Schimke Immuno-osseous DysplasiaEarly Phase 1NCT06769191
Clinical Study on the Safety and Efficacy of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in the Treatment of SIOD
A Early Phase 1 study testing CD7 CAR-T cells injection, Allo-HSCT and Kidney Transplantation in people with Schimke Immuno-osseous Dysplasia, run by Zhejiang University at 1 site in China.
Trial summary
A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in the treatment of Schimke immuno-osseous dysplasia Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Schimke Immuno-osseous Dysplasia
- What is being tested?
- CD7 CAR-T cells injection, Allo-HSCT and Kidney Transplantation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 30 Jan 2025to 30 Jan 2028
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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;
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- 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;
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The first affiliated hospital of medical college of zhejiang university
Hangzhou, Zhejiang, 310003
He Huang, MD, 0571-87233772, hehuangyu@126.com
Yongxian Hu, MD, 0571-87233772, huyongxian2000@aliyun.com
About this study
Official title: 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
A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in the treatment of Schimke immuno-osseous dysplasia
Read the detailed description
This is a single-arm, open-label, dose-escalation clinical trial to evaluate the safety and efficacy of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in patients with Schimke immuno-osseous dysplasia. It is planned to enroll 20 participants in this trial.
- Conditions
- Schimke Immuno-osseous Dysplasia
- Being tested
- CD7 CAR-T cells injection, Allo-HSCT, Kidney Transplantation
- Keywords
- CAR-T, Allo-HSCT, Kidney Transplantation
- Medical subject headings
- Schimke immunoosseous dysplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD7 CAR-T cells injectionBiological
Intravenous infusion, single dose
Allo-HSCTProcedure or surgery
allogeneic hematopoietic stem cell transplantation
Kidney TransplantationProcedure or surgery
Kidney Transplantation
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | Schimke Immuno-osseous Dysplasia Receives: Biological: CD7 CAR-T cells injection; Procedure: Allo-HSCT; Procedure: Kidney Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events
Measured: Up to 2 years after Treatment
Transplant related mortality rate
The proportion of patients who died after transplantation to the total number of transplant patients during the same period
Measured: Up to 100 days after Treatment
Secondary outcomes (5)
Allogeneic hematopoietic stem cell transplant implantation rate
The proportion of the number of patients who achieved hematopoietic reconstitution to the total number of allogeneic hematopoietic stem cell transplantation patients in the same period.
Measured: Up to 100 days after Treatment
Kidney transplantation implantation rate
The ratio of successfully implanted kidneys to the total implanted kidneys
Measured: Up to 100 days after Treatment
Time to neutrophil and platelet engraftment
The time for neutrophils and platelets to reach the implantation criteria after stem cell reinfusion
Measured: Up to 30 days after Treatment
Disease-feesurvival，DFS
The proportion of disease-free patients who survived to the total number of patients who transplanted allogeneic hematopoietic stem cells during the same period.
Measured: Up to 2 years after Treatment
Overall survival, OS
After transplantation until death from any cause.
Measured: Up to 2 years after Treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2025
- Study start30 Jan 2025 (estimated)
- Primary completion30 Jan 2028 (estimated)
- Study completion30 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Schimke Immuno-osseous DysplasiaStudies of the same condition, open and under way.
- More trials run by Zhejiang UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Responsible party
- Principal investigator, He Huang, First Affiliated Hospital of Zhejiang University
- Investigators
- He Huang, MD, Principal investigator, Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06769191
- Sponsor's protocol ID
- TXB2024022
- Status last verified
- Jan 2025
- First submitted
- 2 Jan 2025
- Last updated
- 20 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06769191
- Acronym / sponsor ID
- TXB2024022
- Conditions
- Schimke Immuno-osseous Dysplasia
- Keywords
- CAR-T, Allo-HSCT, Kidney Transplantation
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Study start
- 30 Jan 2025 (estimated)
- Primary completion
- 30 Jan 2028 (estimated)
- Study completion
- 30 Jan 2028 (estimated)
- First posted
- 10 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in the treatment of Schimke immuno-osseous dysplasia
Detailed description
This is a single-arm, open-label, dose-escalation clinical trial to evaluate the safety and efficacy of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in patients with Schimke immuno-osseous dysplasia. It is planned to enroll 20 participants in this trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Biological: CD7 CAR-T cells injection, Procedure: Allo-HSCT, Procedure: Kidney TransplantationSchimke Immuno-osseous Dysplasia |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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%；
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD7 CAR-T cells injectionIntravenous infusion, single dose | Biological | Treatment Group |
| Allo-HSCTallogeneic hematopoietic stem cell transplantation | Procedure or surgery | Treatment Group |
| Kidney TransplantationKidney Transplantation | Procedure or surgery | Treatment Group |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Up to 2 years after Treatment
Incidence of treatment-emergent adverse events
Transplant related mortality rate
Time frame: Up to 100 days after Treatment
The proportion of patients who died after transplantation to the total number of transplant patients during the same period
5 secondary outcomes
Allogeneic hematopoietic stem cell transplant implantation rate
Time frame: Up to 100 days after Treatment
Kidney transplantation implantation rate
Time frame: Up to 100 days after Treatment
Time to neutrophil and platelet engraftment
Time frame: Up to 30 days after Treatment
Disease-feesurvival，DFS
Time frame: Up to 2 years after Treatment
Overall survival, OS
Time frame: Up to 2 years after Treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2025.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
He Huang, MD principal investigator
Zhejiang University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CD7 CAR-T cells injection, Allo-HSCT and Kidney Transplantation in people with Schimke Immuno-osseous Dysplasia, run by Zhejiang University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact He Huang, MD (057187233772, hehuangyu@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) (time frame: Up to 2 years after Treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2025.