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

NCT04155424 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: ECU-NMO-3032019-001829-26
• Study Type: interventional
• Target: 5
• Locations: 7 countries
• Sites: 35

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).

Conditions

Neuromyelitis Optica; Neuromyelitis Optica Spectrum Disorder

Keywords

Neuromyelitis Optica; Neuromyelitis Optica Spectrum Disorder; Devic's Disease; Transverse Myelitis; Optic Neuritis; Relapse; Eculizumab; Soliris; CNS Autoimmune Disorders; Demyelinating Disorders; NMO; NMOSD

Outcome Measures

Primary Outcomes (2)

• Change Between the Baseline Annualized Relapse Rate (ARR) and the On-Trial ARR at Week 52/53

  ARR was calculated as the number of relapses for each participant divided by the number of years of treatment for that participant. Baseline ARR was based on 24 months prior to screening.

  Baseline, Week 52/53

• Time to First On-trial Relapse

  Time to First Relapse was defined as beginning at the time the participant's first dose of eculizumab was administered until the participant's first on-trial relapse was reported by the Investigator. Participants who did not experience an on-trial relapse were censored at the end of the study period.

  Baseline up to Week 52/53

Secondary Outcomes (8)

• Change From Baseline in Expanded Disability Status Scale (EDSS) Score at Week 52/53

  Baseline, Week 52/53

• Change From Baseline in the Hauser Ambulation Index (HAI) Score at Week 52/53

  Baseline, Weeks 52/53

• Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Score at Week 52/53

  Baseline, Weeks 52/53

• Number of Participants With Shift From Baseline in Visual Acuity (VA)

  Baseline, Week 52/53

• Number of Participants With Shift From Baseline in Confrontational Visual Fields (VF)

  Baseline, Weeks 52/53

Study Arms (1)

Eculizumab

EXPERIMENTAL

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.

Drug: Eculizumab

Interventions

Eculizumab DRUG

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.

Also known as: Soliris

Eculizumab

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female participants aged 2 years to \< 18 years with body weight ≥ 10 kilograms (kg).
• 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.
• 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.
• Anti-aquaporin-4 antibody-positive and diagnosis of NMOSD as defined by the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria.
• 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.
• EDSS score ≤ 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.
• 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.
• 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.

You may not qualify if:

• Parent or legal guardian is an Alexion employee.
• Pregnant, breastfeeding, or intending to conceive during the course of the study.
• Participants known to be human immunodeficiency virus positive or with congenital immunodeficiency.
• Unresolved meningococcal or other serious infection.
• 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.
• Use of rituximab or other biologicals such as tocilizumab within 6 months prior to Screening.
• Use of mitoxantrone within 3 months prior to Screening.
• Use of intravenous immunoglobulin or plasma exchange within 3 weeks prior to Screening.
• Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.
• Has previously received treatment with eculizumab or other complement inhibitors.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (35)

Clinical Trial Site

San Francisco, California, 94016, United States

Location

Clinical Trial Site

Washington D.C., District of Columbia, 20001, United States

Location

Clinical Trial Site

Miami, Florida, 33101, United States

Location

Clinical Trial Site

Atlanta, Georgia, 30301, United States

Location

Research Site

Baltimore, Maryland, 21287, United States

Location

Clinical Trial Site

Rockville, Maryland, 20847, United States

Location

Clinical Trial Site

Boston, Massachusetts, 02101, United States

Location

Research Site

Boston, Massachusetts, 02115, United States

Location

Research Site

St Louis, Missouri, 63130, United States

Location

Research Site

Hackensack, New Jersey, 07601, United States

Location

Research Site

New Brunswick, New Jersey, 08901, United States

Location

Research Site

New York, New York, 10016, United States

Location

Clinical Trial Site

Chapel Hill, North Carolina, 27514, United States

Location

Clinical Trial Site

Philadelphia, Pennsylvania, 19019, United States

Location

Research Site

Dallas, Texas, 75390, United States

Location

Research Site

Calgary, Alberta, T3B 6A8, Canada

Location

Clinical Trial Site

Edmonton, Alberta, Canada

Location

Research Site

Toronto, Ontario, M5G 1X8, Canada

Location

Clinical Trial Site

Toronto, Ontario, Canada

Location

Research Site

Montreal, Quebec, H3T1C5, Canada

Location

Clinical Trial Site

Regensburger Straße, Goettingen, Germany

Location

Research Site

Datteln, 45711, Germany

Location

Research Site

Münster, 48149, Germany

Location

Research Site

Catania, 95123, Italy

Location

Research Site

Gallarate, 21013, Italy

Location

Clinical Trial Site

Genoa, Italy

Location

Research Site

Genova, 16132, Italy

Location

Research Site

Rome, 00178, Italy

Location

Clinical Trial Site

Rome, Italy

Location

Research Site

Yokohama, 232-0024, Japan

Location

Clinical Trial Site

Yokohama, Japan

Location

Clinical Trial Site

Seoul, South Korea

Location

Clinical Trial Site

Barcelona, Spain

Location

Research Site

Esplugues de Llobregat, 8950, Spain

Location

Clinical Trial Site

Seville, Spain

Location

Related Links

• Related Info

MeSH Terms

Conditions

Neuromyelitis Optica; Myelitis, Transverse; Optic Neuritis; Recurrence; Demyelinating Diseases

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases; Myelitis; Central Nervous System Infections; Infections; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Central Nervous System Diseases; Spinal Cord Diseases; Neurodegenerative Diseases; Neuroinflammatory Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

The study was terminated by Alexion, as only 5 of the planned 12 participants were enrolled due to difficulty in recruitment.

Results Point of Contact

• Title: Alexion Pharmaceuticals Inc.
• Organization: Alexion Pharmaceuticals Inc.

clinicaltrials@alexion.com

855-752-2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 5, 2019
• First Posted: November 7, 2019
• Study Start: January 14, 2020
• Primary Completion: July 31, 2023
• Study Completion: July 31, 2023
• Last Updated: September 25, 2024
• Results First Posted: September 25, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3); DE (3); IT (6); JP (2); KR (1); US (15); CA (5)