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Not yet recruitingPhase 1

C-CAR168, an Autologous Anti-CD20/BCMA Chimeric Antigen Receptor T-Cell Therapy, in Participants With Systemic Sclerosis (SSc) Refractory to Standard Therapy

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 70 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 15 years
  • Procedures mentioned: Infusions

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Approximately 6-12 participants will undergo leukapheresis, lymphodepletion with fludarabine and cyclophosphamide, and infusion of C-CAR168."
  • "Long-term safety follow-up for gene therapy-related events will continue for up to 15 years following CAR T-cell infusion."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the safety of C-CAR168 and how well it works for adults with systemic sclerosis that has not improved with standard treatment. This disease causes skin to harden and can affect organs. The treatment uses a person's own immune cells, changed to target certain cells, and is given once after chemotherapy.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Can sign and date the study consent form.
  • Are 18–70 years old and weigh at least 40 kg (about 88 pounds).
  • Have diffuse cutaneous systemic sclerosis, a form that causes widespread skin thickening, under the study's diagnosis rules.
  • Had your first disease symptom within 6 years before signing consent, not counting Raynaud's symptoms (fingers or toes changing color with cold or stress).
  • Have all three: a skin thickening score of at least 15, hazy areas linked to the disease on a chest scan, and a breathing capacity or lung gas-transfer test below 70% of the expected value.
  • Test positive for at least one of these disease-linked antibodies (immune proteins): anti-Scl70, anti-RNA polymerase III, anti-centromere, anti-Th/To, or anti-U3RNP.
  • Tried at least two standard treatments that suppress or change the immune system, each for at least 6 months, without lasting improvement.
  • Can lower steroid medicines to the doses allowed by the study.

Who can't join

  • Have breathing capacity below 45% or lung gas transfer below 40% of the expected value.
  • Have severe high blood pressure in the lung arteries on a heart ultrasound, or a recent heart catheter test showing average lung artery pressure above 45 mmHg.
  • Have important chest scan findings that are not caused by systemic sclerosis.
  • Have skin hardening that makes it too hard to reach veins for cell collection, treatment, other study procedures, or emergency care.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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) shows SSc related ground-glass opacities. 3. Percent predicted forced vital capacity (ppFVC)\<70% or precent predicted (diffusing capacity of the lungs for carbon monoxide) ppDLco\<70%. 6. Positivity for at least one SSc-specific autoantibodies: 1. anti-Scl70 antibody. 2. anti-RNA polymerase III antibody. 3. anti-centromere antibody. 4. anti-Th/To antibody. 5. and anti-U3RNP antibody. 7. Refractory to at least two prior standard-of-care regimens, including immunosuppressives and/or a biologic note: "Refractory" is defined as receiving each listed drug for ≥6 months with either no response, disease progression after transient remission, or persistent disease progression on treatment. 8. Ability to taper steroids to protocol allowed doses Exclusion Criteria: 1. ppFVC\<45% or ppDLco \<40%. 2. Severe pulmonary arterial hypertension (PAH) as measured by screening echocardiogram (ECHO), or had documented mean positive airway pressure PAP (mPAP) \> 45mmHg on recent right heart catheterization (RHC). Note: RHC is not mandatory at screening, however participant with high-risk ECHO findings warrant close clinical vigilance, and participant with mPAP\> 45mmHg at baseline shall not proceed with lymphodepletion. 3. Presence of clinically significant abnormalities on chest high-resolution computed tomography (HRCT) that are UNRELATED to SSc. 4. Cutaneous sclerosis that impedes venous access required for leukapheresis, lymphodepletion, C-CAR168 infusion, other study procedures and emergency rescue interventions.

Study sites (1)

AZ