NF2 DeficiencyNCT06834438
Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002
A study testing ST002 in people with NF2 Deficiency, run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences at 1 site in China.
Trial summary
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors. ST002 injection is a gene therapy product designed for NF2. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- NF2 Deficiency
- What is being tested?
- ST002
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 15 Feb 2025to 1 Feb 2027
- Phase
- Interventional
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- age ≥16 years old, gender not limited;
- Patients must meet the diagnostic criteria for NF2 gene mutations, which are benign or malignant solid tumors with confirmed NF2 gene mutations or no expression of the NF2 protein product Merlin in peripheral blood or tumor tissue, and no standard treatment or with standard treatment failure;
- at least one measurable and injectable superficial lesion (according to RECIST or itRECIST criteria);
- Having sufficient organ and bone marrow function:
- Blood routine (no transfusion or treatment with colony-stimulating factor within 14 days): neutrophil count (ANC) ≥ 1.5 × 10 9 /L, hemoglobin (Hb) ≥ 90g/L, platelet count (PLT) ≥ 75 × 10 9 /L; White blood cell count (WBC)>3.0 × 10 9 /L;
- Liver function: Serum total bilirubin (TBIL) ≤ 1.5 × ULN, ALT≤2.5×ULN， AST ≤ 2.5 × ULN (ALT ≤ 5 × ULN, AST ≤ 5 × ULN in patients with liver metastasis);
- Renal function: serum creatinine (Cr) ≤ 1.5 × ULN, or creatinine clearance rate (CrCl) ≥ 50ml/min; d) Coagulation function: Prothrombin time (PT) ≤ 1.5 × ULN, activated partial thromboplastin time (APTT) ≤ 1.5 × ULN, international normalized ratio (INR) ≤ 1.5 × ULN;
- Patients must voluntarily participate in clinical trials, demonstrate adherence to the study protocol, cooperate well with researchers, and sign a written informed consent form.
Who cannot join
- Has participated in or is currently participating in other clinical studies NF2 targeting gene therapy;
- History of severe neurological disorders such as epilepsy (excluding neurofibromatosis);
- Patients who are allergic to lentiviral vectors and their excipients;
- Patients with poorly controlled hypertension (systolic blood pressure>150 mmHg and/or diastolic blood pressure>100 mmHg under regular medication control), as well as those with a history of hypertensive crisis or hypertensive brain disease;
- Suffering from unstable angina or acute myocardial infarction, or having a history of both within the past six months;
- Patients with a history of malignant tumors within the past 5 years, except for cured basal cell carcinoma, squamous cell carcinoma of the skin, cervical cancer, and gastrointestinal cancer;
- During the study period, premenopausal women who are pregnant, breastfeeding, has a positive pregnancy test, or are unwilling to take contraceptive measures.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Open- Label, Single-arm, Dose-escalation Pilot Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ST002 in the Treatment of Patients with NF2 Mutation-related Solid Tumors
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors. ST002 injection is a gene therapy product designed for NF2. By reinserting the normal XXX gene into genetically deficient tumor cells, the product expresses Merlin. This regulates gene transcription in tumor cells, controls the tumor microenvironment, and inhibits tumor growth and invasion, achieving therapeutic effects.
Read the detailed description
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors. Using a 3+3 dose escalation design, three dose groups are formulated, and three to six patients are expected to be enrolled in each dose group. A total of nine to eighteen - patients with NF2 gene mutation related solid tumors will be enrolled.
- Conditions
- NF2 Deficiency
- Being tested
- ST002
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ST002Genetic
Single multi-point intra-tumoral injection of ST002, a lentiviral vector designed to drive expression of the NF2 protein product, Merlin.
| Study group | Role | What they receive |
|---|---|---|
| ST002 | Experimental | Subjects will receive with a single multi-point intra-tumoral injection of ST002 from 1x10^7 to 1x10^8 TU into the tumor. Receives: Genetic: ST002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety evaluation
Evaluate the incidence of drug-related adverse events (AEs) and adverse events (SAEs) from baseline to Week 24 after administration, with a focus on neurotoxicity and genome-wide integration carcinogenic risks.
Measured: From baseline to Week 24 after administration
Secondary outcomes (2)
Efficacy evaluation
Evaluate the objective response rate (ORR) (tumor volume reduction) and duration of response (DOR) according to RECIST and itRECIST
Measured: Day 21 after administration, and at the end of study follow-up, and at early withdrawal. The frequency of checks during the research period can be adjusted according to the researcher's judgement.
Efficacy evaluation
Percentage of necrosis of tumor cells in the tumor tissue at the injection site, estimated by pathological HE staining, at Day 21 after administration.
Measured: Day 21 after injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2025
- Study start15 Feb 2025 (estimated)
- Primary completion1 Feb 2026 (estimated)
- Study completion1 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NF2 DeficiencyStudies of the same condition, open and under way.
- More trials run by Cancer Institute and Hospital, Chinese Academy of Medical SciencesThe 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, NING LI, Cancer Institute and Hospital, Chinese Academy of Medical Sciences
- Investigators
- Shuhang Wang, PhD, Study director, National Cancer Center of China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06834438
- Sponsor's protocol ID
- ST002-IIT-2024-01
- Status last verified
- Feb 2025
- First submitted
- 7 Feb 2025
- Last updated
- 19 Feb 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 19 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open- Label, Single-arm, Dose-escalation Pilot Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ST002 in the Treatment of Patients with NF2 Mutation-related Solid Tumors
- ClinicalTrials.gov ID
- NCT06834438
- Acronym / sponsor ID
- ST002-IIT-2024-01
- Conditions
- NF2 Deficiency
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical SciencesUniversity, hospital or other
- Study start
- 15 Feb 2025 (estimated)
- Primary completion
- 1 Feb 2026 (estimated)
- Study completion
- 1 Feb 2027 (estimated)
- First posted
- 19 Feb 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.
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors. ST002 injection is a gene therapy product designed for NF2. By reinserting the normal XXX gene into genetically deficient tumor cells, the product expresses Merlin. This regulates gene transcription in tumor cells, controls the tumor microenvironment, and inhibits tumor growth and invasion, achieving therapeutic effects.
Detailed description
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors. Using a 3+3 dose escalation design, three dose groups are formulated, and three to six patients are expected to be enrolled in each dose group. A total of nine to eighteen - patients with NF2 gene mutation related solid tumors will be enrolled.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ST002 | Experimental | Genetic: ST002Subjects will receive with a single multi-point intra-tumoral injection of ST002 from 1x10^7 to 1x10^8 TU into the tumor. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- age ≥16 years old, gender not limited;
- Patients must meet the diagnostic criteria for NF2 gene mutations, which are benign or malignant solid tumors with confirmed NF2 gene mutations or no expression of the NF2 protein product Merlin in peripheral blood or tumor tissue, and no standard treatment or with standard treatment failure;
- at least one measurable and injectable superficial lesion (according to RECIST or itRECIST criteria);
- Having sufficient organ and bone marrow function:
- Patients must voluntarily participate in clinical trials, demonstrate adherence to the study protocol, cooperate well with researchers, and sign a written informed consent form.
Key exclusion criteria (7 listed)
- Has participated in or is currently participating in other clinical studies NF2 targeting gene therapy;
- History of severe neurological disorders such as epilepsy (excluding neurofibromatosis);
- Patients who are allergic to lentiviral vectors and their excipients;
- Patients with poorly controlled hypertension (systolic blood pressure>150 mmHg and/or diastolic blood pressure>100 mmHg under regular medication control), as well as those with a history of hypertensive crisis or hypertensive brain disease;
- Suffering from unstable angina or acute myocardial infarction, or having a history of both within the past six months;
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 |
|---|---|---|
| ST002Single multi-point intra-tumoral injection of ST002, a lentiviral vector designed to drive expression of the NF2 protein product, Merlin. | Genetic | ST002 |
Primary outcome
Safety evaluation
Time frame: From baseline to Week 24 after administration
Evaluate the incidence of drug-related adverse events (AEs) and adverse events (SAEs) from baseline to Week 24 after administration, with a focus on neurotoxicity and genome-wide integration carcinogenic risks.
2 secondary outcomes
Efficacy evaluation
Time frame: Day 21 after administration, and at the end of study follow-up, and at early withdrawal. The frequency of checks during the research period can be adjusted according to the researcher's judgement.
Efficacy evaluation
Time frame: Day 21 after injection
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 19 Feb 2025.
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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.
Shuhang Wang, PhD study director
National Cancer Center of China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ST002 in people with NF2 Deficiency, run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences 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 7 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: opening soon, as of the sponsor's last update on 19 Feb 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.
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 Ning Li, M.D. (+86 01087788919, lining@cicas.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety evaluation (time frame: From baseline to Week 24 after administration). 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 19 Feb 2025.