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A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder

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ClinicalTrials.gov Identifier: NCT04155424
Recruitment Status : Not yet recruiting
First Posted : November 7, 2019
Last Update Posted : November 7, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE November 5, 2019
First Posted Date  ICMJE November 7, 2019
Last Update Posted Date November 7, 2019
Estimated Study Start Date  ICMJE November 2019
Estimated Primary Completion Date November 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 5, 2019)
  • Change From Baseline In The Annualized Relapse Rate At 52/53 Weeks [ Time Frame: Baseline, Week 52/53 ]
  • Time To First Relapse [ Time Frame: Baseline up to Week 52/53 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: November 5, 2019)
  • Change From Baseline In Expanded Disability Status Scale (EDSS) Score At 52/53 Weeks In Participants ≥5 Years Of Age [ Time Frame: Baseline, Week 52/53 ]
  • Change From Baseline In The Hauser Ambulatory Index Score At 52/53 Weeks [ Time Frame: Baseline, Week 52/53 ]
  • Change From Baseline In Pediatric Quality Of Life Inventory (PedsQL) At 52/53 Weeks In Participants ≥5 Years Of Age [ Time Frame: Baseline, Week 52/53 ]
  • Change From Baseline In Pediatric Quality Of Life Inventory Parent Proxy (PedsQL Parent Proxy) At 52/53 Weeks In Participants < 5 Years Of Age [ Time Frame: Baseline, Week 52/53 ]
  • Change From Baseline In Visual Acuity As Measured By The Snellen Or LEA Symbols Eye Chart Examination At 52/53 Weeks [ Time Frame: Baseline, Week 52/53 ]
  • Change From Baseline In Confrontational Visual Fields As Measured During Ophthalmologic Examination At 52/53 Weeks [ Time Frame: Baseline, Week 52/53 ]
  • Change From Baseline In Color Vision As Measured During Ophthalmologic Examination At 52/53 Weeks [ Time Frame: Baseline, Week 52/53 ]
  • Serum Eculizumab Concentration Over Time [ Time Frame: Baseline through Week 52/53 ]
  • Serum Free Complement Protein 5 Concentrations Over Time [ Time Frame: Baseline through Week 52/53 ]
  • In Vitro Hemolytic Activity Over Time [ Time Frame: Baseline through Week 52/53 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder
Official Title  ICMJE A Phase 2/3 Open-Label, Single-Arm Trial to Evaluate the Safety and Activity of Eculizumab in Pediatric Patients With Relapsing Neuromyelitis Optica Spectrum Disorder
Brief Summary The objective of this study is to evaluate the safety and efficacy of eculizumab in pediatric participants (aged 2 to < 18 years) with relapsing neuromyelitis optica spectrum disorder (NMOSD).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Neuromyelitis Optica
  • Neuromyelitis Optica Spectrum Disorder
Intervention  ICMJE Drug: Eculizumab
Following a weight-based weekly dose of eculizumab during an induction phase, participants will receive weight-based doses of eculizumab every 2 weeks during the Primary Treatment Period and Extension Treatment Period.
Other Name: Soliris
Study Arms  ICMJE Experimental: Eculizumab

All participants will receive open-label eculizumab by intravenous infusion during the Primary Treatment Period, starting on Day 1 and for a total of 52/53 weeks. The dosing regimen will be based on the participant's body weight. As body weight changes during the study, the participant's weight cohort and dose may change accordingly.

After completing the 52/53-week Primary Treatment Period, participants may continue receiving eculizumab in the Extension Treatment Period for 104 weeks.

Intervention: Drug: Eculizumab
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: November 5, 2019)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2024
Estimated Primary Completion Date November 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Male or female participants aged 2 years to < 18 years with body weight ≥ 10 kilograms (kg).
  2. Vaccinated against Neisseria meningitidis within 3 years prior to, or at the time of initiating eculizumab. Participants who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive appropriate prophylactic antibiotics until 2 weeks after the vaccination.
  3. Documented vaccination against haemophilus influenzae type b and streptococcus pneumoniae infections at least 2 weeks prior to dosing as per local and country-specific immunization guidelines for the appropriate age group.
  4. Anti-aquaporin-4 antibody-positive and diagnosis of NMOSD as defined by the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria.
  5. Historical Relapse Rate of at least 2 relapses in the last 2 years, and with at least 1 relapse in the year prior to Screening.
  6. EDSS score ≤ 7.
  7. Participants who enter the study receiving supportive immunosuppressive therapies (ISTs) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration.
  8. Female participants of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin) at Screening and follow protocol-specified contraception guidance for avoiding pregnancy while on treatment and for 5 months after the last dose of eculizumab.
  9. Male participants with a female spouse/partner of childbearing potential or a pregnant or breastfeeding spouse or partner must agree to use double barrier contraception (male condom plus appropriate barrier method for the female partner) while on treatment and for at least 5 months after the last dose of eculizumab.

Exclusion Criteria:

  1. Parent or legal guardian is an Alexion employee.
  2. Pregnant, breastfeeding, or intending to conceive during the course of the study.
  3. Participants known to be human immunodeficiency virus positive or with congenital immunodeficiency.
  4. Unresolved meningococcal or other serious infection.
  5. Any unresolved acute or chronic systemic bacterial or other infection that is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics.
  6. Use of rituximab or other biologicals such as tocilizumab within 6 months prior to Screening.
  7. Use of mitoxantrone within 3 months prior to Screening.
  8. Use of intravenous immunoglobulin or plasma exchange within 3 weeks prior to Screening.
  9. Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.
  10. Has previously received treatment with eculizumab or other complement inhibitors.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Alexion Pharmaceuticals, Inc. 855-752-2356 clinicaltrials@alexion.com
Listed Location Countries  ICMJE Canada,   Germany,   Italy,   Japan,   Korea, Republic of,   Spain,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04155424
Other Study ID Numbers  ICMJE ECU-NMO-303
2019-001829-26 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Alexion Pharmaceuticals
Study Sponsor  ICMJE Alexion Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date November 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP