Extension Study of AGT-181-102 to Evaluate Long Term Safe... | NCT02597114

Studies
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

NCT02597114

Sponsor

ArmaGen, Inc

Status

Completed

Last Update Posted

Sep 25, 2018Actual

Enrollment

3Actual

Phase

Phase 1

Conditions

Mucopolysaccharidosis I

Interventions

AGT-181

Countries

Not provided

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

Sep 2018

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

Nov 2015Actual

Primary Completion Date

Mar 19, 2018Actual

Completion Date

Aug 2, 2018Actual

First Submitted Date

Nov 3, 2015

First Submission Date that Met QC Criteria

Nov 3, 2015

First Posted Date

Nov 5, 2015Estimated

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

Sep 21, 2018

Last Update Posted Date

Sep 25, 2018Actual

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

AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously. This study is an extension of a safety and dose ranging study to obtain long term safety and exposure data, as well as information on the biological activity of the investigational drug

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

3Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
AGT-181	Experimental	AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase) administered once weekly x 26 weeks at same dose level as subject received in core study	Drug: AGT-181

Interventions

Name	Type	Description	Arm Group Labels	Other Names
AGT-181	Drug	intravenous infusion over 3-4 hours	AGT-181	HIRMAb-IDUA

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of participants with adverse events as a measure of safety and tolerability		104 weeks (2 years)

Secondary Outcomes

Measure	Description	Time Frame
changes in urinary or plasma glycosaminoglycans (GAGs)		104 weeks (2 years)
change in liver size		104 weeks (2 years)
change in spleen size

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Must have completed clinical trial AGT-181-102
Voluntary written consent by patient or legally responsible representative
All women of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
Negative pregnancy test (females)

Exclusion Criteria:

Refusal to complete screening evaluations.
Any medical condition or other circumstances that may significantly interfere with study compliance
Patient is pregnant or lactating
Clinically significant spinal cord compression, evidence of cervical instability.
Subject developed clinically relevant hypersensitivity to AGT-181

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Patrice Rioux, MD PhD	ArmaGen, Inc	Study Director

Condition Browse Module

MeSH Terms

ID	Term
D008059	Mucopolysaccharidosis I

Ancestor Terms

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C546866	valanafusp alpha

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided