- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT07854483
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
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.
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Studietype
Registrering (Antatt)
Fase
- Fase 1
Kontakter og plasseringer
Studiekontakt
- Navn: Kirstin Liechty, Head of Clinical Operations, PhD
- Telefonnummer: 267-566-6081
- E-post: kirstin.liechty@abelzeta.com
Studer Kontakt Backup
- Navn: Yihong Liechty, Head of Clinical Operations, PhD
- Telefonnummer: 267-566-6081
- E-post: kirstin.liechty@abelzeta.com
Studiesteder
-
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Arizona
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Gilbert, Arizona, Forente stater, 85234
- Research Site
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
Key Inclusion Criteria:
-
Able to sign and date the Informed Consent Form (ICF).
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Female or male, aged 18-70 years (inclusive) with body weight ≥40 kg.
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Have a clinical diagnosis of diffuse cutaneous SSc according to the 2013 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification.
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Disease duration between the onset of the first non-Raynaud's sign/symptom and ICF signing shall be ≤ 6 years.
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SSc Disease Activity meet all following criteria:
- Modified Rodnan Skin Score (mRSS) ≥15.
- Chest High Resolution Computed Tomography (HRCT) shows SSc related ground-glass opacities.
- Percent predicted forced vital capacity (ppFVC)<70% or precent predicted (diffusing capacity of the lungs for carbon monoxide) ppDLco<70%.
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Positivity for at least one SSc-specific autoantibodies:
- anti-Scl70 antibody.
- anti-RNA polymerase III antibody.
- anti-centromere antibody.
- anti-Th/To antibody.
- and anti-U3RNP antibody.
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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.
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Ability to taper steroids to protocol allowed doses
Exclusion Criteria:
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ppFVC<45% or ppDLco <40%.
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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.
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Presence of clinically significant abnormalities on chest high-resolution computed tomography (HRCT) that are UNRELATED to SSc.
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Cutaneous sclerosis that impedes venous access required for leukapheresis, lymphodepletion, C-CAR168 infusion, other study procedures and emergency rescue interventions.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: C-CAR168
Participants will receive: • Leukapheresis • Fludarabine • Cyclophosphamide • Single intravenous infusion of C-CAR168 |
Autologous anti-CD20/BCMA chimeric antigen receptor T-cell therapy administered as a single intravenous infusion |
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Incidence and severity of treatment-emergent adverse events
Tidsramme: Through Month 24
|
Incidence and severity of treatment-emergent adverse events, serious adverse events, dose-limiting toxicities, CRS, ICANS, adverse events of special interest, and treatment- or procedure-related adverse events. |
Through Month 24
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Proportion of Participants Achieving Response
Tidsramme: Through Month 24
|
Proportion of participants achieving a response per the revised Composite Response Index for Systemic Sclerosis (r-CRISS) |
Through Month 24
|
|
Pharmacokinetics of C-CAR168 Utilizing Flow Cytometry
Tidsramme: Through Month 24
|
Characterize T-cell expansion and persistence utilizing flow cytometry |
Through Month 24
|
|
Pharmacokinetics of C-CAR168 measured by quantitative polymerase chain reaction (qPCR)
Tidsramme: Through Month 24
|
Characterize CAR T-cell expansion and persistence using qPCR |
Through Month 24
|
|
Proportion of Participants Progressed
Tidsramme: Through Month 24
|
Proportion of participants that progress meeting any of the protocol defined progression criteria |
Through Month 24
|
|
Time to First Response
Tidsramme: Through Month 24
|
Time to participant meeting first response as defined in the protocol |
Through Month 24
|
|
Duration of Response (DOR)
Tidsramme: Through 24 months
|
Time of response from first response until progression as defined in the protocol |
Through 24 months
|
Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Yihong Yao, PhD, AbelZeta Inc.
Studierekorddatoer
Studer hoveddatoer
Studiestart (Antatt)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- ABZT-413
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
Individual participant data collected during this study will not be made available to other researchers.
Legemiddel- og utstyrsinformasjon, studiedokumenter
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