FH-Deficient RCCNCT05535829
Molecular Markers in Predicting Response to Treatment in FH-deficient RCC Patients
An observational study of FH-Deficient RCC and Systemic Treatments, run by RenJi Hospital at 1 site in China.
Trial summary
Fumarate hydratase-deficient renal cell carcinoma (FH-deficient RCC) is a rare subtype of RCC characterized by germline/somatic mutation of the fumarate hydratase (FH) gene, and is an extremely aggressive tumor, with a propensity to disseminate early even in the setting of a small primary tumor. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- FH-Deficient RCC, Systemic Treatments, Biomarkers
- What is being tested?
- Laboratory analysis of samples
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2022to 1 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with advanced FH-deficient RCC who have given prior consent for their samples and data to be used, and who have received immunotherapy-based treatment
Who may be able to join
- ≥18 years old;
- histopathological evidence of FH-deficient RCC, which was confirmed by Sanger or next-generation sequencing after initial screening by IHC.
- included patients must be diagnosed with metastatic renal cell carcinoma or have a TNM stage IV (according to 2009 TNM Classification);
- new FH-deficient RCC patients who has scheduled to start 1st cycle of systemic treatment;
- ECOG score ≤2;
- life expectancy ≥ 3 months;
- sign informed consent, and be able to follow the visit and related procedures stipulated in the program;
- agree to collect tumor tissue, blood and other specimens required by this study and apply them to relevant studies;
Who cannot join
- patients with other malignant tumors with different primary sites or histology from the tumor evaluated in this study within 2 years of personal history.
- major surgery or severe trauma within 4 weeks before enrollment;
- known or suspected active autoimmune diseases (congenital or acquired), such as interstitial pneumonia, uveitis, enteritis, hepatitis, pituitary inflammation, vasculitis, nephritis, thyroiditis, etc. Patients with type 1 diabetes with good insulin control can also be enrolled.
- known allogeneic organ transplantation (except corneal transplantation) or allogeneic hematopoietic stem cell transplantation;
- allergic to any component of monoclonal antibody;
- suffering from other uncontrolled serious diseases, including but not limited to: A) severe infection in the active phase or clinically poorly controlled; B) HIV infection (HIV antibody positive); C) acute or chronic active hepatitis b (HBsAg positive and HBV DNA>1*103/ml) or acute or chronic active hepatitis c (HCV antibody positive and HCV RNA>15IU/ml); D) active tuberculosis, etc.;
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- class iii-iv congestive heart failure (New York heart association classification), poorly controlled and clinically significant arrhythmia;
- uncontrolled arterial hypertension (systolic blood pressure ≥160mmHg or diastolic blood pressure ≥100mmHg);
- pregnant or lactating women.
Trial locations and contacts
1 site in 1 country.
Jin Zhang contact
China1 site, 1 recruiting
- Recruiting
Ethics Committee of Shanghai Renji Hospital
Shanghai, Shanghai Municipality
Qi Lu, +86021-68383364, rjllb3364@163.com
About this study
Fumarate hydratase-deficient renal cell carcinoma (FH-deficient RCC) is a rare subtype of RCC characterized by germline/somatic mutation of the fumarate hydratase (FH) gene, and is an extremely aggressive tumor, with a propensity to disseminate early even in the setting of a small primary tumor. This project is a real-world exploratory study aiming to explore potential molecular markers detectable at baseline that can enable the prediction of clinical efficacy of systemic treatments in advanced FH-deficient RCC.
This project is a real-world exploratory study aiming to explore potential molecular markers detectable at baseline that can enable the prediction of clinical efficacy of immunotherapy combined with target therapy in advanced FH-deficient RCC.
Read more
This study aims to include a total of 100 patients initially diagnosed with advanced FH-deficient RCC. Paired tissue and blood samples collected from all patients before or/ and after the start of immunotherapy-based treatment (at diagnosis or/ and their change with treatment) will be analyzed.
The patient samples will be submitted for molecular analysis, including next-generation sequencing (NGS)-based gene expression profiling (GEP), RNA-sequencing, multiplex immunofluorescence staining and inflammation-related T-cell receptor (TCR) repertoire profiling, ect. The molecular assay results will include but will not be limited to tumor mutation burden (TMB), microsatellite instability (MSI) status, DNA damage repair (DDR)-related gene mutation status, and programmed death-ligand 1 (PD-L1) expression level. Patients will be followed-up for treatment responses until radiological confirmation of disease progression to immunotherapy-based treatment. The molecular assay results will then be analyzed with clinical data including objective responses and progression-free survival outcomes, among others, to identify molecular markers at baseline that are associated with clinical efficacy of immunotherapy-based treatment.
- Conditions
- FH-Deficient RCC, Systemic Treatments, Biomarkers
- Being tested
- Laboratory analysis of samples
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Laboratory analysis of samplesOther
Laboratory analysis of samples
| Study group | Role | What they receive |
|---|---|---|
| Patients with FH-deficient RCC | Laboratory analysis of samples Receives: Other: Laboratory analysis of samples |
What is being measured
The primary outcome is the main question the study is designed to answer.
Systemic treatment OS
Systemic treatment OS was defined as the time from the start of systemic treatment to death from any cause, and patients without a recorded death were right censored to the date of last clinical visit or clinical record.
Measured: Through study completion, an average of 3 year
Secondary outcomes (3)
First-line PFS
First-line progression free survival (PFS) was defined as the time from the start of first-line systemic treatment to the time of radiographic progression, or death from any cause, whichever occurred first.
Measured: Through study completion, an average of 3 year
ORR
Objective response rate (ORR) was defined as complete response (CR)+ partial response (PR)
Measured: Through study completion, an average of 3 year
DCR
disease control rate (DCR) was defined as partial response (PR)+complete response (CR)+stable disease (SD)
Measured: Through study completion, an average of 3 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2022
- Study start1 Aug 2022
- Primary completion1 Dec 2024 (estimated)
- Study completion1 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FH-Deficient RCCStudies of the same condition, open and under way.
- More trials run by RenJi HospitalThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- RenJi Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Tongji Hospital, Changzhou No.2 People's Hospital, Ruijin Hospital, Second Affiliated Hospital, Zhejiang University, School of Medicine, Shanghai 10th People's Hospital, First Affiliated Hospital of Fujian Medical University, Shanghai Zhongshan Hospital, Jiangxi Provincial People's Hopital, First Affiliated Hospital, Sun Yat-Sen University, Zhejiang Provincial People's Hospital, Peking University First Hospital, Zhejiang University, Fudan University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05535829
- Sponsor's protocol ID
- RENJIFHRCC
- Status last verified
- Aug 2023
- First submitted
- 15 Aug 2022
- Last updated
- 31 Aug 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Molecular Markers in Predicting Response to Treatment in FH-deficient RCC Patients
- ClinicalTrials.gov ID
- NCT05535829
- Acronym / sponsor ID
- RENJIFHRCC
- Conditions
- FH-Deficient RCC, Systemic Treatments, Biomarkers
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- RenJi HospitalUniversity, hospital or other
- Collaborators
- Tongji Hospital, Changzhou No.2 People's Hospital, Ruijin Hospital, Second Affiliated Hospital, Zhejiang University, School of Medicine, Shanghai 10th People's Hospital, First Affiliated Hospital of Fujian Medical University, Shanghai Zhongshan Hospital, Jiangxi Provincial People's Hopital, First Affiliated Hospital, Sun Yat-Sen University, Zhejiang Provincial People's Hospital, Peking University First Hospital, Zhejiang University, Fudan University
- Study start
- 1 Aug 2022 (actual)
- Primary completion
- 1 Dec 2024 (estimated)
- Study completion
- 1 Dec 2024 (estimated)
- First posted
- 10 Sep 2022 (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.
Fumarate hydratase-deficient renal cell carcinoma (FH-deficient RCC) is a rare subtype of RCC characterized by germline/somatic mutation of the fumarate hydratase (FH) gene, and is an extremely aggressive tumor, with a propensity to disseminate early even in the setting of a small primary tumor. This project is a real-world exploratory study aiming to explore potential molecular markers detectable at baseline that can enable the prediction of clinical efficacy of systemic treatments in advanced FH-deficient RCC.
This project is a real-world exploratory study aiming to explore potential molecular markers detectable at baseline that can enable the prediction of clinical efficacy of immunotherapy combined with target therapy in advanced FH-deficient RCC.
This study aims to include a total of 100 patients initially diagnosed with advanced FH-deficient RCC. Paired tissue and blood samples collected from all patients before or/ and after the start of immunotherapy-based treatment (at diagnosis or/ and their change with treatment) will be analyzed.
The patient samples will be submitted for molecular analysis, including next-generation sequencing (NGS)-based gene expression profiling (GEP), RNA-sequencing, multiplex immunofluorescence staining and inflammation-related T-cell receptor (TCR) repertoire profiling, ect. The molecular assay results will include but will not be limited to tumor mutation burden (TMB), microsatellite instability (MSI) status, DNA damage repair (DDR)-related gene mutation status, and programmed death-ligand 1 (PD-L1) expression level. Patients will be followed-up for treatment responses until radiological confirmation of disease progression to immunotherapy-based treatment. The molecular assay results will then be analyzed with clinical data including objective responses and progression-free survival outcomes, among others, to identify molecular markers at baseline that are associated with clinical efficacy of immunotherapy-based treatment.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with FH-deficient RCC | – | Other: Laboratory analysis of samplesLaboratory analysis of samples |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with advanced FH-deficient RCC who have given prior consent for their samples and data to be used, and who have received immunotherapy-based treatment
Key inclusion criteria (8 listed)
- ≥18 years old;
- histopathological evidence of FH-deficient RCC, which was confirmed by Sanger or next-generation sequencing after initial screening by IHC.
- included patients must be diagnosed with metastatic renal cell carcinoma or have a TNM stage IV (according to 2009 TNM Classification);
- new FH-deficient RCC patients who has scheduled to start 1st cycle of systemic treatment;
- ECOG score ≤2;
Key exclusion criteria (9 listed)
- patients with other malignant tumors with different primary sites or histology from the tumor evaluated in this study within 2 years of personal history.
- major surgery or severe trauma within 4 weeks before enrollment;
- known or suspected active autoimmune diseases (congenital or acquired), such as interstitial pneumonia, uveitis, enteritis, hepatitis, pituitary inflammation, vasculitis, nephritis, thyroiditis, etc. Patients with type 1 diabetes with good insulin control can also be enrolled.
- known allogeneic organ transplantation (except corneal transplantation) or allogeneic hematopoietic stem cell transplantation;
- allergic to any component of monoclonal antibody;
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 |
|---|---|---|
| Laboratory analysis of samplesLaboratory analysis of samples | Other | Patients with FH-deficient RCC |
Primary outcome
Systemic treatment OS
Time frame: Through study completion, an average of 3 year
Systemic treatment OS was defined as the time from the start of systemic treatment to death from any cause, and patients without a recorded death were right censored to the date of last clinical visit or clinical record.
3 secondary outcomes
First-line PFS
Time frame: Through study completion, an average of 3 year
ORR
Time frame: Through study completion, an average of 3 year
DCR
Time frame: Through study completion, an average of 3 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2023.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Jin Zhang contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of FH-Deficient RCC and Systemic Treatments, run by RenJi Hospital 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 8 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 31 Aug 2023. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yunze Xu (+8618801967501, xuyunze@renji.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Systemic treatment OS (time frame: Through study completion, an average of 3 year). 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 31 Aug 2023.