Mucopolysaccharidosis IPhase 1NCT02597114
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
A Phase 1 study testing AGT-181 in people with Mucopolysaccharidosis I, run by ArmaGen, Inc.
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 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mucopolysaccharidosis I
- What is being tested?
- AGT-181
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- No sites listed yet
- Study dates
- Nov 2015to 2 Aug 2018
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
About this study
Official title: An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102
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
- Conditions
- Mucopolysaccharidosis I
- Being tested
- AGT-181
- 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
Also called HIRMAb-IDUA, fusion protein of monoclonal antibody to human insulin receptor fused to alpha-L-iduronidase
intravenous infusion over 3-4 hours
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: AGT-181 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with adverse events as a measure of safety and tolerability
Measured: 104 weeks (2 years)
Secondary outcomes (4)
changes in urinary or plasma glycosaminoglycans (GAGs)
Measured: 104 weeks (2 years)
change in liver size
Measured: 104 weeks (2 years)
change in spleen size
Measured: 104 weeks (2 years)
change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid
Measured: 104 weeks (2 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2015 (estimated)
- Study startNov 2015
- Primary completion19 Mar 2018
- Study completion2 Aug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- ArmaGen, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Patrice Rioux, MD PhD, Study director, ArmaGen, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02597114
- Sponsor's protocol ID
- AGT-181-103
- Status last verified
- Sep 2018
- First submitted
- 3 Nov 2015
- Last updated
- 25 Sep 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102
- ClinicalTrials.gov ID
- NCT02597114
- Acronym / sponsor ID
- AGT-181-103
- Conditions
- Mucopolysaccharidosis I
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- ArmaGen, IncIndustry (pharma, biotech, devices)
- Study start
- Nov 2015 (actual)
- Primary completion
- 19 Mar 2018 (actual)
- Study completion
- 2 Aug 2018 (actual)
- First posted
- 5 Nov 2015 (estimated)
- 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 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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AGT-181 | Experimental | Drug: AGT-181AGT-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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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)
Key exclusion criteria (5 listed)
- 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
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-181Also: HIRMAb-IDUA, fusion protein of monoclonal antibody to human insulin receptor fused to alpha-L-iduronidaseintravenous infusion over 3-4 hours | Drug | AGT-181 |
Primary outcome
Number of participants with adverse events as a measure of safety and tolerability
Time frame: 104 weeks (2 years)
4 secondary outcomes
changes in urinary or plasma glycosaminoglycans (GAGs)
Time frame: 104 weeks (2 years)
change in liver size
Time frame: 104 weeks (2 years)
change in spleen size
Time frame: 104 weeks (2 years)
change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid
Time frame: 104 weeks (2 years)
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 25 Sep 2018.
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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 Rioux, MD PhD study director
ArmaGen, Inc
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AGT-181 in people with Mucopolysaccharidosis I, run by ArmaGen, Inc.
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 5 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 25 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
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 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.
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 participants with adverse events as a measure of safety and tolerability (time frame: 104 weeks (2 years)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 25 Sep 2018.