A Multi-center, Phase 1b Clinical Study of an Autologous Anti-CD20/BCMA Chimeric Antigen Receptor T-Cell Therapy (C-CAR168) for the Treatment of Systemic Sclerosis (SSc) Refractory to Standard Therapy
AbelZeta Inc.
Summary
This Phase 1b, multicenter, open-label study will evaluate the safety and efficacy of a single infusion of autologous anti-CD20/BCMA chimeric antigen receptor T cells (C-CAR168) following lymphodepleting chemotherapy in participants with refractory Systemic Sclerosis (SSc) who are not responding to standard therapy. Approximately 6-12 participants will undergo leukapheresis, lymphodepletion with fludarabine and cyclophosphamide, and infusion of C-CAR168. Participants will be followed for 24 months (2 years) to evaluate safety, CAR T-cell persistence, pharmacokinetics/pharmacodynamics, biomarkers, and preliminary efficacy results. Long-term safety follow-up for gene therapy-related events will continue for up to 15 years following CAR T-cell infusion.
Eligibility
- Age range
- 18–70 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Able to sign and date the Informed Consent Form (ICF). 2. Female or male, aged 18-70 years (inclusive) with body weight ≥40 kg. 3. Have a clinical diagnosis of diffuse cutaneous SSc according to the 2013 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification. 4. Disease duration between the onset of the first non-Raynaud's sign/symptom and ICF signing shall be ≤ 6 years. 5. SSc Disease Activity meet all following criteria: 1. Modified Rodnan Skin Score (mRSS) ≥15. 2. Chest High Resolution Computed Tomography (HRCT) show…
Interventions
- BiologicalC-CAR168
Autologous anti-CD20/BCMA chimeric antigen receptor T-cell therapy administered as a single intravenous infusion
Location
- Research SiteGilbert, Arizona