Alpha-1 Foundation Research Registry | NCT00499941

Studies
Alpha-1 Foundation Research Registry

NCT00499941

Sponsor

Medical University of South Carolina

Status

Completed

Last Update Posted

Mar 3, 2021Actual

Enrollment

6,655Actual

Phase

Not provided

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2021

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 2000

Primary Completion Date

Jan 2021Actual

Completion Date

Jan 2021Actual

First Submitted Date

Jul 11, 2007

First Submission Date that Met QC Criteria

Jul 11, 2007

First Posted Date

Jul 12, 2007Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 1, 2021

Last Update Posted Date

Mar 3, 2021Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Charlie Strange, Professor of Pulmonary and Critical Care Medicine, Medical University of South CarolinaPrincipal Investigator

Lead Sponsor

Medical University of South CarolinaOTHER

Collaborators

Name	Class
Alpha-1 Foundation	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The Alpha-1 Foundation Research Registry is a confidential database made up of individuals diagnosed with severe alpha-1 antitrypsin deficiency (Alpha-1) or the carrier state.

Detailed Description

The Registry was established in 1997 by the Alpha-1 Foundation to facilitate research initiatives and promote the development of improved treatments and a cure for Alpha-1. Located at the Medical University of South Carolina (MUSC) in Charleston, the Registry employs procedures that ensure the most stringent confidentiality of participants. The Registry operates under the direction of the Alpha-1 Foundation Board of Directors and is guided by an Advisory Committee comprised of leaders in the medical, ethical, scientific and Alpha communities. Individuals enrolled in the Registry have the ongoing opportunity to participate directly in clinical trials of new therapeutic approaches in addition to other research opportunities.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

20 Years

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

6,655Actual

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
To study the natural history of PiZZ, PiSZ, and PiMZ alpha-1 antitrypsin deficiency		Not limited

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Individuals of all ages who are diagnosed with severe alpha-1 antitrypsin deficiency or the carrier state

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Individuals of all ages who are diagnosed with severe alpha-1 antitrypsin deficiency or the carrier state

Sampling Method

Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Charlie Strange, M.D.	Medical University of South Carolina	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Medical University of South Carolina. Division of Pulmonary and Critical Care Medicine		Charleston	South Carolina	29425	United States

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
24295190	Background	Williams PH, Shore L, Sineath M, Quill J, Warner B, Keith J, Walker D, Wienke S, Flavin S, Strange C. Genetics' influence on patient experiences with a rare chronic disorder: a photovoice study of living with alpha-1 antitrypsin deficiency. Nurs Clin North Am. 2013 Dec;48(4):627-36. doi: 10.1016/j.cnur.2013.09.008.
23934327	Background

ID	Term
D008107	Liver Diseases
D004066	Digestive System Diseases
D008171	Lung Diseases
D012140	Respiratory Tract Diseases