Mucopolysaccharidosis IPhase 1/2NCT03071341
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
A Phase 1/2 study testing AGT-181 in people with Mucopolysaccharidosis I, run by ArmaGen, Inc at 1 site in Brazil.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Mucopolysaccharidosis I
- What is being tested?
- AGT-181
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteBrazil
- Study dates
- Oct 2016to 1 Feb 2018
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 2 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Brazil1 site
HCPA - Hospital das Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903
About this study
Official title: An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181
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.
Read the detailed description
AGT-181-101E is a 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 be collected as well as longitudinal safety information.
- Conditions
- Mucopolysaccharidosis I
- Being tested
- AGT-181
- Keywords
- MPS I; Hurler Syndrome
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AGT-181Drug
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein
| Study group | Role | What they receive |
|---|---|---|
| AGT-181 | Experimental | Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein Receives: Drug: AGT-181 |
What is being measured
The primary outcome is the main question the study is designed to answer.
number of patients with adverse events as a measure of safety and tolerability
Incidence and prevalence of adverse events
Measured: 24 months
Secondary outcomes (5)
Total urinary glycosaminoglycans (GAGs)
Change in total urinary glycosaminoglycans (GAGs)
Measured: 24 months
Urinary heparan sulfate and dermatan sulfate
Change in urinary heparan sulfate and dermatan sulfate
Measured: 24 months
Plasma heparan sulfate and dermatan sulfate
Change in plasma heparan sulfate and dermatan sulfate
Measured: 24 months
CSF heparan sulfate and dermatan sulfate
Change in CSF heparan sulfate and dermatan sulfate
Measured: 24 months
liver and/or spleen volume
Change in liver and/or spleen volume measured by MRI
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2017
- Study startOct 2016
- Primary completion1 Feb 2018
- Study completion1 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mucopolysaccharidosis IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients will be assigned to 1.0, 3.0 or 6.0 mg/kg (same dose taken during the previous study, AGT-181-101)
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- ArmaGen, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Patrice P Rioux, MD PhD, Study director, ArmaGen, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03071341
- Sponsor's protocol ID
- AGT-181-101E
- Status last verified
- Mar 2023
- First submitted
- 27 Feb 2017
- Last updated
- 15 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 29976218
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181
- ClinicalTrials.gov ID
- NCT03071341
- Acronym / sponsor ID
- AGT-181-101E
- Conditions
- Mucopolysaccharidosis I
- Keywords
- MPS I; Hurler Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- ArmaGen, IncIndustry (pharma, biotech, devices)
- Study start
- Oct 2016 (actual)
- Primary completion
- 1 Feb 2018 (actual)
- Study completion
- 1 Feb 2018 (actual)
- First posted
- 6 Mar 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
AGT-181-101E is a 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 be collected as well as longitudinal safety information.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients will be assigned to 1.0, 3.0 or 6.0 mg/kg (same dose taken during the previous study, AGT-181-101)
| Arm | Type | Receives |
|---|---|---|
| AGT-181 | Experimental | Drug: AGT-181Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 2 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
Key exclusion criteria (6 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AGT-181Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein | Drug | AGT-181 |
Primary outcome
number of patients with adverse events as a measure of safety and tolerability
Time frame: 24 months
Incidence and prevalence of adverse events
5 secondary outcomes
Total urinary glycosaminoglycans (GAGs)
Time frame: 24 months
Urinary heparan sulfate and dermatan sulfate
Time frame: 24 months
Plasma heparan sulfate and dermatan sulfate
Time frame: 24 months
CSF heparan sulfate and dermatan sulfate
Time frame: 24 months
liver and/or spleen volume
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2023.
- Results publication: PubMed 29976218Giugliani 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Patrice P Rioux, MD PhD study director
ArmaGen, Inc
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AGT-181 in people with Mucopolysaccharidosis I, run by ArmaGen, Inc at 1 site in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
number of patients with adverse events as a measure of safety and tolerability (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03071341, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Mar 2023.