Gaucher Disease Type 1Phase 1/2GDNCT06818838
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
A Phase 1/2 study testing LY-M001 in people with Gaucher Disease Type 1, run by Lingyi Biotech Co., Ltd. at 3 sites in China.
Trial summary
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate, Glucosylceramide (GlcCer), in macrophages in the liver, spleen, kidney, bone, lung, and even the… Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Gaucher Disease Type 1
- What is being tested?
- LY-M001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 3 sitesChina
- Study dates
- 5 Jul 2024to 30 Jul 2031
- Phase
- Phase 1/2
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age ≥ 18 years and ≤ 60 years, male or female.
- The subjects should fully understand the purpose, nature, and method of this study as well as possible adverse reactions, and sign the informed consent form (ICF) voluntarily.
- Patients with confirmed double mutations in the GBA1 allele through laboratory testing, and the glucocerebrosidase activity was reduced to less than 30% of the normal value(For example, the result of the dried blood spot (DBS) method is < 1.19 μmol/L/h), and meeting the standard clinical diagnosis criteria for GD1.
- Patients who meet a) or b) below:
- Treated patients with Gaucher disease type I who had previously received enzyme replacement therapy (ERT) or substrate clearance therapy (SRT) with GD, were on stable medication, eluted 5 drugs for a half-life or more before administration, or were comprehensively judged to be stable by the investigator.
- Newly treated or untreated GD1 patients who meet one or more of the following criteria at screening:
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- Hemoglobin ≥80g/L and less than the lower limit of normal;
- Platelets ≥40×10^9/L and less than the lower limit of normal;
- Hepatomegaly;
- Splenomegaly.
- Negative pregnancy test for female subjects of childbearing potential (WOCBP). Notes: WOCBP is defined as the absence of postmenopausal status (continuous amenorrhea of at least 12 months with no identifiable cause other than menopause), and the absence of surgical (i.e., ovarian, salpingectomy, and/or hysterectomy) or Investigator-determined cause of permanent infertility due to other causes (e.g., lenticular hypoplasia) after menarche in female subjects.
- Subjects and their partners have no childbearing plans from the screening period to 6 months after the end of the study, and voluntarily adopt effective contraceptive measures (e.g., abstinence, condoms, etc.); subjects have no plans to donate sperm or eggs.
- Subjects are not to donate blood during the study and for at least 1 year after the end of the study.
Who cannot join
- AAV8 neutralizing antibody positive (Antibody titer > 1:40).
- Patients with clinically diagnosed Gaucher disease type II or III (GD2 or GD3).
- Active and progressive bone disease that is expected to require surgical treatment within the next 6 months.
- Subject has idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), thrombocytopenia, anemia, hepatomegaly, splenomegaly, and/or osteoporosis unrelated to GD as judged by the Investigator.
- Treatment or disposal of investigational drugs or investigational devices received in other clinical studies within 28 days prior to screening or within 5 half-lives (drugs only), whichever is older.
- Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins that meets, but is not limited to, any of the following at the time of screening:
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- Progressive hepatomegaly larger than 3 times the normal volume.
- History of stage 2 or above liver fibrosis.
- AST, ALT, or TBIL are 1.5 times higher than ULN.
- A history of alcohol or drug abuse within the previous 2 years (defined as having consumed more than 14 standard units of alcohol per week [1 standard unit containing 14 g of alcohol, such as 360 mL beer, 45 mL spirits containing 40% or more alcohol, or 150 mL wine]).
- Hepatitis B surface antigen (HBsAg) positive and HBV deoxyribonucleic acid (HBV-DNA) positive (HBV-DNA>10^3 copy number /mL); Or take hepatitis B drugs (such as interferon, lamivudine, adefovir and entecavir); Or antibodies to hepatitis C virus (HCV) and positive for hepatitis C virus RNA.
- Human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibody positive.
- Severe hyperlipidemia (triglycerides > 11.29mol/L).
- Uncontrolled concomitant or infectious diseases (need to be determined by the investigator based on clinical practice).
- The subject has received or plans to receive bone marrow transplantation, hematopoietic stem cell transplantation and/or major organ transplantation, including but not limited to liver transplantation, kidney transplantation, etc.
- Subject has received erythropoietin, transfusion, or red blood cell transfusion within 3 months prior to screening; or platelet transfusion within 1 month prior to screening.
- Clinically diagnosed or investigator-determined serious cardiovascular disease (such as heart failure ≥3 from the New York College of Cardiology [NYHA]).
- Hypersensitivity to any component of LY-M001 injection.
- Previous treatment with any type of gene therapy or cell therapy.
- Use of systemic immunosuppressive agents or steroid therapy other than those required by the protocol for prophylactic administration within 3 months prior to dosing.
- History of cancer within 5 years prior to screening, or currently active neoplastic disease, except for basal or squamous cell carcinoma of the skin or carcinoma in situ that has been definitively treated.
- Has received a live attenuated vaccine within 4 months prior to screening or plans to receive a live attenuated vaccine during the clinical trial.
- Other conditions that, in the opinion of the Investigator, make the subject unsuitable for the study.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China3 sites, 3 recruiting
- Recruiting
Guangzhou First People's Hospital
Guangzhou, Guangdong, 510000
Shunqing Wang, PhD, 86+13437801998, Shqwang_cn@163.com
Shunqing Wang, PhD
- Recruiting
Shanxi Bethune Hospital
Taiyuan, Shanxi, 030000
Liangming Ma, PhD, 86+18634309908, maliangming620928@163.com
Liangming Ma, PhD
- Recruiting
Hematology Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300011
Fengkui Zhang, PhD, 86+13821700281, fkzhang@ihcamd.ac.cn
Fengkui Zhang, PhD
About this study
Official title: A Multicenter, Open, Single-arm, Single-dose, Dose-escalation, and Expanded Phase I/II Study Evaluating the Safety, Tolerability, and Efficacy of LY-M001 Injection in Adult Patients With Type I Gaucher Disease
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate, Glucosylceramide (GlcCer), in macrophages in the liver, spleen, kidney, bone, lung, and even the brain, inducing their transformation into Gaucher cells whose cell cytoplasm presenting a characteristic "crumpled tissue paper" appearance, leading to pathological changes in involved tissues and organs.LY-M001 Injection is an rAAV8 vector gene therapy product. It can specifically transduce the target organ liver after a single intravenous administration and express the GCase protein in liver cells for a long period of time.
Read the detailed description
The purpose of this study is to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacokinetics and pharmacokinetics of LY-M001 injection in patients with GD1 using a multicenter, open, single-arm, single-dose, dose-escalation and extended clinical design. This study includes the main study phase and the long-term follow-up study phase. The primary study period is 52 weeks after LY-M001 infusion, and the long-term follow-up period is 53 weeks to 5 years after LY-M001 infusion.Subjects who complete the 52 weeks follow up period or who prematurely withdraw in this study will enter the long-term follow-up study phase to obtain long-term assessment data.
Phase I is a dose escalation study consists of three preset dose groups, including one rollback dose group and two incremental dose groups, which are: Backdose (5 × 10^12 vg/kg) group, dose group 1 (1.5 × 10^13 vg/kg) and dose group 2 (3.0 × 10^13 vg/kg), where dose group 1 was the starting dose of the Phase I study. Three subjects are enrolled in each dose group one by one, and each subject is added to the next subject after at least 28 days of DLT observation to determine safety. Phase I studies enrolled approximately 6 to 12 (up to 12) evaluable subjects.
Phase II is a dose expansion study. After all subjects in Phase I study have completed the Day 28 (D28) observation following LY-M001 infusion, the Recommended Phase 2 Dose (RP2D) will be determined by the Safety Review Committee (SRC), which will also decide whether to proceed to the dose-expansion Phase II study.
- Conditions
- Gaucher Disease Type 1
- Being tested
- LY-M001
- Keywords
- anaemia, Gene therapy
- Medical subject headings
- Gaucher Disease, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LY-M001Genetic
Single Intravenous Infusion of LY-M001 Injection.
| Study group | Role | What they receive |
|---|---|---|
| Phase I: LY-M001 Backdose | Experimental | Participants receive a single, peripheral intravenous (IV) infusion of LY-M001 at backdose. Receives: Genetic: LY-M001 |
| Phase I: LY-M001 Dose group 1 | Experimental | Participants receive a single, peripheral intravenous (IV) infusion of LY-M001 at dose group 1. Receives: Genetic: LY-M001 |
| Phase I: LY-M001 Dose group 2 | Experimental | Participants receive a single, peripheral intravenous (IV) infusion of LY-M001 at dose group 2. Receives: Genetic: LY-M001 |
| Phase II: LY-M001 at the recommended dose | Experimental | Participants receive a single, peripheral IV infusion of LY-M001 at the recommended dose. Receives: Genetic: LY-M001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase I: Incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks after LY-M001 infusion
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
Measured: From enrollment to 52 weeks after administration
Phase I: Incidence rate of dose-limiting toxicity (DLT) events determined by the data safety review committee (SRC) within at least 28 days after LY-M001 infusion.
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.The adverse events defined as dose-limiting toxicity (DLT) have been clearly specified in the protocol.
Measured: From enrollment to 52 weeks after administration
Phase I: Liver function levels (including alanine aminotransferase [ALT], aspartate aminotransferase [AST], total bilirubin [TBIL], alkaline phosphatase [ALP], gamma-glutamyl transferase [GGT]) within 52 weeks after LY-M001 infusion.
Incidence rate of liver function-related adverse events evaluated by CTCAE V5.0.
Measured: From enrollment to 52 weeks after administration
Phase II: Blood glucocerebrosidase (GCase) activity level.
Evaluation based on the detected values of blood glucocerebrosidase（GCase).
Measured: From enrollment to 52 weeks after administration
Phase II: The incidence rates of adverse events (AEs) and serious adverse events (SAEs), as well as the occurrence of abnormalities in 12-lead electrocardiogram (ECG) findings, vital signs, laboratory test parameters, and physical examination results.
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
Measured: From enrollment to 52 weeks after administration
Secondary outcomes (15)
Phase I: Liver volume and spleen volume (if applicable)
- Change from baseline in liver volume as measured by MRI through 52 weeks after infusion of LY-M001;
- Change from baseline in spleen volume as measured by MRI through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase I: Hemoglobin levels
Evaluate the change from baseline in hemoglobin levels through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase I:Bone mineral density (BMD) after administration
Evaluate the change from baseline in bone mineral density (BMD) by dual-energy X-ray absorptiometry through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase I: Glucocerebrosidase (GCase) protein levels in blood
Evaluate the change from baseline in GCase protein levels through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase I: Glucosylsphingosine (Lyso-GL1) in blood
Evaluate the change from baseline in Lyso-GL1 levels in blood through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase I: Platelet count
Evaluate the change from baseline in platelet count through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase I: Bone marrow burden (BMB) after administration
Evaluate the change from baseline in bone marrow burden as measured by MRI through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase I: Glucocerebrosidase (GCase) enzyme activity levels in blood
Evaluate the change from baseline in GCase enzyme activity levels in blood through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase II: Glucocerebrosidase (GCase) protein levels in blood
Evaluate the change from baseline in GCase protein levels in blood through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase II: Glucosylsphingosine (Lyso-GL1) in blood
Evaluate the change from baseline in Lyso-GL1 levels in blood through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase II: Liver volume and spleen volume (if applicable)
- Change from baseline in liver volume as measured by MRI through 52 weeks after infusion of LY-M001;
- Change from baseline in spleen volume as measured by MRI through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase II: Hemoglobin levels
Evaluate the change from baseline in hemoglobin levels through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase II: Platelet count
Evaluate the change from baseline in platelet count through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase II: Bone mineral density (BMD)
Evaluate the change from baseline in bone mineral density by dual-energy X-ray absorptiometry through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Phase II: Bone marrow burden (BMB)
Evaluate the change from baseline in bone marrow burden as measured by MRI through 52 weeks after infusion of LY-M001.
Measured: From enrollment to 52 weeks after administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2025
- Study start5 Jul 2024
- Primary completion30 Jul 2027 (estimated)
- Study completion30 Jul 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease Type 1Studies 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
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lingyi Biotech Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Fengkui Zhang, PhD, Principal investigator, Hematology Hospital, Chinese Academy of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06818838
- Sponsor's protocol ID
- LY-M001-GD-101
- Acronym
- GD
- Status last verified
- Jan 2026
- First submitted
- 24 Jan 2025
- Last updated
- 26 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The study is in its early stages and will consider releasing data and related information when detailed and sufficient safety and efficacy data are available in subjects.
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open, Single-arm, Single-dose, Dose-escalation, and Expanded Phase I/II Study Evaluating the Safety, Tolerability, and Efficacy of LY-M001 Injection in Adult Patients With Type I Gaucher Disease
- ClinicalTrials.gov ID
- NCT06818838
- Acronym / sponsor ID
- GD / LY-M001-GD-101
- Conditions
- Gaucher Disease Type 1
- Keywords
- anaemia, Gene therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Lingyi Biotech Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 5 Jul 2024 (actual)
- Primary completion
- 30 Jul 2027 (estimated)
- Study completion
- 30 Jul 2031 (estimated)
- First posted
- 11 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.
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate, Glucosylceramide (GlcCer), in macrophages in the liver, spleen, kidney, bone, lung, and even the brain, inducing their transformation into Gaucher cells whose cell cytoplasm presenting a characteristic "crumpled tissue paper" appearance, leading to pathological changes in involved tissues and organs.LY-M001 Injection is an rAAV8 vector gene therapy product. It can specifically transduce the target organ liver after a single intravenous administration and express the GCase protein in liver cells for a long period of time.
Detailed description
The purpose of this study is to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacokinetics and pharmacokinetics of LY-M001 injection in patients with GD1 using a multicenter, open, single-arm, single-dose, dose-escalation and extended clinical design. This study includes the main study phase and the long-term follow-up study phase. The primary study period is 52 weeks after LY-M001 infusion, and the long-term follow-up period is 53 weeks to 5 years after LY-M001 infusion.Subjects who complete the 52 weeks follow up period or who prematurely withdraw in this study will enter the long-term follow-up study phase to obtain long-term assessment data.
Phase I is a dose escalation study consists of three preset dose groups, including one rollback dose group and two incremental dose groups, which are: Backdose (5 × 10^12 vg/kg) group, dose group 1 (1.5 × 10^13 vg/kg) and dose group 2 (3.0 × 10^13 vg/kg), where dose group 1 was the starting dose of the Phase I study. Three subjects are enrolled in each dose group one by one, and each subject is added to the next subject after at least 28 days of DLT observation to determine safety. Phase I studies enrolled approximately 6 to 12 (up to 12) evaluable subjects.
Phase II is a dose expansion study. After all subjects in Phase I study have completed the Day 28 (D28) observation following LY-M001 infusion, the Recommended Phase 2 Dose (RP2D) will be determined by the Safety Review Committee (SRC), which will also decide whether to proceed to the dose-expansion Phase II study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Phase I: LY-M001 Backdose | Experimental | Genetic: LY-M001Participants receive a single, peripheral intravenous (IV) infusion of LY-M001 at backdose. |
| Phase I: LY-M001 Dose group 1 | Experimental | Genetic: LY-M001Participants receive a single, peripheral intravenous (IV) infusion of LY-M001 at dose group 1. |
| Phase I: LY-M001 Dose group 2 | Experimental | Genetic: LY-M001Participants receive a single, peripheral intravenous (IV) infusion of LY-M001 at dose group 2. |
| Phase II: LY-M001 at the recommended dose | Experimental | Genetic: LY-M001Participants receive a single, peripheral IV infusion of LY-M001 at the recommended dose. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (13 listed)
- Age ≥ 18 years and ≤ 60 years, male or female.
- The subjects should fully understand the purpose, nature, and method of this study as well as possible adverse reactions, and sign the informed consent form (ICF) voluntarily.
- Patients with confirmed double mutations in the GBA1 allele through laboratory testing, and the glucocerebrosidase activity was reduced to less than 30% of the normal value(For example, the result of the dried blood spot (DBS) method is < 1.19 μmol/L/h), and meeting the standard clinical diagnosis criteria for GD1.
- Patients who meet a) or b) below:
- Negative pregnancy test for female subjects of childbearing potential (WOCBP). Notes: WOCBP is defined as the absence of postmenopausal status (continuous amenorrhea of at least 12 months with no identifiable cause other than menopause), and the absence of surgical (i.e., ovarian, salpingectomy, and/or hysterectomy) or Investigator-determined cause of permanent infertility due to other causes (e.g., lenticular hypoplasia) after menarche in female subjects.
Key exclusion criteria (23 listed)
- AAV8 neutralizing antibody positive (Antibody titer > 1:40).
- Patients with clinically diagnosed Gaucher disease type II or III (GD2 or GD3).
- Active and progressive bone disease that is expected to require surgical treatment within the next 6 months.
- Subject has idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), thrombocytopenia, anemia, hepatomegaly, splenomegaly, and/or osteoporosis unrelated to GD as judged by the Investigator.
- Treatment or disposal of investigational drugs or investigational devices received in other clinical studies within 28 days prior to screening or within 5 half-lives (drugs only), whichever is older.
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 |
|---|---|---|
| LY-M001Single Intravenous Infusion of LY-M001 Injection. | Genetic | Phase I: LY-M001 Backdose, Phase I: LY-M001 Dose group 1, Phase I: LY-M001 Dose group 2, Phase II: LY-M001 at the recommended dose |
Primary outcome
Phase I: Incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks after LY-M001 infusion
Time frame: From enrollment to 52 weeks after administration
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
Phase I: Incidence rate of dose-limiting toxicity (DLT) events determined by the data safety review committee (SRC) within at least 28 days after LY-M001 infusion.
Time frame: From enrollment to 52 weeks after administration
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.The adverse events defined as dose-limiting toxicity (DLT) have been clearly specified in the protocol.
Phase I: Liver function levels (including alanine aminotransferase [ALT], aspartate aminotransferase [AST], total bilirubin [TBIL], alkaline phosphatase [ALP], gamma-glutamyl transferase [GGT]) within 52 weeks after LY-M001 infusion.
Time frame: From enrollment to 52 weeks after administration
Incidence rate of liver function-related adverse events evaluated by CTCAE V5.0.
Phase II: Blood glucocerebrosidase (GCase) activity level.
Time frame: From enrollment to 52 weeks after administration
Evaluation based on the detected values of blood glucocerebrosidase（GCase).
Phase II: The incidence rates of adverse events (AEs) and serious adverse events (SAEs), as well as the occurrence of abnormalities in 12-lead electrocardiogram (ECG) findings, vital signs, laboratory test parameters, and physical examination results.
Time frame: From enrollment to 52 weeks after administration
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
15 secondary outcomes
Phase I: Liver volume and spleen volume (if applicable)
Time frame: From enrollment to 52 weeks after administration
Phase I: Hemoglobin levels
Time frame: From enrollment to 52 weeks after administration
Phase I:Bone mineral density (BMD) after administration
Time frame: From enrollment to 52 weeks after administration
Phase I: Glucocerebrosidase (GCase) protein levels in blood
Time frame: From enrollment to 52 weeks after administration
Phase I: Glucosylsphingosine (Lyso-GL1) in blood
Time frame: From enrollment to 52 weeks after administration
Phase I: Platelet count
Time frame: From enrollment to 52 weeks after administration
Phase I: Bone marrow burden (BMB) after administration
Time frame: From enrollment to 52 weeks after administration
Phase I: Glucocerebrosidase (GCase) enzyme activity levels in blood
Time frame: From enrollment to 52 weeks after administration
Phase II: Glucocerebrosidase (GCase) protein levels in blood
Time frame: From enrollment to 52 weeks after administration
Phase II: Glucosylsphingosine (Lyso-GL1) in blood
Time frame: From enrollment to 52 weeks after administration
Phase II: Liver volume and spleen volume (if applicable)
Time frame: From enrollment to 52 weeks after administration
Phase II: Hemoglobin levels
Time frame: From enrollment to 52 weeks after administration
Phase II: Platelet count
Time frame: From enrollment to 52 weeks after administration
Phase II: Bone mineral density (BMD)
Time frame: From enrollment to 52 weeks after administration
Phase II: Bone marrow burden (BMB)
Time frame: From enrollment to 52 weeks after administration
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 26 Jan 2026.
- Individual participant dataSharing planned: No. The study is in its early stages and will consider releasing data and related information when detailed and sufficient safety and efficacy data are available in subjects.
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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.
Fengkui Zhang, PhD principal investigator
Hematology Hospital, Chinese Academy of Medical Sciences
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing LY-M001 in people with Gaucher Disease Type 1, run by Lingyi Biotech Co., Ltd. at 3 sites 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 13 inclusion and 23 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 26 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Qing Lin, PhD (86+19121572057, qing.lin@lingyimed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase I: Incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks after LY-M001 infusion (time frame: From enrollment to 52 weeks after administration). The study lists 5 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 26 Jan 2026.