Trials Sponsors Conditions Drugs Pricing

Conditions

Familial LCAT Deficiency

Keywords

Recombinant LCATNatural History

Outcome Measures

Primary Outcomes (1)

HDL
Increased
weekly

Secondary Outcomes (1)

LCAT
weekly

Study Arms (1)

1

This is an expanded use protocol for a named subject.

Drug: Recombinant LCAT

Interventions

Recombinant LCATDrug

iv infusion

Eligibility Criteria

Age18 Years - 100 Years

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

Single patient

You may qualify if:

Greater than or equal to 18 years at screening;

Sponsors & Collaborators

National Heart, Lung, and Blood Institute (NHLBI)
NIHlead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Related Publications (3)

Barter PJ. Hugh sinclair lecture: the regulation and remodelling of HDL by plasma factors. Atheroscler Suppl. 2002 Dec;3(4):39-47. doi: 10.1016/s1567-5688(02)00041-7.
PMID: 12573362BACKGROUND
Lee JY, Badeau RM, Mulya A, Boudyguina E, Gebre AK, Smith TL, Parks JS. Functional LCAT deficiency in human apolipoprotein A-I transgenic, SR-BI knockout mice. J Lipid Res. 2007 May;48(5):1052-61. doi: 10.1194/jlr.M600417-JLR200. Epub 2007 Feb 1.
PMID: 17272829BACKGROUND

MeSH Terms

Conditions

Lecithin Cholesterol Acyltransferase Deficiency

Condition Hierarchy (Ancestors)

HypoalphalipoproteinemiasHypolipoproteinemiasLipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

Robert D Shamburek, M.D.
National Heart, Lung, and Blood Institute (NHLBI)
PRINCIPAL INVESTIGATOR

Enrollment

1 participants (actual enrollment reported by the registry).

Study Design

Study Type: observational
Observational Model: OTHER
Time Perspective: PROSPECTIVE
Sponsor Type: NIH
Responsible Party: SPONSOR

Study Record Dates

First Submitted

February 3, 2021

First Posted

February 4, 2021

Study Start

January 24, 2013

Actual (reported)

Intravenous ACP-501 for Familial LCAT Deficiency (rhLCAT)

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

Primary Completion

First Submitted

First Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (1)

Study Arms (1)

1

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (3)

Related Links

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

Primary Completion

First Submitted

First Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (1)

Study Arms (1)

1

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (3)

Related Links

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations