Treatment for Schimke Immuno-osseous Dysplasia using CAR-T and Kidney Transplantation

Clinical Study on the Safety and Efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation and Kidney Transplantation in the Treatment of Schimke Immuno-osseous Dysplasia

This study is testing a new treatment that combines special immune cells and kidney transplants to see if it can help people with Schimke immuno-osseous dysplasia who have severe kidney disease.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sex: All
• Sponsor: Zhejiang University (Academic / other)
• Drugs / interventions: alemtuzumab, car-t, cyclophosphamide
• Locations: 1 site (Hangzhou, Zhejiang)
• Trial ID: NCT06769191 on ClinicalTrials.gov

What this trial studies

This clinical study evaluates the safety and efficacy of a novel treatment approach combining CD7 CAR-T cell therapy with sequential allogeneic hematopoietic stem cell transplantation (Allo-HSCT) and kidney transplantation for patients diagnosed with Schimke immuno-osseous dysplasia (SIOD). It is a single-arm, open-label, dose-escalation trial aiming to enroll 20 participants. The study will assess the outcomes of this combined treatment strategy in patients who are in stage 5 of chronic kidney disease. Participants will receive CD7 CAR-T cell injections followed by Allo-HSCT and kidney transplantation.

Who should consider this trial

Good fit: Ideal candidates for this study are patients diagnosed with Schimke immuno-osseous dysplasia who are in stage 5 of chronic kidney disease and have suitable donors for transplantation.

Not a fit: Patients with active pulmonary infections or those who do not meet the eligibility criteria for transplantation may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve kidney function and overall health outcomes for patients with SIOD.

How similar studies have performed: While the combination of CAR-T therapy and transplantation is a novel approach, similar studies have shown promise in treating other conditions, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

* 1\. Diagnosed as SIOD and was in stage 5 of chronic kidney disease
* 2\. Having allogeneic HSCT indications, at least suitable donors (relatives) for haploidentical allogeneic transplantation and kidneys from stem cell transplantation donors;
* 3\. serum total bilirubin ≤ 1.5 times the upper limit of normal, and serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were both ≤ 3 times the upper limit of the normal range.
* 4\. Echocardiogram shows left ventricular ejection fraction (LVEF) ≥ 50%;
* 5\. There is no active pulmonary infection, and the oxygen saturation during air inhalation is more than 92%；
* 6\. Estimated survival time ≥ 3 months;
* 7\. ECOG performance status 0 to 1;
* 8\. Pregnant/lactating women, or male or female patients who have fertility and are willing to take effective contraceptive measures at least 6 months after the last cell infusion during the study period;
* 9\. Those who voluntarily participated in this trial and provided informed consent;

Exclusion Criteria:

* 1\. Allergic to pretreatment measures
* 2\. received any containing ATG/ALG such IST、alemtuzumab、high-dose cyclophosphamide (≥ 45mg/kg/day) , received CsA treatment within 6 months, or used thrombopoietin receptor (tpo-r) agonists in the past;
* 3\. Patients with the history of epilepsy or other CNS disease;
* 4\. Patients with prolonged QT interval time or severe heart disease;
* 5\. Previous recipients of allogeneic hematopoietic stem cell transplantation or organ transplantation
* 6\. People infected with HIV, active hepatitis B or hepatitis C virus, and patients with active infection who are not cured;
* 7\. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
* 8\. Patients with malignant tumor;
* 9\. People with other genetic diseases；
* 10\. After receiving CD7 car-t treatment, patients who were unable to accept subsequent kidney transplantation due to severe infection or poor amplification of car-t in vivo.
* 11\. Any situation that researchers believe may increase the risk to the subjects or interfere with the trial results.

Where this trial is running

Hangzhou, Zhejiang

• The first affiliated hospital of medical college of zhejiang university — Hangzhou, Zhejiang, China (Recruiting)

Study contacts

• Principal investigator: He Huang, MD — Zhejiang University
• Study coordinator: He Huang, MD

Conditions Schimke Immuno-osseous Dysplasia; CAR-T; Allo-HSCT; Kidney Transplantation