A Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-A101 in the Treatment of Knee Osteoarthritis.

NCT07198555 | Recruiting Phase 1

• 1 other identifier: BBM007-CLN1001
• Study Type: interventional
• Target: 81
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-A101 to treat participants with knee osteoarthritis.

Conditions

Knee Osteoarthritis

Keywords

knee osteoarthritis; gene therapy; Adeno-Associated Virus

Outcome Measures

Primary Outcomes (2)

• Incidence of dose limiting toxicity (DLT) events

  within 4 weeks

• Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

  To evaluate the safety of BBM-A101 by AEs and SAEs.

  within 52 weeks

Secondary Outcomes (5)

• The protein levels of gene expression products in blood and joint fluid

  within5 years

• The shedding of recombinant adeno-associated virus (rAAV) vectors in peripheral blood, joint fluid, urine, feces, saliva, and semen

  within 5 years

• The change in the joint space width as detected by X-ray of the knee joint compared to the baseline

  within 5 years

• The changes in the titers of AAV capsid-binding antibodies in the blood and joint fluid compared to the baseline level

  within 5 years

• The changes in the titers of antibodies binding to the expressed products in the blood and joint fluid compared to the baseline level

  within 5 years

Study Arms (2)

Arm of Placebo

PLACEBO COMPARATOR

Drug: Placebo

Arm of BBM-A101

EXPERIMENTAL

Drug: BBM-A101

Interventions

BBM-A101 DRUG

Single-dose intra-articular （IA）injection （2ml）(phase Ia) Low dose group， Middle dose group； High dose group； Control group ：Placebo

Arm of BBM-A101

Placebo DRUG

Placebo(phase Ia)

Arm of Placebo

Eligibility Criteria

• Age: 40 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants with moderate knee osteoarthritis .
• The knee joint was evaluated as having Kellgren-Lawrence grade 2 or 3.
• The Body Mass Index (BMI) ranges from 18.0 to 35.0 kg/m2 (including the boundary values).
• Lower titers of antibodies binding to the BBM-A101 capsid in the blood.
• Participants who had received adequate treatment with conventional medications previously, experienced recurrence or aggravation of target knee arthritis after discontinuation of medication, and still had moderate pain; during the screening period, the WOMAC pain score of the target knee was ≥ 8 .
• From the time of screening to 52 weeks after dosing , the participants had no intention of having children and voluntarily took effective contraceptive measures, and there were no plans for sperm donation or ova donation.
• Voluntarily sign the informed consent form and be able to complete the research as required by the clinical trial protocol.

You may not qualify if:

• There is a possibility of secondary knee osteoarthritis, or the knee joint symptoms may not be caused by knee osteoarthritis, or there may be other medical histories of diseases that other researchers believe could potentially lead to knee osteoarthritic inflammation.
• History of lumbar dischernaiation, accompanied by lower limb nerve symptoms that may affect the assessment of the study.
• Participants who are unable or unwilling to undergo MRI examination, or who have contraindications to MRI.
• Participants who are unable or unwilling to undergo joint fluid extraction.
• During the screening period and before dosing, there were clinically significant abnormal values in the laboratory tests, and the researchers believed that these might pose risks to the participants in the clinical trial.
• Those with positive results for hepatitis B virus surface antigen or hepatitis B virus deoxyribonucleic acid or hepatitis C virus ribonucleic acid test or human immunodeficiency virus antibody or Treponema pallidum antibody.
• Currently undergoing treatment for hepatitis B and hepatitis C.
• Participants who had persistent chronic or active infections within the previous 4 weeks were selected.
• Participants who had coagulation disorders (such as hemophilia) or existing medical conditions that require anticoagulation therapy and make knee joint injection impossible.
• Having or having had cancer or malignant tumors
• Having received genetic therapy or stem cell therapy before screening or having plans for genetic therapy or stem cell therapy during the study period.
• Any history of vaccination within 30 days prior to dosing or any vaccination schedule within 30 days after dosing.
• Screening will be conducted on female participants who are pregnant or breastfeeding or whose HCG test result is positive; or who have plans to get pregnant within the next 52 w.
• History of alcohol addiction or drug dependence, or inability to limit alcohol consumption during the research process.
• Participants had any mental disorders that might affect the assessment of the study.

Sponsors & Collaborators

• Belief BioMed (Beijing) Co., Ltd: lead

Study Sites (2)

Shenzhen Second People's Hospital

Shenzhen, Guangdong, 518035, China

RECRUITING

The Ninth People's Hospital of Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200011, China

RECRUITING

MeSH Terms

Conditions

Osteoarthritis, Knee

Condition Hierarchy (Ancestors)

Osteoarthritis; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Study Officials

• Jie Zhao

  The Ninth People's Hospital of Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wenshu Luo, Ph.D

CONTACT

021-33588288; luowenshu@beliefbiomed.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Masking Details: The masking subjects in the Ia phase is participant , but the phase Ib study is double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2025
• First Posted: September 30, 2025
• Study Start: October 9, 2025
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 30, 2031
• Last Updated: November 18, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)