Clinical Trial: Efficacy and Safety of Mitoxantrone in Patients With Refractory Neuromyelitis Optica and Spectrum Disorders

Study Status: Active, not recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Efficacy and Safety of Mitoxantrone in Patients With Refractory Neuromyelitis Optica and Spectrum Disorders

Brief Summary: The treatment protocol consisted of 12 mg/m2 MITO intravenous infusions every 3 months for 2 years. Dosage was adjusted according to side effects. Neurological assessment including the determination of the Expanded Disability Status Scale (EDSS) score and ophthalmologic evaluations were performed every 3 months and during relapses. Flow cytometric analysis, brain and spinal cord MRI was performed at baseline, 6, 12, 18, and 24 months.

Detailed Summary:
Sponsor: Xuanwu Hospital, Beijing

Current Primary Outcome:

• annual relapse rate (ARR) [ Time Frame: one year ]
  ARR is defined as the number of confirmed relapses in a year. The number of annual relapse rate was used as parameters of effectiveness and was compared between premitoxantrone and postmitoxantrone treatment during the follow-up period.
• EDSS [ Time Frame: six months ]
  Expanded Disability Status Scale (EDSS) scores was used as parameters of effectiveness and was compared between premitoxantrone and postmitoxantrone treatment during the follow-up period.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Changes in LVEF [ Time Frame: six months ]
  - Assessment of cardiac function: Changes in LVEF by transthoracic echocardiography and determination of cardiac side effects by ECG and by measurement of CK-MB, Troponin and BNP.
• blood cell count [ Time Frame: three months ]
  - Assessment of hematological system: Monitoring blood cell count regularly. Considering marrow puncture if necessary.
• Flow cytometric analysis [ Time Frame: six months ]
  Immunofluorescent staining of wholeblood samples were performed of blood drawing using antibodies against CD3/CD4/CD8/CD19/CD20/CD56 with isotype controls, followed by lysis of red blood cells and immediate acquisition and analysis by flow cytometry.

Original Secondary Outcome: Same as current

Information By: Xuanwu Hospital, Beijing

Dates:
Date Received: September 3, 2013
Date Started: March 2009
Date Completion: December 2015
Last Updated: December 19, 2013
Last Verified: December 2013