Trial · NCT03679598

Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

NCT03679598, Phase 2, Completed, University of Alabama at Birmingham; results first posted 2024-07-09

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency. Trials, record NCT03679598. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03679598

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
NCT number: NCT03679598
Status: Completed
Phases: Phase 2
Enrollment: 63 participants, actual
Lead sponsor: University of Alabama at Birmingham
Conditions: Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations, Emphysema or COPD
Interventions: Alvelestat (MPH966), Placebo
First posted: 2018-09-20
Last updated: 2024-08-20
Results first posted: 2024-07-09

Source ClinicalTrials.gov

Registered outcomes3

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Within-individual % Change in Plasma Desmosine/Isodesmosine	baseline, week 12	To evaluate the effect of alvelestat (MPH966) administered twice daily (bid) for 12 weeks on blood markers of within-individual % change in plasma desmosine/isodesmosine will be measured.
Primary	Numbers and % of Subjects Who Experience at Least 1 Treatment-emergent Adverse Event	baseline, week 16	To evaluate the safety and tolerability of alvelestat (MPH966) administered twice daily (bid) for 12 weeks treatment numbers and % of subjects who experience at least 1 treatment-emergent adverse event (TEAE) will be measured.
Secondary	Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo	baseline, week 12	To evaluate the effect of Alvelestat (MPH966) on other blood pharmacodynamic markers of neutrophil activation and elastase activity including plasma desmosine/isodesmosine (change compared to placebo; within-individual change is the primary outcome), plasma Aa-Val-360, serum NE activity, plasma proteinase 3, plasma cathepsin B, plasma CRP, plasma IL-6, plasma IL-8, plasma IL-1b, plasma RANTES, plasma LTB4, plasma MMP9, plasma MMP12, plasma MPO, and plasma PGP.

Source ClinicalTrials.gov

Outcomes3

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Within-individual % Change in Plasma Desmosine/Isodesmosine	baseline, week 12	% change	MEAN
PRIMARY	Numbers and % of Subjects Who Experience at Least 1 Treatment-emergent Adverse Event	baseline, week 16	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo	baseline, week 12	ng/ mL	MEAN

Source ClinicalTrials.gov

Results by group34

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Within-individual % Change in Plasma Desmosine/Isodesmosine	Alvelestat (MPH966)		10.0619	Standard Deviation 27.8489	% change
Within-individual % Change in Plasma Desmosine/Isodesmosine	Placebo		8.5627	Standard Deviation 29.2973	% change
Numbers and % of Subjects Who Experience at Least 1 Treatment-emergent Adverse Event	Alvelestat (MPH966)		25		Participants
Numbers and % of Subjects Who Experience at Least 1 Treatment-emergent Adverse Event	Placebo		23		Participants
Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo	Alvelestat (MPH966)	Plasma desmosine/isodesmosine	0.0307	Standard Deviation 0.1012	ng/ mL
Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo	Placebo	Plasma desmosine/isodesmosine	0.0047	Standard Deviation 0.0817	ng/ mL
Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo	Alvelestat (MPH966)	Plasma Aa-Val-360	-1.6279	Standard Deviation 4.1028	ng/ mL
Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo	Placebo	Plasma Aa-Val-360	-0.9937	Standard Deviation 4.3209	ng/ mL
Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo	Alvelestat (MPH966)	Serum NE	-16.7926	Standard Deviation 41.1503	ng/ mL
Blood Pharmacodynamic Markers of Neutrophil Activation and Elastase Activity. Change From Baseline in the Following Outcomes at End of the Treatment Within Patient and Compared to Placebo	Placebo	Serum NE	3.9660	Standard Deviation 19.4462	ng/ mL

1 to 10 of 34Source ClinicalTrials.gov

See all 34 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Alvelestat (MPH966)	32
Overall Study	STARTED	Placebo	31
Overall Study	COMPLETED	Alvelestat (MPH966)	30
Overall Study	COMPLETED	Placebo	31
Overall Study	NOT COMPLETED	Alvelestat (MPH966)	2
Overall Study	NOT COMPLETED	Placebo	0

Source ClinicalTrials.gov

Adverse events24

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Headache	Nervous system disorders	Alvelestat (MPH966)	12	32	15
other	Headache	Nervous system disorders	Placebo	7	31	7
other	Migraine	Nervous system disorders	Alvelestat (MPH966)	2	32	2
other	Migraine	Nervous system disorders	Placebo	2	31	3
other	Restless legs syndrome	Nervous system disorders	Alvelestat (MPH966)	1	32	2
other	Restless legs syndrome	Nervous system disorders	Placebo	0	31	0
other	Sinus headache	Nervous system disorders	Alvelestat (MPH966)	2	32	2
other	Sinus headache	Nervous system disorders	Placebo	0	31	0
other	COPD	Respiratory, thoracic and mediastinal disorders	Alvelestat (MPH966)	1	32	1
other	COPD	Respiratory, thoracic and mediastinal disorders	Placebo	7	31	7

1 to 10 of 24Source ClinicalTrials.gov

See all 24 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Alvelestat (MPH966)	0	32
Total, all-cause mortality	Placebo	0	31
Total, other adverse events	Alvelestat (MPH966)	25	32
Total, other adverse events	Placebo	23	31
Total, serious adverse events	Alvelestat (MPH966)	0	32
Total, serious adverse events	Placebo	0	31

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants1

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
UH3TR002450	AZD9688: A First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency a Genetically Linked Orphan Disease	DRANSFIELD, MARK T; ABAN, INMACULADA	UNIVERSITY OF ALABAMA AT BIRMINGHAM	NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES	2018 to 2021

Source NIH RePORTER

Trial comparisons3

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Alvelestat (MPH966) vs Placebo: Numbers and % of Subjects Who Experience at Least 1 Treatment-emergent Adverse Event	Trial comparisons
Alvelestat (MPH966) vs Placebo: deaths from any cause	Trial comparisons
Alvelestat (MPH966) vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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