High-grade Squamous Intraepithelial Lesions (HSIL)Early Phase 1NCT07170254
A Clinical Study on the Evaluation of BD114 for the Treatment HPV-16-Related Cervical HSIL
A Early Phase 1 study testing BD114VLP, or BD114 in people with High-grade Squamous Intraepithelial Lesions (HSIL), run by Shanghai BDgene Co., Ltd. at 1 site in China.
Trial summary
This study is intented to evaluate the safety, and tolerability and preliminary efficacy of Instantaneous CRISPR/Cas9 Gene Editing Therapy (BD114 virus-like particle, also BD114) for the treatment of high-grade squamous intraepithelial lesions (HSIL) associated with HPV-16 infection. Read more about this study
- Who is it for?
- Ages 18 to 50 years, women only
- Conditions the study is for
- High-grade Squamous Intraepithelial Lesions (HSIL)
- What is being tested?
- BD114VLP, or BD114
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 15 Sep 2025to 21 Aug 2026
- Phase
- Early Phase 1
- 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 to 50 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Female, aged 18 to 50 years, without childbearing demand;
- Confirmed histopathological evidence of cervical HSIL (CIN3) at the screening period or within 1 month prior to screening;
- Confirmed cervical HPV-16 positive by HPV test during the screening period or within 1 month prior to screening, without other high-risk HPV types detected;
- Complete visibility of both the squamocolumnar junction (SCJ Types 1/2) and the upper margin of acetate-white epithelium or suspected HSIL lesions under colposcopy examine during the screening period or within 1 month prior to screening;
- No evidence of precancerous lesions or malignancy by endocervical curettage (ECC) ;
- The biopsy sampling of cervical lesions are performed;
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- Visible residual cervical lesions after screening biopsy;
- Willing to maintain abstinence or use a highly effective contraceptive method (oral contraceptives, injections, implants, or barrier methods) for women of childbearing potential (WOCBP) from enrollment until Week 36, or her partner undergone surgical sterilization (e.g., vasectomy);
- Good compliance to protocol-specified procedure in study duration assessed by investigator;
- Voluntarily participating in the study and willing to provided signed informed consent.
Who cannot join
- Positive detection of antibodies or viral test of human immunodeficiency virus (HIV), or hepatitis B virus (HBV), or hepatitis C virus (HCV), or Treponema pallidum (TP) at screening;
- Confirmed histopathologically epithelial carcinoma, glandular carcinoma or precancerous lesions in the endometrium at screening;
- Confirmed histopathologically or macroscopically high-grade intraepithelial neoplasia or invasive carcinoma in the vulva, vagina or anus at screening;
- HSIL with partial location in cervical canal and incomplete colposcopic visualization ;
- Undergone the treatment for cervical HSIL within 4 weeks prior to screening;
- Vaccination history of any therapeutic HPV vaccine;
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- Family history of malignancy, or a history/current presence of any malignant tumor;
- Severe uncontrolled diseases of major organs, including but not limited to: acute myocardial infarction, stroke, liver cirrhosis, severe kidney disease, diabetes mellitus, chronic obstructive pulmonary disease (COPD), hematologic disorders, psychiatric disorders, etc;
- Pregnant (a positive urine or serum pregnancy test) or lactating women;
- Participating in another drug or device clinical trial at screening, or participated in one within 3 months prior to screening;
- The history of any form of gene and/or cell therapy;
- Drug abuse or alcohol addiction no compliance to protocol-specific procedure;
- Any other condition unsuitable for participating this study judged by the investigator.
Trial locations and contacts
1 site in 1 country.
China1 site
Obstetrics & Gynecology Hospital of Fudan University
Shanghai, 200090
About this study
Official title: An Open-Label, Two-Arm, Non-Randomized Clinical Study On The Safety And Efficacy Of Instantaneous CRISPR/Cas9 Gene Editing Therapy For Treating Chinese Patients With HPV-16-Related High-Grade Squamous Intraepithelial Lesions (HSIL)
This study is intented to evaluate the safety, and tolerability and preliminary efficacy of Instantaneous CRISPR/Cas9 Gene Editing Therapy (BD114 virus-like particle, also BD114) for the treatment of high-grade squamous intraepithelial lesions (HSIL) associated with HPV-16 infection.
Read the detailed description
This study is an open-label, two-arm, balanced-group, single-dose, non-randomized exploration clinical study. A total of 6 patients with HPV-16 Related HSIL of the Cervix will be enrolled and divided into two arms. Participant in Arm 1(n=1) receive topically low-dose of BD114 with intraepithelial injection of lesions, and participants in Arm 2 (n=5) receive high-dose of BD114 with intraepithelial injection of lesions. The total follow-up duration for each participant is 40 weeks (including screening stage). The treatment-emergent adverse events (TEAEs) for safe evaluation, components detection of the BD114 for pharmacokinetics (PK) assessment, histologically lesion regression or clearness and virologically HPV-16 clearness for efficacy evaluation are conducted and explored during follow-up visits.
- Being tested
- BD114VLP, or BD114
- Keywords
- High-grade squamous intraepithelial lesions, HPV-16 infection, Gene Editing Therapy
- Medical subject headings
- Squamous Intraepithelial Lesions
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BD114VLP, or BD114Genetic
CRISPR/Cas9 instantaneous gene editing therapy, called BD114VLP (also BD114) which is a developing product of gene therapy from modified third-generation integrated defective lentivirus, can deliver gRNA/Cas9 ribonucleoprotein complex (RNP). It works to knock out or knock down HPV-16 E6/E7 genes integrated in HSIL cell genome resulting to lesion clearness or regression.
A single dosing BD114 injection by topical intraepithelial injection of HSIL lesion.
| Study group | Role | What they receive |
|---|---|---|
| BD114 Low-dose group | Experimental | BD114 Low-dose group: Low-dose (n=1)
* Small lesions (≤5 mm^2): BD114 injection, 0.2 mL
* Large lesions (>5 mm^2): BD114 injection 0.4 mL Receives: Genetic: BD114VLP, or BD114 |
| BD114 High-dose group | Experimental | BD114 High-dose group: High-dose (n=5)
* Small lesions (≤5 mm^2): BD114 injection, 0.3 mL
* Large lesions (>5 mm^2): BD114 injection 0.6 mL Receives: Genetic: BD114VLP, or BD114 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events (AEs) and serious adverse events (SAEs)
All AEs and SAEs occurring during the study period (from informed consent signing to Week 36 post-BD114 intervention) were recorded and assessed according to the NCI-CTCAE v5.0 grading system, including their classification by System Organ Class (SOC), severity grade, number of affected Participants, and incidence rates. Gynecological AEs (beyond NCI-CTCAE v5.0 specifications) shall be evaluated by investigators for classification and severity grading according to current clinical guidelines/expert consensus.
Measured: ~36 weeks
Percentage of Participants with No Histologic Evidence of HSIL
Cervical tissue biopsies obtained at Week 24 and Week 36 undergo histopathological evaluation, and judge the HSIL Clearance by complete regression or disappear of the original lesions, with clearance rates calculated per the following formula:
Percentage of Participants with No Histologic Evidence of HSIL (%) = Number of Participants Achieving HSIL Clearance / Total number of Participants treated with BD114 ×100%
Measured: Week 24, Week 36
Secondary outcomes (8)
Percentage of Participants with No Histologic Evidence of HSIL and No Evidence of HPV-16
Virological HPV-16 assessment was performed using clinically validated type-specific HPV testing kits, meanwhile colposcopy-guided cervical tissue biopsies are histopathologically evaluated. The calculating formula as follow:
Percentage of Participants with No Histologic Evidence of HSIL and No Evidence of HPV-16 = Number of Participants with No Histologic Evidence of HSIL and No Evidence of HPV-16 / Total number of Participants treated with BD114 ×100%
Measured: Week 24, Week 36
Percentage of Participants with No Evidence of HPV-16
Histological samples (cervical exfoliated cells) is detected for HPV-16 by real-time fluorescent quantifying PCR technology. The calculating formula as follow:
Percentage of Participants with No Evidence of HPV-16 = Number of Participants with No Evidence of HPV-16 / Total number of Participants treated with BD114 ×100%
Measured: Week 24, Week 36
Percentage of Participants with No Evidence of HSIL or LSIL
Cervical biopsies at Week 36 are performed for histopathological evaluation, no histologically evidence of HSIL or LSIL of histological samples are judged as no histology findings of HSIL or LSIL. The calculating formula as follow:
Percentage of Participants with No Evidence of HSIL or LSIL = Number of Participants with No Evidence of HSIL or LSIL / Total number of Participants treated with BD114 ×100%
Measured: Week 36
Percentage of Participants with No Evidence of HSIL or LSIL and No Evidence of HPV-16
Cervical biopsies at Week 36 are performed for histopathological evaluation, no histologically evidence of HSIL or LSIL of histological samples are judged as no histology findings of HSIL or LSIL. Histological samples (cervical exfoliated cells) is detected HPV-16 by real-time fluorescent quantifying PCR technology.
Calculation Formula:
Percentage of Participants with No Evidence of HSIL or LSIL and No Evidence of HPV-16 = Number of Participants A with No Evidence of HSIL or LSIL and No Evidence of HPV-16 / Total number of Participants treated with BD114 ×100%
Measured: Week 36
Changes in Serum Inflammatory Cytokine (IL-1/IL-6/TNF-α/IFN-γ) Levels Relative to Baseline
Blood samples were collected and analyzed using clinically validated ELISA kits to quantify serum levels of four inflammatory cytokines (IL-1, IL-6, TNF-α, and IFN-γ).
Measured: Day0, Day 2, Week 1, Week 2, Week 4, Week 12, Week 24, and Week 36.
Concentrations of Cas9 Protein and gRNA in Peripheral Blood
The concentration of Cas9 protein are tested by ELISA and gRNA are detected by qPCR in blood.
Measured: At Day0, Day 2, Week 1, Week 4, Week 12.
Detection of Anti-p24/Cas9 Protein Antibodies in Peripheral Blood
The detection of anti-p24/Cas9 protein antibodies in blood are performed at scheduled study visits.
Measured: At Day0, Week 2, Week 4, Week 36
Off-Target Analysis
Off-Target analysis of cervical biopsy samples is conducted by applying DNA deep sequencing or whole genome sequencing (WGS) to assess BD114-related off-target effects based on the sequencing results.
Measured: D0, Week 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2025
- Study start15 Sep 2025
- Primary completion21 Aug 2026
- Study completion21 Aug 2026
Why it stopped: Adjustment Per devolpment strategy
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for High-grade Squamous Intraepithelial Lesions (HSIL)Studies of the same condition, open and under way.
- 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shanghai BDgene Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Obstetrics & Gynecology Hospital of Fudan University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07170254
- Sponsor's protocol ID
- BD-HSIL-114001
- Status last verified
- Aug 2026
- First submitted
- 5 Sep 2025
- Last updated
- 4 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Two-Arm, Non-Randomized Clinical Study On The Safety And Efficacy Of Instantaneous CRISPR/Cas9 Gene Editing Therapy For Treating Chinese Patients With HPV-16-Related High-Grade Squamous Intraepithelial Lesions (HSIL)
- ClinicalTrials.gov ID
- NCT07170254
- Acronym / sponsor ID
- BD-HSIL-114001
- Keywords
- High-grade squamous intraepithelial lesions, HPV-16 infection, Gene Editing Therapy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Adjustment Per devolpment strategy
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Shanghai BDgene Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Obstetrics & Gynecology Hospital of Fudan University
- Study start
- 15 Sep 2025 (actual)
- Primary completion
- 21 Aug 2026 (actual)
- Study completion
- 21 Aug 2026 (actual)
- First posted
- 12 Sep 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 study is intented to evaluate the safety, and tolerability and preliminary efficacy of Instantaneous CRISPR/Cas9 Gene Editing Therapy (BD114 virus-like particle, also BD114) for the treatment of high-grade squamous intraepithelial lesions (HSIL) associated with HPV-16 infection.
Detailed description
This study is an open-label, two-arm, balanced-group, single-dose, non-randomized exploration clinical study. A total of 6 patients with HPV-16 Related HSIL of the Cervix will be enrolled and divided into two arms. Participant in Arm 1(n=1) receive topically low-dose of BD114 with intraepithelial injection of lesions, and participants in Arm 2 (n=5) receive high-dose of BD114 with intraepithelial injection of lesions. The total follow-up duration for each participant is 40 weeks (including screening stage). The treatment-emergent adverse events (TEAEs) for safe evaluation, components detection of the BD114 for pharmacokinetics (PK) assessment, histologically lesion regression or clearness and virologically HPV-16 clearness for efficacy evaluation are conducted and explored during follow-up visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BD114 Low-dose group | Experimental | Genetic: BD114VLP, or BD114BD114 Low-dose group: Low-dose (n=1) * Small lesions (≤5 mm^2): BD114 injection, 0.2 mL * Large lesions (>5 mm^2): BD114 injection 0.4 mL |
| BD114 High-dose group | Experimental | Genetic: BD114VLP, or BD114BD114 High-dose group: High-dose (n=5) * Small lesions (≤5 mm^2): BD114 injection, 0.3 mL * Large lesions (>5 mm^2): BD114 injection 0.6 mL |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Female, aged 18 to 50 years, without childbearing demand;
- Confirmed histopathological evidence of cervical HSIL (CIN3) at the screening period or within 1 month prior to screening;
- Confirmed cervical HPV-16 positive by HPV test during the screening period or within 1 month prior to screening, without other high-risk HPV types detected;
- Complete visibility of both the squamocolumnar junction (SCJ Types 1/2) and the upper margin of acetate-white epithelium or suspected HSIL lesions under colposcopy examine during the screening period or within 1 month prior to screening;
- No evidence of precancerous lesions or malignancy by endocervical curettage (ECC) ;
Key exclusion criteria (13 listed)
- Positive detection of antibodies or viral test of human immunodeficiency virus (HIV), or hepatitis B virus (HBV), or hepatitis C virus (HCV), or Treponema pallidum (TP) at screening;
- Confirmed histopathologically epithelial carcinoma, glandular carcinoma or precancerous lesions in the endometrium at screening;
- Confirmed histopathologically or macroscopically high-grade intraepithelial neoplasia or invasive carcinoma in the vulva, vagina or anus at screening;
- HSIL with partial location in cervical canal and incomplete colposcopic visualization ;
- Undergone the treatment for cervical HSIL within 4 weeks prior to screening;
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 |
|---|---|---|
| BD114VLP, or BD114CRISPR/Cas9 instantaneous gene editing therapy, called BD114VLP (also BD114) which is a developing product of gene therapy from modified third-generation integrated defective lentivirus, can deliver gRNA/Cas9 ribonucleoprotein complex (RNP). It works to knock out or knock down HPV-16 E6/E7 genes integrated in HSIL cell genome resulting to lesion clearness or regression. A single dosing BD114 injection by topical intraepithelial injection of HSIL lesion. | Genetic | BD114 High-dose group, BD114 Low-dose group |
Primary outcome
Adverse events (AEs) and serious adverse events (SAEs)
Time frame: ~36 weeks
All AEs and SAEs occurring during the study period (from informed consent signing to Week 36 post-BD114 intervention) were recorded and assessed according to the NCI-CTCAE v5.0 grading system, including their classification by System Organ Class (SOC), severity grade, number of affected Participants, and incidence rates. Gynecological AEs (beyond NCI-CTCAE v5.0 specifications) shall be evaluated by investigators for classification and severity grading according to current clinical guidelines/expert consensus.
Percentage of Participants with No Histologic Evidence of HSIL
Time frame: Week 24, Week 36
Cervical tissue biopsies obtained at Week 24 and Week 36 undergo histopathological evaluation, and judge the HSIL Clearance by complete regression or disappear of the original lesions, with clearance rates calculated per the following formula: Percentage of Participants with No Histologic Evidence of HSIL (%) = Number of Participants Achieving HSIL Clearance / Total number of Participants treated with BD114 ×100%
8 secondary outcomes
Percentage of Participants with No Histologic Evidence of HSIL and No Evidence of HPV-16
Time frame: Week 24, Week 36
Percentage of Participants with No Evidence of HPV-16
Time frame: Week 24, Week 36
Percentage of Participants with No Evidence of HSIL or LSIL
Time frame: Week 36
Percentage of Participants with No Evidence of HSIL or LSIL and No Evidence of HPV-16
Time frame: Week 36
Changes in Serum Inflammatory Cytokine (IL-1/IL-6/TNF-α/IFN-γ) Levels Relative to Baseline
Time frame: Day0, Day 2, Week 1, Week 2, Week 4, Week 12, Week 24, and Week 36.
Concentrations of Cas9 Protein and gRNA in Peripheral Blood
Time frame: At Day0, Day 2, Week 1, Week 4, Week 12.
Detection of Anti-p24/Cas9 Protein Antibodies in Peripheral Blood
Time frame: At Day0, Week 2, Week 4, Week 36
Off-Target Analysis
Time frame: D0, Week 1
Results and safety
The study was stopped early (reason given: Adjustment Per devolpment strategy). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 BD114VLP, or BD114 in people with High-grade Squamous Intraepithelial Lesions (HSIL), run by Shanghai BDgene Co., Ltd. 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 10 inclusion and 13 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: stopped early, as of the sponsor's last update on 4 Sep 2026. 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?
The study was stopped early (reason given: Adjustment Per devolpment strategy). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (AEs) and serious adverse events (SAEs) (time frame: ~36 weeks). 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 4 Sep 2026.