NMO Spectrum DisorderPhase 1NCT04227470
A Study of HBM9161 in NMOSD Patients
A Phase 1 study testing HBM9161 Injection in people with NMO Spectrum Disorder, run by Harbour BioMed (Guangzhou) Co. Ltd. at 1 site in China.
Trial summary
Primary Objectives:To investigate the safety and tolerability of HBM 9161 in patients with attack of NMOSD in China Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- NMO Spectrum Disorder
- What is being tested?
- HBM9161 Injection
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 31 Mar 2020to 24 Dec 2021
- Phase
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- In visit 1, Male or female aged ≥ 18 years.
- Patient with NMOSD as defined by 2015 NMOSD diagnostic criteria by IPND (International Panel for NMO Diagnosis).
- Core clinical manifestations characterized by new acute optic neuritis and/or transverse myelitis. A clinical event is defined as an episode of inflammation in the spinal cord and/or optic nerve leading to neurologic deficits which can be identified by physical examination and not attributable to another disease process.
- The EDSS score should be ≥ 2.5 and ≤7.5 at visit 1.
- AQP4-IgG is positive at visit 1 or had AQP4-IgG positive medical records before visit 1.
- Be able to recognize English letters.
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- Patients should be on stable treatment of the following medications before screening (if anyone had a stable treatment ):
- Immunosuppressant or immunomodulatory drugs (for example, azathioprine, cyclophosphamide, mycophenolate mofetil, tacrolimus, methotrexate and so on) must be stable for at least 8 weeks before screening and keep stable during study
- Corticosteroids
- At screening, the treatment dose must be stable for at least 1 month. • If patients accepted plasmapheresis or IVIg treatment, the last treatment dose/procedure must be finished at least 4 weeks ago before screening
Who cannot join
- No acute optic neuritis and/or transverse myelitis symptoms or signs.
- Severe NMOSD which may require plasmapheresis or intravenous immunoglobulin (IVIG) treatment, in opinion of investigator, very soon.
- Have received plasmapheresis or IVIG treatment, the last treatment dose/procedure is less than 4 weeks before visit 1.
- Have known autoimmune diseases other than NMOSD that would interfere with efficacy assessment or participation in this study (such as uncontrolled thyroid disease or severe rheumatoid arthritis), or have any comorbid diseases which would interfere with the efficacy evaluation of HBM9161 on NMOSD.
- Have received rituximab or other anti-CD20 drugs treatment within 6 months before visit 1.
- Have been used any monoclonal antibodies or research drugs for immunomodulatory effects within 3 months before visit 1 or within 5 half-life periods of the drug.
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- Females who are pregnant or lactating.
- Patients who can't tolerate or have contraindication to high dose intravenous methylprednisolone per Investigator's opinion.
- Have active infection at screening, or recent serious infection (i.e., requiring intravenous antimicrobial therapy or hospitalization) within 8 weeks before screening; history of or existing infection of human immunodeficiency virus(HIV), hepatitis C virus (HCV), or Mycobacterium tuberculosis. Patients must have negative test results for HCV antibody, HIV 1 and HIV 2 antibodies, and a mycobacterium tuberculosis test (test method to be determined) at visit 1.
- Patients have positive test result for HBsAg; or HBsAg negative meanwhile HBcAb positive and HBV-DNA level>2000IU/mL.
- Serum total IgG <700mg/dL at visit 1.
- Absolute neutrophil count <1500个/mm3 at visit 1 and/or visit 2
- Patients with acute liver function impairment (e.g., hepatitis) or severe liver cirrhosis (Child-Pugh Score, Class C)
- Any malignant tumor.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety, Tolerability, Pharmacodynamics and Efficacy of HBM9161 Weekly Subcutaneous Administration in Patients With Neuromyelitis Optica Spectrum Disorders (NMOSD) in China
Primary Objectives:To investigate the safety and tolerability of HBM 9161 in patients with attack of NMOSD in China
Read the detailed description
This is an open-label, dose exploration study.The investigational drug is HBM9161 injection, and the indication is NMOSD.
HBM9161(HL161BKN) is a human monoclonal antibody. HBM9161 targets the neonatal Fc receptor (FcRn) . By blocking the FcRn IgG-Fc binding site and accelerating the degradation of IgG, it can significantly reduce the total IgG level in blood (including pathological IgG).The serum aquaporin 4 antibody (AQP4-IgG) associated with NMOSD is a pathological IgG, so the combination of standard of care which is intravenous methylprednisolone (ivMP) with HBM9161 is expected to rapidly reduce AQP4-IgG levels.
Two dose groups (340 mg and 680 mg) were planned, and each dose group plans to enroll approximately 6 subjects. All subjects are weekly administered the HBM9161 by subcutaneous injection for a period of 4 weeks, together with standard of care which is of intravenous methylprednisolone (ivMP) by subcutaneous for a period of 4 weeks. The study will investigate the safety, and tolerability, pharmacodynamics and efficacy of HBM 9161 in patients with attack of NMOSD in China.
- Conditions
- NMO Spectrum Disorder
- Being tested
- HBM9161 Injection
- Keywords
- NMOSD
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HBM9161 InjectionDrug
Subcutaneous injection; Weekly administered for a period of 4 weeks. All subjects are treated with the testing drug, add on intravenous methylprednisolone (ivMP) with gradually reduce the dose then to oral prednisone. After the administration of the testing drug, if the subject's symptoms get worsen, a rescue therapy need to be adopted as based on Investigator's judgement, the testing drug injection should be discontinued.
| Study group | Role | What they receive |
|---|---|---|
| Experimental: HBM9161, 340mg | Experimental | HBM 9161 injection, 340mg, weekly administered by subcutaneous for a period of 4 weeks. Receives: Drug: HBM9161 Injection |
| Experimental: HBM9161, 680mg | Experimental | HBM 9161 injection, 680mg, weekly administered by subcutaneous for a period of 4 weeks. Receives: Drug: HBM9161 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of treatment related adverse events (AEs)
Number of treatment related adverse events (AEs)
Measured: 189 days
Secondary outcomes (8)
Immunoglobins changes from baseline to week 27
Change of concentration of immunoglobins in mg/ml overtime after administration of HBM9161 from baseline to week 27
Measured: 189 days
Neurological Disability changes from baseline to week 27
Neurological Disability changes from baseline to week 27 as measured by Expanded Disability Scale Score (EDSS, Score 0-10, higher means a worse outcome)
Measured: 189 days
Low Contrast Visual Acuity (LCVA) changes from baseline to week 27
Low Contrast Visual Acuity (LCVA) changes from baseline to week 27 as measured by Sloan Low Contrast Letter Scale (SLCLS Letter, Score 0-70, higher means a better outcome)
Measured: 189 days
Patient reported improvement changes from baseline to week 27
Patient reported improvement changes from baseline to week 27 as measured by Patient Global Impression-Improvement (PGI-I, Score 1-7, higher means a worse outcome)
Measured: 189 days
Percentage of patients who received rescue therapy
Percentage of patients who received rescue therapy
Measured: 189 days
Percentage of patients who have relapse
Percentage of patients who have relapse
Measured: 189 days
Walking ability changes from baseline to week 27
Walking ability changes from baseline to week 27 as measured by time used for 25-foot Walk (applicable for patients who are able to walk)
Measured: 189 days
The seropositive rate of anti-HBM9161 antibody after treatment
Evaluation of the seropositive rate of anti-HBM9161 antibody after treatment
Measured: 189 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2020
- Study start31 Mar 2020
- Primary completion3 Dec 2021
- Study completion24 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for NMO Spectrum DisorderStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Dose exploration study. Two dose groups (340 mg and 680 mg) were planned, 6 subjects at most for 340 mg group and 6 to 12 subjects for 680mg group. Each subject will only participate in one dose group. Escalation to the next dose level decided by PIs and sponsor after evaluating safety data and PD data for lower dose group.
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Harbour BioMed (Guangzhou) Co. Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wei Qiu, Principal investigator, Third Affiliated Hospital, Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04227470
- Sponsor's protocol ID
- 9161.2
- Status last verified
- Jan 2022
- First submitted
- 21 Nov 2019
- Last updated
- 25 Jan 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wang J, Cai G, Xia S, Qin J, Liu B. Research hotspots and emerging topics in neuromyelitis optica spectrum disorder treatment: Insights from a bibliometric analysis. Medicine (Baltimore). 2025 Jun 6;104(23):e42850. doi: 10.1097/MD.0000000000042850.
PubMed 40489809
Study at a glance
From the registry record, last updated by the sponsor on 25 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Tolerability, Pharmacodynamics and Efficacy of HBM9161 Weekly Subcutaneous Administration in Patients With Neuromyelitis Optica Spectrum Disorders (NMOSD) in China
- ClinicalTrials.gov ID
- NCT04227470
- Acronym / sponsor ID
- 9161.2
- Conditions
- NMO Spectrum Disorder
- Keywords
- NMOSD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Harbour BioMed (Guangzhou) Co. Ltd.Industry (pharma, biotech, devices)
- Study start
- 31 Mar 2020 (actual)
- Primary completion
- 3 Dec 2021 (actual)
- Study completion
- 24 Dec 2021 (actual)
- First posted
- 13 Jan 2020 (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.
Primary Objectives:To investigate the safety and tolerability of HBM 9161 in patients with attack of NMOSD in China
Detailed description
This is an open-label, dose exploration study.The investigational drug is HBM9161 injection, and the indication is NMOSD.
HBM9161(HL161BKN) is a human monoclonal antibody. HBM9161 targets the neonatal Fc receptor (FcRn) . By blocking the FcRn IgG-Fc binding site and accelerating the degradation of IgG, it can significantly reduce the total IgG level in blood (including pathological IgG).The serum aquaporin 4 antibody (AQP4-IgG) associated with NMOSD is a pathological IgG, so the combination of standard of care which is intravenous methylprednisolone (ivMP) with HBM9161 is expected to rapidly reduce AQP4-IgG levels.
Two dose groups (340 mg and 680 mg) were planned, and each dose group plans to enroll approximately 6 subjects. All subjects are weekly administered the HBM9161 by subcutaneous injection for a period of 4 weeks, together with standard of care which is of intravenous methylprednisolone (ivMP) by subcutaneous for a period of 4 weeks. The study will investigate the safety, and tolerability, pharmacodynamics and efficacy of HBM 9161 in patients with attack of NMOSD in China.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Dose exploration study. Two dose groups (340 mg and 680 mg) were planned, 6 subjects at most for 340 mg group and 6 to 12 subjects for 680mg group. Each subject will only participate in one dose group. Escalation to the next dose level decided by PIs and sponsor after evaluating safety data and PD data for lower dose group.
| Arm | Type | Receives |
|---|---|---|
| Experimental: HBM9161, 340mg | Experimental | Drug: HBM9161 InjectionHBM 9161 injection, 340mg, weekly administered by subcutaneous for a period of 4 weeks. |
| Experimental: HBM9161, 680mg | Experimental | Drug: HBM9161 InjectionHBM 9161 injection, 680mg, weekly administered by subcutaneous for a period of 4 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- In visit 1, Male or female aged ≥ 18 years.
- Patient with NMOSD as defined by 2015 NMOSD diagnostic criteria by IPND (International Panel for NMO Diagnosis).
- Core clinical manifestations characterized by new acute optic neuritis and/or transverse myelitis. A clinical event is defined as an episode of inflammation in the spinal cord and/or optic nerve leading to neurologic deficits which can be identified by physical examination and not attributable to another disease process.
- The EDSS score should be ≥ 2.5 and ≤7.5 at visit 1.
- AQP4-IgG is positive at visit 1 or had AQP4-IgG positive medical records before visit 1.
Key exclusion criteria (14 listed)
- No acute optic neuritis and/or transverse myelitis symptoms or signs.
- Severe NMOSD which may require plasmapheresis or intravenous immunoglobulin (IVIG) treatment, in opinion of investigator, very soon.
- Have received plasmapheresis or IVIG treatment, the last treatment dose/procedure is less than 4 weeks before visit 1.
- Have known autoimmune diseases other than NMOSD that would interfere with efficacy assessment or participation in this study (such as uncontrolled thyroid disease or severe rheumatoid arthritis), or have any comorbid diseases which would interfere with the efficacy evaluation of HBM9161 on NMOSD.
- Have received rituximab or other anti-CD20 drugs treatment within 6 months before visit 1.
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 |
|---|---|---|
| HBM9161 InjectionSubcutaneous injection; Weekly administered for a period of 4 weeks. All subjects are treated with the testing drug, add on intravenous methylprednisolone (ivMP) with gradually reduce the dose then to oral prednisone. After the administration of the testing drug, if the subject's symptoms get worsen, a rescue therapy need to be adopted as based on Investigator's judgement, the testing drug injection should be discontinued. | Drug | Experimental: HBM9161, 340mg, Experimental: HBM9161, 680mg |
Primary outcome
Number of treatment related adverse events (AEs)
Time frame: 189 days
Number of treatment related adverse events (AEs)
8 secondary outcomes
Immunoglobins changes from baseline to week 27
Time frame: 189 days
Neurological Disability changes from baseline to week 27
Time frame: 189 days
Low Contrast Visual Acuity (LCVA) changes from baseline to week 27
Time frame: 189 days
Patient reported improvement changes from baseline to week 27
Time frame: 189 days
Percentage of patients who received rescue therapy
Time frame: 189 days
Percentage of patients who have relapse
Time frame: 189 days
Walking ability changes from baseline to week 27
Time frame: 189 days
The seropositive rate of anti-HBM9161 antibody after treatment
Time frame: 189 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jan 2022.
- Derived from this study: PubMed 40489809Wang J, Cai G, Xia S, Qin J, Liu B. Research hotspots and emerging topics in neuromyelitis optica spectrum disorder treatment: Insights from a bibliometric analysis. Medicine (Baltimore). 2025 Jun 6;104(23):e42850. doi: 10.1097/MD.0000000000042850.
- Individual participant dataSharing planned: No
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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.
Wei Qiu principal investigator
Third Affiliated Hospital, Sun Yat-Sen University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HBM9161 Injection in people with NMO Spectrum Disorder, run by Harbour BioMed (Guangzhou) 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 14 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 25 Jan 2022. 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?
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.
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?
Number of treatment related adverse events (AEs) (time frame: 189 days). 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 NCT04227470, 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 25 Jan 2022.