Neuromyelitis Optica Spectrum DisorderPhase 2NCT05551598
Efficacy and Safety of Mitoxantrone Hydrochloride Liposome Injection in the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)
A Phase 2 study testing Mitoxantrone Hydrochloride Liposome Injection in people with Neuromyelitis Optica Spectrum Disorder, run by CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd. at 1 site in China.
Trial summary
This is a multicenter, randomized, double-blind, placebo-controlled Phase Ⅱ study to evaluate the efficacy and safety of Mitoxantrone Hydrochloride Liposome Injection with different doses in participants with neuromyelitis optica spectrum disorder (NMOSD). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- Mitoxantrone Hydrochloride Liposome Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 46 participants
- Where
- 1 siteChina
- Study dates
- 28 Nov 2022to 18 Jul 2024
- Phase
- Phase 2
- 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 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 18-60 years (inclusive) at the time of signing the informed consent form, both men and women;
- Participants meet the 2015 international consensus diagnostic criteria for neuromyelitis optica spectrum disease (NMOSD) and are AQP4-IgG positive or negative (acceptable test results within 24 weeks before signing the informed consent form);
- Experienced at least 2 attacks within 1 year before screening and/or at least 3 relapses in the past 24 months, but have at least 1 documented clinical evidence of attack or relapse (including the first attack) within 12 months before screening;
- Expanded disability status scale (EDSS) ≤ 7.5 points at screening and baseline retest;
- Participants voluntarily sign the informed consent form and voluntarily complete the trial according to the protocol requirements.
Who cannot join
- Pregnant or lactating female participants, or participants planning to have a child during the study;
- Any surgical procedure within 4 weeks before randomization, with evidence of other demyelinating diseases or progressive multifocal leukoencephalopathy (PML);
- Known active infection (excluding nail bed fungal infection or dental caries) within 4 weeks before randomization;
- Hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCVAb), syphilis antibody, or human immunodeficiency virus (HIV antibody) is positive;
- History of drug or alcohol abuse or mental disorder within 1 year before randomization;
- Evidence of active tuberculosis (TB; excluding patients receiving drugs to prevent latent TB infection);
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- Evidence of active interstitial lung disease;
- Receipt of any live or live attenuated vaccine within 6 weeks before randomization;
- History of malignancy, including solid tumors, hematological malignancies, and carcinoma in situ (except completely resected and cured basal cell carcinoma and squamous cell carcinoma or cervical carcinoma in situ) within the past 5 years;
- History of severe drug allergy, allergy or intolerance to Mitoxantrone and liposomes (shock, allergic reactions);
- Participants with other chronic active immune system diseases other than NMOSD or stable disease but requiring high-dose glucocorticoid maintenance therapy: such as rheumatoid arthritis, scleroderma, Schogren' s syndrome, ulcerative colitis, genetic immunodeficiency or drug-induced immunodeficiency; participants with positive autoantibodies only but without clinical manifestations can be enrolled in the trial;
- Participants with other diseases not suitable for this study as judged by the investigator;
- Received any investigational agent within 3 months prior to randomization, or participated in a medical device clinical study and judged by the investigator to have a potential impact on the results of this trial;
- History of prior NMOSD therapy with any of the following: a. received prior mitoxantrone or anthracycline therapy, total body irradiation, or bone marrow transplantation at any time; b. received rituximab or any experimental B-cell depleting agent within 6 months prior to randomization, unless the participant had a B-cell count higher than LLN in the central laboratory; c. used any of the following within 3 months prior to randomization: Satralizumab,Tocilizumab,inebilizumab,Eculizumab,Alemtuzumab,Ciclosporin,Azathioprine,Cyclophosphamide,Tacrolimus,Mycophenolate mofetil. Any other treatment to prevent relapses in multiple sclerosis (MS) (eg, Interferon, Natalizumab, Glatiramer acetate, Fingolimod, Teriflunomide, or Dimethyl fumarate); d. intravenous immunoglobulin (IVIG) therapy, plasma exchange therapy (PE) within 1 month prior to randomization; e. participants who are receiving oral corticosteroids at doses higher than 30 mg/day in prednisolone conversion; f. anti-CD4, cladribine within 2 years prior to randomization.
- Presence of the following clinically significant diseases: a. cardiac ejection fraction (EF) less than 50% by echocardiography or lower than the lower limit of laboratory values at the study site; history of chronic congestive heart failure, cardiac function NYHA class Ш ~ Ⅳ; b. any of the following events within 3 months before randomization: myocardial infarction, acute coronary syndrome, viral myocarditis, pulmonary embolism, stroke; coronary revascularization within 6 months; c. screening ECG showed QTc interval > 480 ms (according to Fridericia correction formula, QTc = QT/RR^0.33), or a history of severe QTc prolongation.
- Patients with the following abnormalities in laboratory values during screening: a. aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN), alanine aminotransferase (ALT) > 3 × ULN, total bilirubin > 1.5 × ULN (unless due to Gilbert 's syndrome); b. platelet count < 50,000/μL (or < 50 × 10^9/L), hemoglobin < 9 g/dL (or < 90 g/L), white blood cells < 2.0 × 10^3/μL, absolute neutrophil count < 1.0 × 10^3/μL; c. creatinine clearance (CLcr) < 60 mL/min (calculated using the Cockcroft-Gault formula: [140 - age (years)] × [body weight (kg)] × (0.85, if female)/[72 × serum creatinine (mg/dL)]) or receiving dialysis during screening;
Trial locations and contacts
1 site in 1 country.
China1 site
Xuanwu Hospital Capital Medical University
Beijing, Beijing Municipality, 100000
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Phase Ⅱ Clinical Study to Evaluate the Efficacy and Safety of Mitoxantrone Hydrochloride Liposome Injection in the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)
This is a multicenter, randomized, double-blind, placebo-controlled Phase Ⅱ study to evaluate the efficacy and safety of Mitoxantrone Hydrochloride Liposome Injection with different doses in participants with neuromyelitis optica spectrum disorder (NMOSD). Participants will be randomly enrolled into three groups: Mitoxantrone Hydrochloride Liposome Injection 8 mg/m^2 group, Mitoxantrone Hydrochloride Liposome Injection 12 mg/m^2 group, and Placebo group. The primary outcome measure is time to first protocol-defined relapse.
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Being tested
- Mitoxantrone Hydrochloride Liposome Injection
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mitoxantrone Hydrochloride Liposome InjectionDrug
IV, once every 12 weeks (Q12W)
| Study group | Role | What they receive |
|---|---|---|
| Mitoxantrone Hydrochloride Liposome Injection 8 mg/m^2 group | Experimental | Not described Receives: Drug: Mitoxantrone Hydrochloride Liposome Injection |
| Mitoxantrone Hydrochloride Liposome Injection 12 mg/m^2 group | Experimental | Not described Receives: Drug: Mitoxantrone Hydrochloride Liposome Injection |
| Placebo Injection every 12 weeks (Q12W). | Placebo comparator | Not described Receives: Drug: Mitoxantrone Hydrochloride Liposome Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to First Protocol-defined Relapse
Time to First Protocol-defined Relapse was defined as time from randomization to first occurrence of relapse. Protocol-defined relapse was occurrence of new or worsening neurological symptoms attributable to neuromyelitis optica spectrum disorder (NMOSD).
Measured: Up to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2022
- Study start28 Nov 2022
- Primary completion18 Jul 2024
- Study completion18 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.The sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05551598
- Sponsor's protocol ID
- HE071-CSP-028
- Status last verified
- Jul 2022
- First submitted
- 20 Sep 2022
- Last updated
- 7 Aug 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Phase Ⅱ Clinical Study to Evaluate the Efficacy and Safety of Mitoxantrone Hydrochloride Liposome Injection in the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)
- ClinicalTrials.gov ID
- NCT05551598
- Acronym / sponsor ID
- HE071-CSP-028
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 28 Nov 2022 (actual)
- Primary completion
- 18 Jul 2024 (actual)
- Study completion
- 18 Jul 2024 (actual)
- First posted
- 22 Sep 2022 (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.
This is a multicenter, randomized, double-blind, placebo-controlled Phase Ⅱ study to evaluate the efficacy and safety of Mitoxantrone Hydrochloride Liposome Injection with different doses in participants with neuromyelitis optica spectrum disorder (NMOSD). Participants will be randomly enrolled into three groups: Mitoxantrone Hydrochloride Liposome Injection 8 mg/m^2 group, Mitoxantrone Hydrochloride Liposome Injection 12 mg/m^2 group, and Placebo group. The primary outcome measure is time to first protocol-defined relapse.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Mitoxantrone Hydrochloride Liposome Injection 8 mg/m^2 group | Experimental | Drug: Mitoxantrone Hydrochloride Liposome Injection |
| Mitoxantrone Hydrochloride Liposome Injection 12 mg/m^2 group | Experimental | Drug: Mitoxantrone Hydrochloride Liposome Injection |
| Placebo Injection every 12 weeks (Q12W). | Placebo comparator | Drug: Mitoxantrone Hydrochloride Liposome Injection |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 18-60 years (inclusive) at the time of signing the informed consent form, both men and women;
- Participants meet the 2015 international consensus diagnostic criteria for neuromyelitis optica spectrum disease (NMOSD) and are AQP4-IgG positive or negative (acceptable test results within 24 weeks before signing the informed consent form);
- Experienced at least 2 attacks within 1 year before screening and/or at least 3 relapses in the past 24 months, but have at least 1 documented clinical evidence of attack or relapse (including the first attack) within 12 months before screening;
- Expanded disability status scale (EDSS) ≤ 7.5 points at screening and baseline retest;
- Participants voluntarily sign the informed consent form and voluntarily complete the trial according to the protocol requirements.
Key exclusion criteria (16 listed)
- Pregnant or lactating female participants, or participants planning to have a child during the study;
- Any surgical procedure within 4 weeks before randomization, with evidence of other demyelinating diseases or progressive multifocal leukoencephalopathy (PML);
- Known active infection (excluding nail bed fungal infection or dental caries) within 4 weeks before randomization;
- Hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCVAb), syphilis antibody, or human immunodeficiency virus (HIV antibody) is positive;
- History of drug or alcohol abuse or mental disorder within 1 year before randomization;
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 |
|---|---|---|
| Mitoxantrone Hydrochloride Liposome InjectionIV, once every 12 weeks (Q12W) | Drug | Mitoxantrone Hydrochloride Liposome Injection 12 mg/m^2 group, Mitoxantrone Hydrochloride Liposome Injection 8 mg/m^2 group, Placebo Injection every 12 weeks (Q12W). |
Primary outcome
Time to First Protocol-defined Relapse
Time frame: Up to Week 48
Time to First Protocol-defined Relapse was defined as time from randomization to first occurrence of relapse. Protocol-defined relapse was occurrence of new or worsening neurological symptoms attributable to neuromyelitis optica spectrum disorder (NMOSD).
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 7 Aug 2024.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mitoxantrone Hydrochloride Liposome Injection in people with Neuromyelitis Optica Spectrum Disorder, run by CSPC Zhongnuo Pharmaceutical (Shijiazhuang) 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 5 inclusion and 16 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 7 Aug 2024. 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. Some participants in this study may receive a placebo. 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 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?
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?
Time to First Protocol-defined Relapse (time frame: Up to Week 48). 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 7 Aug 2024.