NCT03071341

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 is a long term safety and tolerability study of AGT-181 in patients with MPS I who completed the previous 26-week study, AGT-181-101. Information on the biological activity of the investigational drug will also be collected.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
19

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Oct 2016

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2016

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

February 27, 2017

Completed
7 days until next milestone

First Posted

Study publicly available on registry

March 6, 2017

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2018

Completed
Last Updated

March 15, 2023

Status Verified

March 1, 2023

Enrollment Period

1.3 years

First QC Date

February 27, 2017

Last Update Submit

March 13, 2023

Conditions

Mucopolysaccharidosis I

Keywords

MPS I; Hurler Syndrome

Outcome Measures

Primary Outcomes (1)

  • number of patients with adverse events as a measure of safety and tolerability

    Incidence and prevalence of adverse events

    24 months

Secondary Outcomes (5)

  • Total urinary glycosaminoglycans (GAGs)

    24 months

  • Urinary heparan sulfate and dermatan sulfate

    24 months

  • Plasma heparan sulfate and dermatan sulfate

    24 months

  • CSF heparan sulfate and dermatan sulfate

    24 months

  • liver and/or spleen volume

    24 months

Study Arms (1)

AGT-181

EXPERIMENTAL

Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein

Drug: AGT-181

Interventions

AGT-181DRUG

Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein

AGT-181

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • years of age or older (and less than 18)
  • Must have been previously enrolled in study AGT-181-101
  • Written consent and assent as required
  • Female patients must not be pregnant, willing to utilize appropriate birth control methods and undergo pregnancy testing during the study

You may not qualify if:

  • Refusal to complete all assessments
  • Pregnant or Lactating
  • Received investigational drug within 1 year prior to study enrollment
  • Medical condition or extenuating circumstance that, in the opinion of the investigator, may interfere with study compliance
  • Clinically significant spinal cord compression or evidence of cervical instability (i.e. expected to require intervention during study participation)
  • Has developed clinically relevant hypersensitivity/anaphylactoid reactions to AGT-181

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

HCPA - Hospital das Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

Location

Related Publications (1)

  • Giugliani R, Giugliani L, de Oliveira Poswar F, Donis KC, Corte AD, Schmidt M, Boado RJ, Nestrasil I, Nguyen C, Chen S, Pardridge WM. Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial. Orphanet J Rare Dis. 2018 Jul 5;13(1):110. doi: 10.1186/s13023-018-0849-8.

    PMID: 29976218RESULT

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

valanafusp alpha

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Patrice P Rioux, MD PhD

    ArmaGen, Inc

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Patients will be assigned to 1.0, 3.0 or 6.0 mg/kg (same dose taken during the previous study, AGT-181-101)
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 27, 2017

First Posted

March 6, 2017

Study Start

October 1, 2016

Primary Completion

February 1, 2018

Study Completion

February 1, 2018

Last Updated

March 15, 2023

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will not share

Locations

BR(1)