Recurrent NMOSD-ON
Conditions
Interventions
200mg/ day :Subjects will receive single dose 100mg of ACT001 in the morning at Day 1 of Cycle 1, followed by a 48-hour washout period, and then receive consecutive treatment with ACT001 BID (100mg x
400mg/ day:Subjects will receive single dose 200mg of ACT001 in the morning at Day 1 of Cycle 1, followed by a 48-hour washout period, and then receive consecutive treatment with ACT001 BID (200mg x 2
800mg/ day:Subjects will receive single dose 400mg of ACT001 in the morning at Day 1 of Cycle 1, followed by a 48-hour washout period, and then receive consecutive treatment with ACT001 BID (400mg x 2
1200mg/ day:Subjects will receive single dose 600mg of ACT001 in the morning at Day 1 of Cycle 1, followed by a 48-hour washout period, and then receive consecutive treatment with ACT001 BID (600mg x
Sponsors
The First Medical Center of PLA General Hospital
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: On screening, patients must satisfy all inclusion criteria listed below before entering in this study: 1. Patients who meet the diagnostic criteria of NMOSD established in 2015 by International Panel for NMO Diagnosis (IPND), and are detected with positive serum AQP4-IgG; 2. Patients who have experienced the attack of ON (in accordance with ONTT diagnostic criteria for ON), and received the pulse treatment with hormone for previous attacks; 3. Patients of either sex who are aged >=18 years; 4. Patients with an Extended Disability Status Scale (EDSS) score ranging from 0 to 6.5 (0 and 6.5 included) at screening; 5. Patients who have normal organ function at screening and baseline: absolute neutrophil count >= 1.5 x 10^9/L, platelet (PLT) >= 100 x 10^9/L, hemoglobin >= 10 g/dL; total bilirubin = 1 year); b) Female subjects of childbearing potential must have negative serum pregnancy tests at the screening (within 7 days prior to first dose of the investigational product), and agree to adopt medically confirmed contraceptive measures (such as intrauterine device, contraceptive drugs or condoms) before enrollment and during the study until 30 days after last dose of the investigational drug; 7. Male subjects with active sex activities must agree to take barrier contraception or total abstinence; 8. Subjects who agree to sign the informed consent form (ICF) prior to enrollment.
Exclusion criteria
Exclusion criteria: Subjects who satisfy any criteria below will be excluded from this study: 1. Patient has uncontrollable infection, for example, pulmonary infection, urinary tract infection, intra-abdominal infection, infection of biliary tract, infectious meningitis, cellulitis, abscess etc., body temperature >= 38 degrees C. 2. Patients who have suffered from serious heart diseases, such as unstable angina, myocardial infarction, heart failure (NYHA grade>II), or cerebral apoplexy (excluding lacunar infarction) within 6 months prior to enrollment. 3. Patients who are known to be allergic to ACT001, its similar compounds, or any of its ingredients. 4. Patient is receiving or received the following treatments within 3 months prior to screening: immunosuppressive agents (for example: Azathioprine, Mycophenolate Mofetil, Cyclophosphamide, Methotrexate, Mitoxantrone, Tacrolimus, A ring spore element etc.), or biological preparations (for example: Rituximab, Tocilizumab, Eculizumab), or plasma exchange. Patients with hypertension which can not be controlled by drugs (systolic pressure >= 140 mmHg, diastolic pressure >= 90 mmHg) at screening and baseline; 5. Patients with diabetes mellitus which can not be controlled by drugs (glycated hemoglobin >= 7%) at screening or which can not be controlled by drugs as judged by investigator at baseline; 6. Patients who have undergone any major surgery within 4 weeks prior to first medication; 7. Female patients who are during pregnancy or lactation; 8. Patients who have human immunodeficiency virus (HIV), active hepatitis B (HBsAg positive or HBV-DNA positive), or hepatitis C (HCV antibody positive), or tuberculosis (including history of tuberculosis, active tuberculosis, latent tuberculosis, tuberculosis infection T-spot positive) or syphilis (treponema pallidum antibody positive) will be excluded; for safety, patients with HBsAg negative and HBV-DNA negative, but hepatitis B e-antibody (HBeAb) positive or hepatitis B core antibody (HbcAb) positive must be followed up for HBV-DNA at baseline and every treatment cycle, otherwise they will be excluded; 9. Patients who have an interval of <4 weeks from their withdrawal from previous study to the time ICF is obtained in this study; 10. Patients who have a definite history of mental disorders; 11. Patients who are judged by investigators to have other reasons that make it unsuitable for them to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| AE;DLT;Single-dose PK parameters including: t1/2, AUC, Cmax, AUC_%Extrap_obs, Tmax, CL/F, Vd/F, etc. In addition, for AUC0-inf and Cmax, polygonal maps are drawn with the geometric mean of each dosage group as Y-axis and dose level as X-axis, and regression analysis will be carried out to determine whether the dos;PK parameters in urine: urine output, total urine excretion rate and renal clearance rate (CLr); | — |
Secondary
| Measure | Time frame |
|---|---|
| TFR;BCVA;RP2D; | — |
Countries
China
Contacts
Public ContactWei Shihui
The First Medical Center of PLA General Hospital
Outcome results
None listed