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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-001829-26 | EudraCT Number |
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Pediatric clinical study is not feasible.
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Expanded Disability Status Scale (EDSS) Score at Week 52/53 | Disease-related disability was measured by the EDSS. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. | Baseline, Week 52/53 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | San Francisco | California | 94016 | United States | ||
| Clinical Trial Site |
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| Label | URL |
|---|---|
| Related Info | View source |
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All 5 participants were in the >=40 kilograms (kg) weight cohort at enrollment. Therefore, all 5 participants received the same dose of study drug for the Induction Period and the Maintenance Period as described in the study arm.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab | Induction Period: Participants received eculizumab (900 milligrams [mg]) via intravenous (IV) infusion once a week for 4 weeks. Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks from fifth dose (Week 4) onwards and then every 2 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 3, 2021 | Jul 29, 2024 |
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|
| Change From Baseline in the Hauser Ambulation Index (HAI) Score at Week 52/53 |
The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranged from 0 to 9, with 0 being the best score (asymptomatic; fully ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement. |
| Baseline, Weeks 52/53 |
| Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Score at Week 52/53 | PedsQL included a child self-report for participants 5 to 18 years with a 23-item PedsQL Generic Core Scales report. The PedsQL Generic Core Scales report included 4 scales, physical functioning, emotional functioning, social functioning, and school functioning. Each item used a 5-point rating scale (from 0=never to 4=almost always). Items are reverse scored and linearly transformed to a 0 (almost always) -100 (never) scale. All summary/total scores were mean of specific items where higher score indicated better HRQoL. | Baseline, Weeks 52/53 |
| Number of Participants With Shift From Baseline in Visual Acuity (VA) | The Snellen chart was used to assess VA. The Snellen chart quantifies ability to read letters of varying sizes at a fixed distance in relation to the distance at which a participant with normal vision could read the same letters. The test was performed at a standard distance, typically 6 meters or 20 feet. The Snellen chart is typically recorded as acuity ratio distance (6 meters or 20 feet), so for normal VA it would be recorded as 20/20 or 6/6. Visual acuity was summarized according to the eye with greater worsening at the end of primary treatment period (Week 52/53). Data are presented for number of participants with a shift from baseline in VA presented per the different levels of acuity ratio distance. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group. Visual Acuity data are only reported for the categories with available data at Baseline and Week 52/53. | Baseline, Week 52/53 |
| Number of Participants With Shift From Baseline in Confrontational Visual Fields (VF) | Confrontational visual fields were summarized according to the number of quadrants with deficits across both eyes. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group. | Baseline, Weeks 52/53 |
| Number of Participants With Shift From Baseline in Color Vision | Color vision was evaluated as the shift from baseline and described for participants with normal color vision at baseline in at least one eye. Participants with 13 or less correctly identified Ishihara plates were considered as having abnormal color vision, participants with 14 or more correctly identified plates were considered as having normal color vision. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group. | Baseline, Weeks 52/53 |
| Serum Eculizumab Concentration at Week 52 | Week 52 |
| Change From Baseline in Serum Free Complement Protein 5 (C5) Concentrations at Week 52 | Baseline, Week 52 |
| Washington D.C. |
| District of Columbia |
| 20001 |
| United States |
| Clinical Trial Site | Miami | Florida | 33101 | United States |
| Clinical Trial Site | Atlanta | Georgia | 30301 | United States |
| Research Site | Baltimore | Maryland | 21287 | United States |
| Clinical Trial Site | Rockville | Maryland | 20847 | United States |
| Clinical Trial Site | Boston | Massachusetts | 02101 | United States |
| Research Site | Boston | Massachusetts | 02115 | United States |
| Research Site | St Louis | Missouri | 63130 | United States |
| Research Site | Hackensack | New Jersey | 07601 | United States |
| Research Site | New Brunswick | New Jersey | 08901 | United States |
| Research Site | New York | New York | 10016 | United States |
| Clinical Trial Site | Chapel Hill | North Carolina | 27514 | United States |
| Clinical Trial Site | Philadelphia | Pennsylvania | 19019 | United States |
| Research Site | Dallas | Texas | 75390 | United States |
| Research Site | Calgary | Alberta | T3B 6A8 | Canada |
| Clinical Trial Site | Edmonton | Alberta | Canada |
| Research Site | Toronto | Ontario | M5G 1X8 | Canada |
| Clinical Trial Site | Toronto | Ontario | Canada |
| Research Site | Montreal | Quebec | H3T1C5 | Canada |
| Clinical Trial Site | Regensburger Straße | Goettingen | Germany |
| Research Site | Datteln | 45711 | Germany |
| Research Site | Münster | 48149 | Germany |
| Research Site | Catania | 95123 | Italy |
| Research Site | Gallarate | 21013 | Italy |
| Clinical Trial Site | Genoa | Italy |
| Research Site | Genova | 16132 | Italy |
| Research Site | Rome | 00178 | Italy |
| Clinical Trial Site | Rome | Italy |
| Research Site | Yokohama | 232-0024 | Japan |
| Clinical Trial Site | Yokohama | Japan |
| Clinical Trial Site | Seoul | South Korea |
| Clinical Trial Site | Barcelona | Spain |
| Research Site | Esplugues de Llobregat | 8950 | Spain |
| Clinical Trial Site | Seville | Spain |
| Received at Least 1 Dose of Study Drug |
|
| Number of Participants Who Received Study Drug During the Induction Period |
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| Number of Participants Who Received Study Drug During the Maintenance Period |
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| COMPLETED |
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| NOT COMPLETED |
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Safety Set included all participants who received at least 1 dose of eculizumab.
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| ID | Title | Description |
|---|---|---|
| BG000 | Eculizumab | Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week for 4 weeks. Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks from fifth dose (Week 4) onwards and then every 2 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Full Analysis Set included all participants who received at least 1 dose of eculizumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | relapses per participant per year | Baseline, Week 52/53 |
|
|
| |||||||||||||||||||||||||
| Primary | 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. | Full Analysis Set included all participants who received at least 1 dose of eculizumab. Note that since no participants experienced a On-trial Relapse, data was not collected for this Outcome Measure. | Posted | Baseline up to Week 52/53 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Expanded Disability Status Scale (EDSS) Score at Week 52/53 | Disease-related disability was measured by the EDSS. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. | Full Analysis Set included all participants who received at least 1 dose of eculizumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 52/53 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in the Hauser Ambulation Index (HAI) Score at Week 52/53 | The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranged from 0 to 9, with 0 being the best score (asymptomatic; fully ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement. | Full Analysis Set included all participants who received at least 1 dose of eculizumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Weeks 52/53 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Score at Week 52/53 | PedsQL included a child self-report for participants 5 to 18 years with a 23-item PedsQL Generic Core Scales report. The PedsQL Generic Core Scales report included 4 scales, physical functioning, emotional functioning, social functioning, and school functioning. Each item used a 5-point rating scale (from 0=never to 4=almost always). Items are reverse scored and linearly transformed to a 0 (almost always) -100 (never) scale. All summary/total scores were mean of specific items where higher score indicated better HRQoL. | Full Analysis Set included all participants who received at least 1 dose of eculizumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Weeks 52/53 |
|
| ||||||||||||||||||||||||||
| Secondary | Number of Participants With Shift From Baseline in Visual Acuity (VA) | The Snellen chart was used to assess VA. The Snellen chart quantifies ability to read letters of varying sizes at a fixed distance in relation to the distance at which a participant with normal vision could read the same letters. The test was performed at a standard distance, typically 6 meters or 20 feet. The Snellen chart is typically recorded as acuity ratio distance (6 meters or 20 feet), so for normal VA it would be recorded as 20/20 or 6/6. Visual acuity was summarized according to the eye with greater worsening at the end of primary treatment period (Week 52/53). Data are presented for number of participants with a shift from baseline in VA presented per the different levels of acuity ratio distance. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group. Visual Acuity data are only reported for the categories with available data at Baseline and Week 52/53. | Full Analysis Set included all participants who received at least 1 dose of eculizumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | Baseline, Week 52/53 |
| ||||||||||||||||||||||||||||
| Secondary | Number of Participants With Shift From Baseline in Confrontational Visual Fields (VF) | Confrontational visual fields were summarized according to the number of quadrants with deficits across both eyes. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group. | Full Analysis Set included all participants who received at least 1 dose of eculizumab. | Posted | Count of Participants | Participants | Baseline, Weeks 52/53 |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Participants With Shift From Baseline in Color Vision | Color vision was evaluated as the shift from baseline and described for participants with normal color vision at baseline in at least one eye. Participants with 13 or less correctly identified Ishihara plates were considered as having abnormal color vision, participants with 14 or more correctly identified plates were considered as having normal color vision. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group. | Full Analysis Set included all participants who received at least 1 dose of eculizumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | Baseline, Weeks 52/53 |
|
| |||||||||||||||||||||||||||
| Secondary | Serum Eculizumab Concentration at Week 52 | Pharmacokinetic/Pharmacodynamic (PK/PD) analysis set included participants who received at least 1 dose of eculizumab and who had evaluable PK/PD data for the endpoint. | Posted | Mean | Standard Deviation | micrograms/milliliters | Week 52 |
|
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Serum Free Complement Protein 5 (C5) Concentrations at Week 52 | PK/PD analysis set included participants who received at least 1 dose of eculizumab and who had evaluable PK/PD data. for the endpoint. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure . | Posted | Mean | Standard Deviation | micrograms/milliliters | Baseline, Week 52 |
|
|
Baseline up to Week 53
Treatment emergent adverse events (TEAEs) are AEs with a start date on or after the date of the first dose of study intervention. TEAEs were analyzed for Safety Set which included all participants who received at least 1 dose of eculizumab.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Eculizumab | Induction Period: Participants received eculizumab (900 mg) via IV infusion once a week for 4 weeks. Maintenance Period: Participants received eculizumab (1200 mg) via IV infusion every 2 weeks from fifth dose (Week 4) onwards and then every 2 weeks. | 0 | 5 | 2 | 5 | 5 | 5 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Liver function test increased | Investigations | MedDRA v26.0 | Non-systematic Assessment |
| |
| Encephalitis meningococcal | Infections and infestations | MedDRA v26.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain lower | Gastrointestinal disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Bowel movement irregularity | Gastrointestinal disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Feeling abnormal | General disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Vaccination site erythema | General disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Vaccination site pain | General disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v26.0 | Non-systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA v26.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v26.0 | Non-systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA v26.0 | Non-systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA v26.0 | Non-systematic Assessment |
| |
| Vulvovaginal candidiasis | Infections and infestations | MedDRA v26.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Growing pains | Musculoskeletal and connective tissue disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Kyphosis | Musculoskeletal and connective tissue disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Weight increased | Investigations | MedDRA v26.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Chalazion | Eye disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Post vaccination fever | Injury, poisoning and procedural complications | MedDRA v26.0 | Non-systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Lactic acidosis | Metabolism and nutrition disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Impulse-control disorder | Psychiatric disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Amenorrhoea | Reproductive system and breast disorders | MedDRA v26.0 | Non-systematic Assessment | The N for this adverse event has been adjusted to the number of females in the study as it is a sex-specific event. |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v26.0 | Non-systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA v26.0 | Non-systematic Assessment |
|
The study was terminated by Alexion, as only 5 of the planned 12 participants were enrolled due to difficulty in recruitment.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals Inc. | Alexion Pharmaceuticals Inc. | 855-752-2356 | clinicaltrials@alexion.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 15, 2023 | Jul 29, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D009471 | Neuromyelitis Optica |
| D009188 | Myelitis, Transverse |
| D009902 | Optic Neuritis |
| D012008 | Recurrence |
| D003711 | Demyelinating Diseases |
| ID | Term |
|---|---|
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D009187 | Myelitis |
| D002494 | Central Nervous System Infections |
| D007239 | Infections |
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D002493 | Central Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D019636 | Neurodegenerative Diseases |
| D000090862 | Neuroinflammatory Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
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| ID | Term |
|---|---|
| C481642 | eculizumab |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
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|
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