TCTR20210716002RecruitingPhase 3
The pilot study of autologous hematopoietic stem cell transplantation in neuromyelitis optica patients at Siriraj Hospital
Faculty of Medicine Siriraj Hospital, Mahidol University0 sites10 target enrollmentStarted: July 16, 2021Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Enrollment
- 10
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 18 Years to 55 Years (—)
- Sex
- All
Inclusion Criteria
- •Participants are eligible to be included in the study only if all the following criteria apply:
- •1. Are diagnosed with aquaporin-4 seropositive NMOSD
- •2. Have one or more documented relapses within the past 2 years before screening with either:
- •a. one clinical attack OR
- •b. one gadolinium-enhanced lesion on a T1-weighted MRI
- •3. Are fully ambulatory with or without any gait aid and have a baseline (day 1) EDSS score of 4.0 to 6.5
- •4. Suffered from at least one severe attack as defined by arm or leg weakness worse than MRC grade 3 or a visual acuity below 20/200 in at least one eye
- •5. Are neurologically stable for > 30 days prior to screening
- •6. Have a disease duration of 2 to 10 years
- •7. Have no relapses within 3 months before HSCT
- •8. Have been treated with an immunosuppressive agent such as azathioprine, mycophenolate mofetil, or an immunomodulatory agent such as rituximab without disease remission
- •9. Agree to use barrier contraceptive methods or abstain from sexual intercourse throughout the study period
- •10. Accept the risk of infertility as a side effect of HSCT
- •11. Have no other significant medical comorbidities such as liver or renal diseases as determined by investigators
- •12. Are capable of giving a sign informed consent which includes adherence to the requirements and restrictions listed in the informed consent form and this protocol
- •13. Must be contractable by email or telephone throughout the study
Exclusion Criteria
- •Participants are excluded from the study if any of the following criteria apply:
- •1. Attempted suicide within 6 months prior to screening
- •2. Have major depression within 6 months prior to screening (clinically stable minor depression is not exclusionary)
- •3. Have a history of splenectomy at any time or any major surgery within 2 months prior to screening
- •4. Have a history of myocardial infarction or cerebrovascular event within 6 months prior to screening, or current active angina pectoris, history of or current congestive heart failure New York Heart Association (NYHA) Class III or Class IV, uncontrolled seizures (remote infantile febrile seizures are not exclusionary), prolonged untreated hypertension (systolic above 160 mmHg and/or diastolic above 100 mmHg), active gastrointestinal bleeding, or any other significant active medical condition in the investigator's opinion
- •5. Have a left ventricular ejection fraction (LVEF) of less than 45 percent from echocardiography
- •6. Have a history or current diagnosis of progressive multifocal leukoencephalopathy (PML)
- •7. Have an active, clinically significant viral, bacterial, or fungal infection, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 4 weeks of screening, or completion of oral anti-infectives within 2 weeks before or during screening, or a history of recurrent infections (i.e., 3 or more of the same type of infection in a 12-month rolling period). Vaginal candidiasis, onychomycosis, and genital or oral herpes simplex virus infection considered by the investigator to be sufficiently controlled would not be exclusionary
- •8. Have a history or current diagnosis of active tuberculosis (TB) or is currently undergoing treatment for latent TB infection (LTBI)
- •9. Have any of the following:
- •a. History of or positive for human immunodeficiency virus (HIV) at screening
- •b. History of or positive for hepatitis C virus (HCV) antibody and/or HCV RNA by PCR at screening. However, if a participant has a history of HCV infection and has completed and documented appropriate treatment at least 1 year prior to screening AND is negative for HCV RNA by PCR at screening, participants will not be excluded from the study
- •c. Positive for hepatitis B surface antigen (HBsAg) at screening
- •d. For participants who are negative for HBsAg at screening but are anti-hepatitis B surface antibody positive without history of vaccination for Hepatitis B and/or anti-hepatitis B core antibody positive with or without history of vaccination for Hepatitis B at screening, reflex testing for hepatitis B virus DNA (HBV DNA) by PCR will be performed:
- •i. Hepatitis B antibody positive participants who have detectable HBV DNA are excluded
- •ii. Hepatitis B antibody positive participants who are HBV DNA negative are not excluded from the study. However, these participants will have HBV DNA monitoring by PCR
- •10. Have an estimated glomerular filtration rate (eGFR) of less than 60 mL/min/1.73 m2 as calculated by the 4 variable Modification of Diet in Renal-Disease equation by the central laboratory or any renal condition that would preclude the administration of gadolinium (e.g., acute kidney injury)
- •11. Have ALT, AST more than 2 times the ULN of laboratory reference range or total bilirubin higher than 1.5 times the ULN, or any other clinically significant laboratory abnormality
- •12. Have significant cytopenia, including neutrophil co
Investigators
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