for systemic lupus erythematosus: first outcomes of an international taskforce. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Performance of systemic lupus erythematosus responder index for detecting clinician-rated responders in patients with active systemic lupus erythematosus. [2023]

3. England pubmed.ncbi.nlm.nih.gov

CD4+Foxp3+ regulatory T cells prolong drug-induced disease remission in (NZBxNZW) F1 lupus mice. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Top 10 things to know about lupus activity measures. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

The GSTA1 polymorphism and cyclophosphamide therapy outcomes in lupus nephritis patients. [2015]