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Parkinson's DiseaseEarly Phase 1NCT05822739

Safety and Efficacy Study of Parkinson's Disease Gene Therapy Drug (BBM-P002)

A Early Phase 1 study testing BBM-P002 in people with Parkinson's Disease, run by Xiangya Hospital of Central South University at 2 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Safety and Efficacy Study of BBM-P002 in participants with primary advanced Parkinson's disease Read more about this study

Who is it for?
Ages 40 to 65 years
Conditions the study is for
Parkinson's Disease
What is being tested?
BBM-P002
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
2 sitesChina
Study dates
14 Apr 2023to 30 Oct 2025
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 40 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Clinically confirmed diagnosis of idiopathic Parkinson's Disease (PD) in accordance with the 2015 Movement Disorder Society (MDS) diagnostic criteria.
  • Participants aged between 40 and 65 years, inclusive, of any gender.
  • A disease history of five years or more.
  • A Hoehn and Yahr (H-Y) stage of 2.5 to 4 in the Of-state.
  • An adeno-associated virus (AAV) neutralizing antibody titer of fit thecriteria.
  • A Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score greater than 35 in the Off-state, with an improvement of at least 30% following an acute levodopa challenge test.
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  • Stable anti-Parkinsonian medication regimen prior to the screening period for at least four weeks.
  • The subject must agree to postpone any other neurosurgical procedures (including deep brain stimulation) during the main study phase, with the exception of emergency neurosurgery for life-threatening conditions.
  • The subject must agree not to participate in other therapeutic interventional studies during the main study phase.
  • The subject must agree not to undergo vaccination during the main study phase.
  • Commitment to using a reliable method of contraception from the screening period until at least 52 weeks post-infusion.
  • The subject must be compliant and capable of attending regular follow-up appointments. The subject must be able to accurately complete a PD patient diary during the follow-up period, with assistance from family members, guardians, or caregivers permitted.
  • The subject must be fully informed about the nature, objectives, procedures, and potential adverse effects of the clinical trial, and must voluntarily consent to participate by signing the informed consent form. In cases where the subject is unable to read, a legally authorized representative or an impartial witness may read the informed consent form and other written materials to them and witness the consent process.

Who cannot join

  • Diagnosis of atypical or secondary parkinsonian syndromes (e.g., Parkinson-plus syndromes, hereditary parkinsonian syndromes, drug-induced parkinsonism).
  • Presence of surgical contraindications, a history of prior brain surgery such as deep brain stimulation (DBS), pallidotomy, or extrapyramidal surgery, or any other neurosurgical procedure that the investigator deems would interfere with participation in this trial.
  • Previous cranial imaging showing structural brain abnormalities, cerebrovascular malformations, intracranial tumors, risk of intracranial hemorrhage, traumatic brain injury, or other significant anomalies.
  • A Mini-Mental State Examination (MMSE) score of <24.
  • A Patient Health Questionnaire-9 (PHQ-9) score of ≥16.
  • Abnormal liver or kidney function, defined as: Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) >1.5 times the upper limit of normal (ULN), or serum creatinine (Cr) >1.5 times the ULN.
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  • Coagulation dysfunction or current use of anticoagulant agents.
  • Positive screening for infectious diseases: positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B virus DNA (HBV-DNA), positive for Hepatitis C virus RNA (HCV-RNA), positive for Human Immunodeficiency Virus (HIV), or seropositive for syphilis.
  • Currently undergoing antiviral treatment for Hepatitis B or C.
  • Presence of unstable or severe concomitant systemic diseases, including but not limited to active tuberculosis, or diseases of the cardiovascular, respiratory, digestive, urinary, neuropsychiatric (e.g., epilepsy), hematologic, or immune systems, or abnormal laboratory values that, in the judgment of the investigator, would make the subject unsuitable for the trial.
  • Current or prior history of a malignant tumor.
  • History of a severe allergic reaction, allergy to contrast agents, or inability to tolerate surgical anesthesia.
  • Current participation in another clinical trial, or participation in any other clinical trial within 3 months prior to the screening period.
  • Prior history of gene therapy before screening.
  • Receipt of stem cell therapy within 6 months prior to the screening period.
  • Use of other investigational drugs within 4 weeks prior to screening or within 5 half-lives of the drug (whichever is longer), or any medication judged by the investigator to interfere with the trial.
  • Receipt of a live vaccine within 2 months prior to the screening period, or any vaccination within 30 days prior to the screening period.
  • History of alcohol dependence or drug addiction, with an inability to adhere to medical advice to abstain from alcohol during the trial period.
  • Female subjectsparticipants who are pregnant or lactating.
  • Any subject deemed unsuitable for enrollment based on the investigator's assessment.

Trial locations and contacts

2 sites in 1 country.

China2 sites

  • Xiangya Hospital of Central South University

    Changsha, Hunan, 410008

  • Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

    Shanghai

Other trials in China

About this study

Official title: A Single-Arm, Open-Label, Exploratory Clinical Study of BBM-P002 Gene Therapy for Parkinson's Disease

Safety and Efficacy Study of BBM-P002 in participants with primary advanced Parkinson's disease

Read the detailed description

Parkinson's disease is a neurodegenerative disorder involving loss of dopaminergic neurons in the substantia nigra. This study is a single-center, single-arm, open-label, treatment clinical study to evaluate the safety, tolerability and efficacy of an adeno-associated virus (AAV)-based gene therapy, termed BBM-P002, will be injected into the brain via a neurosurgical procedure.

Being tested
BBM-P002
Keywords
Parkinson's Disease, Gene therapy, Adeno-Associated Virus
Medical subject headings
Parkinson Disease

What participants receive

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

  • BBM-P002Drug

    Also called BBM003

    Genetic: single- dose intracranial injection of BBM-P002 Neurosurgical delivery of BBM-P002 to the brain

Study groups in NCT05822739
Study groupRoleWhat they receive
Arm of BBM-P002Experimentalsingle-arm
Receives: Drug: BBM-P002

What is being measured

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

  1. Incidence of Adverse Events and Serious Adverse Events

    Safety and Tolerability of BBM-P002 assessed by Adverse Events and Serious Adverse Event

    Measured: Day 1 through week 52

Secondary outcomes (4)

  1. Efficacy of BBM-P002

    Changes in gene expression product activity levels assessed by brain ¹⁸F-FDOPA PET

    Measured: Change from Baseline to week 4，week 52

  2. Efficacy of BBM-P002

    Changes in daily oral Levodopa (L-DOPA) dose or Levodopa Equivalent Dose (LEDD);

    Measured: Change from Baseline to Week 52

  3. Efficacy of BBM-P002

    Changes in motor/no motor MDS-Unified Parkinson's Disease Rating Scale-Part III （UPDRS III) The UPDRS III score assesses motor symptoms of Parkinson's disease. The total score ranges from 0 to 132 points, with each of the 33 scoring points rated on a 5-point scale from 0 to 4. A score of 0 indicates normal, with no abnormalities. A score of 1 indicates mild, where symptoms are present but usually do not affect function. A score of 2 indicates mild, where symptoms are mild and have a minor impact on function. A score of 3 indicates moderate, where symptoms are moderate and have a significant impact on function. A score of 4 indicates severe, where symptoms are severe and lead to loss of function. The higher the score, the more severe the motor symptoms.

    Measured: Change from Baseline to Week 52

  4. Efficacy of BBM-P002

    sleep disturbances were characterized using the Parkinson's Disease Sleep Scale-2 (PDSS-2) The PASS scale is a self-rating scale widely used internationally and specifically designed for patients with Parkinson's disease. It is used to systematically assess the occurrence frequency and severity of various sleep disorders in patients. There are a total of 15 items, each with a score range of 0 to 4 points, and a 5-level scoring method is adopted: 0 points: very mild/never (symptoms never occur or are very mild and do not cause disturbance) 1 point: Mild (symptoms occur occasionally and cause slight disturbance) · 2 points Moderate (symptoms occur frequently, moderate distress) · 3 points: Severe (symptoms occur frequently, severe distress) · 4 points: very severe/always (symptoms occur all the time or extremely severe). The score range is from 0 to 60 points. The higher the score, the more severe the sleep disorder.

    Measured: Change from Baseline to Week 52

Timeline

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

  1. First posted21 Apr 2023
  2. Study start14 Apr 2023
  3. Primary completion30 Oct 2025
  4. Study completion30 Oct 2025

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
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05822739
Sponsor's protocol ID
BBM003- IIT1003
Status last verified
Jan 2026
First submitted
1 Mar 2023
Last updated
15 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Niu M, Guo J, Yang Y, Xu Q, Cheng C, Li H, Luo N, Zhou H, Zhang P, Li Y, Wang N, Wang C, Li D, Lin Z, Huang P, Yang Z, Yang Z, Song Y, Zheng J, Jiang W, Du Z, Qi Y, Peng H, Tan Y, Hu X, Xiao X, Shen L, Lin J, Liu J. Dual-target gene therapy in Parkinson's disease: a multicenter phase 1 trial. Nat Med. 2026 Jul;32(7):2602-2609. doi: 10.1038/s41591-026-04436-0. Epub 2026 Jun 10.

    PubMed 42271071

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing BBM-P002 in people with Parkinson's Disease, run by Xiangya Hospital of Central South University at 2 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 20 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: finished, as of the sponsor's last update on 15 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 2 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 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Incidence of Adverse Events and Serious Adverse Events (time frame: Day 1 through week 52). 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 NCT05822739, which also lists 1 linked publication.

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 15 Jan 2026.

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