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Gaucher Disease Type ILY-M001NCT06162338

A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I

A study testing LY-M001 Injection in people with Gaucher Disease Type I, run by He Huang at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This is a prospective single-center, open, single-arm, single-dose intravenous infusion study to evaluate the safety and initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection.This study mainly includes the main study stage and the long-term… Read more about this study

Who is it for?
Ages 18 to 60 years
Conditions the study is for
Gaucher Disease Type I
What is being tested?
LY-M001 Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
1 siteChina
Study dates
15 Nov 2023to 17 Dec 2031
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 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 GD1 who have confirmed double mutations in the Gba1 allele by laboratory testing and meet the standard for clinical diagnosis of Gaucher disease (i.e., reduced GCase enzyme activity to less than 30% of normal values).
  • The subjects were type I patients with Gaucher disease. Patients with type I Gaucher disease who had received specific treatment in the past required 5 half-lives of elution
  • Negative pregnancy test for female subjects of childbearing potential 6.6.The subject and his/her partner have no plans to have children during the screening period and within 6 months after the end of the study, and voluntarily take effective contraceptive measures (such as abstinence, condom, etc.); and the subject had no plans to donate sperm or eggs.
  • 7.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 is strongly positive.
  • Patients with clinically suspected Gaucher disease type II (GD2) or Gaucher disease type III (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 Gaucher disease as judged by the investigator.
  • Treatment or disposition of an investigational drug or investigational device for another clinical investigation within 28 days or 5 half-lives (only for drugs), whichever is longer, prior to Screening.
  • Evidence of clinically significant liver disease, fragile liver, or history of hepatotoxin exposure, meeting at screening, but not limited to, any of the following:
Show all 25Show fewer
  • Progressive hepatomegaly and greater than 3 times normal volume.
  • History of stage 2 or greater liver fibrosis.
  • AST, ALT or TBIL greater than 1.5 times the upper limit of normal (ULN).
  • History of alcohol or drug abuse within the previous 2 years.
  • Hepatitis B surface antigen (HBsAg) positive, and hepatitis B virus DNA positive (HBV-DNA > 103 copies/mL); or taking hepatitis B virus drugs (such as interferon, lamivudine, adefovir and entecavir); or hepatitis C virus (HCV) antibody positive.
  • Human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibody positive.
  • Severe hyperlipidemia (triglycerides > 1000 mg/dL).
  • Uncontrolled concomitant disease or infectious disease (need to be judged by the investigator based on clinical practice).
  • Subject had undergone splenectomy and were scheduled to undergo splenectomy during the study period.
  • Karnofsky score (KPS) < 70.
  • 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 significant cardiovascular disease as judged by the investigator (e.g., New York Heart Association [NYHA] class ≥ 3 heart failure).
  • 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 of screening, except for completely resected non-melanoma skin cancer, non-metastatic prostate cancer, and completely treated ductal carcinoma in situ.
  • 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

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

He Huang, PhD contact

0571-87236703

hehuangyu@126.com

China1 site, 1 recruiting

  • The First Affiliated Hospital of Zhejiang University School of Medicine

    Hanzhou, Zhejiang, 310003

    He Huang, PhD, 0571-87236703, hehuangyu@126.com

    Recruiting

Other trials in China

About this study

Official title: A Prospective, Single-center, Open-arm, Single-arm Study of the Safety and Preliminary Efficacy of Single Intravenous Infusion Administration of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I

This is a prospective single-center, open, single-arm, single-dose intravenous infusion study to evaluate the safety and initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection.This study mainly includes the main study stage and the long-term follow-up study stage.

Read the detailed description

This study included the screening period (weeks -8 to days -2), the baseline period (days -1), the treatment and safety observation period (days 0 to 28), and the short-term follow-up period (weeks 5 [from day 29] to week 38). Participants eligible for the screening period will be admitted to the study center for a single LY-M001 treatment and a short-term follow-up period after the end of the treatment and safety observation period. Participants who complete all follow-up during the main study phase or who withdraw early from the study are required to complete all assessments required for the End of Study (EOS) visit.The study included up to three adult Gaucher disease type I subjects at preset dose group levels.With 5.0 × 1012 vg/kg as the initial effective dose (first dose group), 1 to 2 subjects are expected to be included. The first dose group was enrolled by sentinel method, and the first subject in this group was observed for at least 28 days after receiving LY-M001 (dose-limited toxicity [DLT] observation period) to enroll the next subject.

Participants enrolled in the long-term follow-up study were those who completed the main study or withdrew early, and the duration of the long-term follow-up study and the main study lasted for a total of 5 years.

Being tested
LY-M001 Injection
Medical subject headings
Gaucher Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LY-M001 InjectionBiological

    With 5.0 × 10^12 vg/kg as the initial effective dose (first dose group), 1 to 2 subjects are expected to be included. The first dose group was enrolled by sentinel method, and the first subject in this group was observed for at least 28 days after receiving LY-M001 (dose-limited toxicity [DLT] observation period) to enroll the next subject. After the first participant completes the safety evaluation of dosing for at least 28 days, the investigator (PI) and the partner discuss the safety and efficacy data and decide to maintain the first dose or increase/reduce it to another dose group

Study groups in NCT06162338
Study groupRoleWhat they receive
A Prospective, single-center, open, single-arm studyExperimentalThe study is single-arm. LY-M001 injection is administered by intravenous infusion at a rate of 1 mL per minute.LY-M001 Injection is Clear and colorless liquid.According to the dose cohort of the subject, the required dosage was accurately calculated by the patient's body weight for infusion.
Receives: Biological: LY-M001 Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of treatment- related adverse events

    Number and severity of adverse events and serious adverse events and relationship to LY-M001

    Measured: within 38 weeks after LY-M001 infusion

  2. Incidence of events adjudicated as dose limiting toxicity (DLT)

    Number and severity of dose limiting toxicity

    Measured: within 28 days after LY-M001 infusion

Secondary outcomes (2)

  1. Glucocerebrosidase (GCase) protein in plasma and GCase enzyme activity

    Change from baseline in plasma GCase protein and GCase enzyme activity level

    Measured: Within 38 Weeks after LY-M001 infusion

  2. Glucosylsphingosine (Lyso-GL1)in plasma

    Change from baseline in plasma Lyso-GL1 level

    Measured: Within 38 Weeks after LY-M001 infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Dec 2023
  2. Study start15 Nov 2023
  3. Primary completion17 Dec 2026 (estimated)
  4. Study completion17 Dec 2031 (estimated)

Resources

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
Model description
A Prospective, Single-center, Open-label, Single-arm study
Masking
None (open label)
Enrolment
3 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06162338
Sponsor's protocol ID
LY-M001-GD-001
Acronym
LY-M001
Status last verified
Feb 2025
First submitted
20 Nov 2023
Last updated
14 Feb 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Nabizadeh A, Amani B, Kadivar M, Toroski M, Asl AA, Bayazidi Y, Mojahedian M, Davari M. The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review. J Res Pharm Pract. 2018 Oct-Dec;7(4):171-177. doi: 10.4103/jrpp.JRPP_18_24.

    PubMed 30622983

  • Barranger JA, Brady RO, Grabowski GA, Mankin H, Mistry PK, Weinreb NJ. Position statement: National Gaucher Foundation Medical Advisory Board, January 7, 2014. Am J Hematol. 2014 May;89(5):457-8. doi: 10.1002/ajh.23687. Epub 2014 Mar 7. No abstract available.

    PubMed 24488939

  • Franco M, Reihani N, Marin M, De Person M, Billette de Villemeur T, Rose C, Colin Y, Moussa F, Belmatoug N, Le Van Kim C. Effect of velaglucerase alfa enzyme replacement therapy on red blood cell properties in Gaucher disease. Am J Hematol. 2017 Sep;92(9):E561-E563. doi: 10.1002/ajh.24816. Epub 2017 Jul 19. No abstract available.

    PubMed 28621801

  • Kuter DJ, Wajnrajch M, Hernandez B, Wang R, Chertkoff R, Zimran A. Open-label, expanded access study of taliglucerase alfa in patients with Gaucher disease requiring enzyme replacement therapy. Blood Cells Mol Dis. 2020 May;82:102418. doi: 10.1016/j.bcmd.2020.102418. Epub 2020 Feb 24.

    PubMed 32146279

  • Bennett LL, Fellner C. Pharmacotherapy of Gaucher Disease: Current and Future Options. P T. 2018 May;43(5):274-309.

    PubMed 29719368

  • Tsai P, Lipton JM, Sahdev I, Najfeld V, Rankin LR, Slyper AH, Ludman M, Grabowski GA. Allogenic bone marrow transplantation in severe Gaucher disease. Pediatr Res. 1992 May;31(5):503-7. doi: 10.1203/00006450-199205000-00019.

    PubMed 1603628

  • Konkle BA, Walsh CE, Escobar MA, Josephson NC, Young G, von Drygalski A, McPhee SWJ, Samulski RJ, Bilic I, de la Rosa M, Reipert BM, Rottensteiner H, Scheiflinger F, Chapin JC, Ewenstein B, Monahan PE. BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression. Blood. 2021 Feb 11;137(6):763-774. doi: 10.1182/blood.2019004625.

    PubMed 33067633

  • Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309.

    PubMed 25409372

  • Xu YH, Quinn B, Witte D, Grabowski GA. Viable mouse models of acid beta-glucosidase deficiency: the defect in Gaucher disease. Am J Pathol. 2003 Nov;163(5):2093-101. doi: 10.1016/s0002-9440(10)63566-3.

    PubMed 14578207

  • Sun Y, Zhang W, Xu YH, Quinn B, Dasgupta N, Liou B, Setchell KD, Grabowski GA. Substrate compositional variation with tissue/region and Gba1 mutations in mouse models--implications for Gaucher disease. PLoS One. 2013;8(3):e57560. doi: 10.1371/journal.pone.0057560. Epub 2013 Mar 8.

    PubMed 23520473

  • Chen RL, Hou JW, Chang PY, Tsai FJ, Wang PJ. Matched unrelated bone marrow transplantation without splenectomy for a child with Gaucher disease caused by homozygosity of the L444P mutation, who also suffered from schizencephaly. J Pediatr Hematol Oncol. 2007 Jan;29(1):57-9. doi: 10.1097/MPH.0b013e3180308793.

    PubMed 17230068

  • Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissee M, Long A, Nathwani A. Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B. N Engl J Med. 2022 Jul 21;387(3):237-247. doi: 10.1056/NEJMoa2119913.

    PubMed 35857660

Questions about this trial

What is this clinical trial studying?

A study testing LY-M001 Injection in people with Gaucher Disease Type I, run by He Huang 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 6 inclusion and 25 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 14 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 He Huang, PhD (0571-87236703, 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- related adverse events (time frame: within 38 weeks after LY-M001 infusion). 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?

On the ClinicalTrials.gov record for NCT06162338, which also lists 12 linked publications.

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 14 Feb 2025.

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