Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency (ATALANTa)
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ClinicalTrials.gov Identifier: NCT03679598 |
Recruitment Status :
Completed
First Posted : September 20, 2018
Last Update Posted : December 15, 2023
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Tracking Information | |||||
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First Submitted Date ICMJE | September 18, 2018 | ||||
First Posted Date ICMJE | September 20, 2018 | ||||
Last Update Posted Date | December 15, 2023 | ||||
Actual Study Start Date ICMJE | April 8, 2019 | ||||
Actual Primary Completion Date | November 30, 2023 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | |||||
Current Secondary Outcome Measures ICMJE | Not Provided | ||||
Original Secondary Outcome Measures ICMJE | Not Provided | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency | ||||
Official Title ICMJE | A First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency a Genetically Linked Orphan Disease | ||||
Brief Summary | This is a Phase 2, multicenter, double-blind, randomized (1:1), placebo-controlled, 12-week, proof-of-concept study to evaluate the safety and tolerability as well as the mechanistic effect of oral administration of alvelestat (MPH966) in subjects with confirmed AATD defined as Pi*ZZ, Pi*SZ, Pi*null, or another rare phenotype/genotype known to be associated with either low (serum AAT level <11 μM or <57.2 mg/dL) or functionally impaired AAT including "F" or "I" mutations. | ||||
Detailed Description | Alpha-1 antitrypsin deficiency (AATD) is the most common genetic cause of chronic obstructive pulmonary disease (COPD) and early-onset emphysema. AATD is characterized by low AAT levels; leading to excessive neutrophil elastase (NE) mediated lung destruction. Current treatment requires the periodic infusion of pooled AAT derived from human plasma, but this therapeutic approach (termed augmentation) does not definitively slow the rate of emphysema progressionlung function decline and is very expensive. In addition, it is not clear that the currently recommended dose for augmentation fully controls lung inflammation and destruction. Alvelestat (MPH966, formerly AZD9668) is a potent, selective, and reversible, oral inhibitor of human NE. Suppression of NE is expected to reduce lung damage and may slow disease progression. This study is to establish proof of clinical concept by investigating the mechanistic effect and safety of alvelestat (MPH966) in patients with AATD. | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 2 | ||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Intervention Model Description: randomized (1:1), placebo-controlled Masking: Triple (Participant, Investigator, Outcomes Assessor)Masking Description: double blind Primary Purpose: Treatment
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Condition ICMJE |
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Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
63 | ||||
Original Estimated Enrollment ICMJE |
66 | ||||
Actual Study Completion Date ICMJE | December 1, 2023 | ||||
Actual Primary Completion Date | November 30, 2023 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria: Participants are eligible to be included in the study only if ALL of the following criteria apply:
Exclusion Criteria: Participants are excluded from the study if ANY of the following criteria apply: Excluded Medical Conditions
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years to 80 Years (Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT03679598 | ||||
Other Study ID Numbers ICMJE | IRB-300002338 4UH3TR002450-02 ( U.S. NIH Grant/Contract ) |
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Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | Mark Dransfield, MD, University of Alabama at Birmingham | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | University of Alabama at Birmingham | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | University of Alabama at Birmingham | ||||
Verification Date | December 2023 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |