IC14 for Treatment of Amyotrophic Lateral Sclerosis
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|ClinicalTrials.gov Identifier: NCT03508453|
Recruitment Status : Not yet recruiting
First Posted : April 25, 2018
Last Update Posted : July 2, 2018
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis Motor Neuron Disease||Biological: IC14 Other: Placebo||Phase 2|
This will be a placebo-controlled, double-blind, parallel-group comparison.
Fifty patients with rapidly progressive ALS will be randomised to receive one of the following regimens:
- IC14 4 mg/kg given intravenously twice weekly for 12 weeks; or
- Identical-appearing placebo given intravenously twice weekly for 12 weeks. There will be an interim safety review by an independent Data Safety Monitoring Board after the initial 20 subjects have completed 4 weeks and 8 weeks of treatment. Study observation will continue until 12 weeks after the last dose of study drug.
The primary endpoint is:
• Treatment-related change in disease biomarker profiles [e.g., neurofilaments (Nf), urinary p75 neurotrophin receptor (p75NTR), cytokines, and soluble CD14].
The secondary endpoints are:
- Safety, tolerability and lack of immunogenicity of IC14.
- Treatment-related change in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R).
- Treatment-related change in respiratory function by seated forced vital capacity (FVC) parameters.
- Treatment-related change in inspiratory muscle strength by sniff nasal pressure (SNP) test.
- Treatment-related change in quality of life by the ALS Specific Quality of Life-Revised (ALSSQOL-R) score.
- Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioural Assessment (ECAS) score.
- Treatment-related changes stratified by disease severity and prognostic indicators.
- Peak serum IC14 concentration following administration of the initial dose and peak serum concentration following a course of treatment.
- Area under the serum IC14 concentration versus time curve (AUC) following administration of the initial dose and following a course of treatment.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Randomized, Double-Blind, Placebo-Controlled|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||Pharmacy preparation of identical-appearing placebo|
|Official Title:||A Phase 2, Randomised, Double-Blind, Placebo-Controlled Study of IC14 for Treatment of Patients With Rapidly Progressive Motor Neuron Disease|
|Estimated Study Start Date :||August 15, 2018|
|Estimated Primary Completion Date :||May 15, 2020|
|Estimated Study Completion Date :||December 15, 2020|
Active Comparator: IC14 (monoclonal anti-CD14 antibody)
IC14 4 mg/kg intravenously twice weekly for 12 weeks
Monoclonal antibody against CD14
Placebo Comparator: Placebo
Placebo intravenously twice weekly for 12 weeks
sterile normal saline for injection prepared to be identical to study drug
- Neurofilament (biomarker) [ Time Frame: 12 weeks ]Treatment-related change in concentration of neurofilament (picograms per milliliter)
- Urinary p75 neurotrophin receptor (biomarker) [ Time Frame: 12 weeks ]Treatment-related change in concentration of urinary p75 neurotrophin receptor (nanograms per milligram creatinine)
- Monocyte CD14 receptor occupancy [ Time Frame: 12 weeks ]Treatment-related change in percent monocyte receptor occupancy
- Functional status [ Time Frame: 12 weeks ]Treatment-related change in Revised Amyotrophic Lateral Sclerosis Functional Rating Scale [0 (worst) to 48 (best)]
- Respiratory function [ Time Frame: 12 weeks ]Treatment-related change in percent normal Forced Vital Capacity [0% (worst) to 100%(best)]
- Muscle function [ Time Frame: 12 weeks ]Treatment-related change in percent normal Sniff Nasal Pressure [0% (worst) to 100% (best)]
- Quality of life [ Time Frame: 12 weeks ]Treatment-related change in Amyotrophic Lateral Sclerosis Specific Quality of Life - Revised questionnaire [0 (worst) to 460 (best)]
- Cognitive and behavioural assessment [ Time Frame: 12 weeks ]Treatment-related change in Edinburgh Cognitive and Behavioural Assessment Score [0(worst) to 136 (best)]
- Maximum plasma concentration (Cmax) [ Time Frame: 12 weeks ]Maximum serum IC14 concentration (micrograms per milliliter)
- Area under the curve [ Time Frame: 12 weeks ]Area under the curve for serum IC14 (microgram x hr/mL)
- Immunogenicity [ Time Frame: 16 weeks ]Development of human anti-monoclonal antibodies following treatment
- Adverse events (safety, tolerability) [ Time Frame: 16 weeks ]Incidence of treatment-emergent adverse events (safety, tolerability) classified by MedDRA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03508453
|Contact: Susan Heggie||+61 7 3646 firstname.lastname@example.org|
|Contact: Kathryn Thorpe||+61 7 3646 email@example.com|
|Royal Brigham and Women's Hospital|
|Herston, Queensland, Australia, 4006|
|Study Chair:||Robert D Henderson, MBBS||Royal Brisbane & Women's Hospital|