Amyotrophic Lateral SclerosisPhase 1ARMORNCT07290062
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
A Phase 1 study testing INS1202 in people with Amyotrophic Lateral Sclerosis, run by Insmed Gene Therapy LLC at 7 sites in United States.
Trial summary
The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to <80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation. Read more about this study
- Who is it for?
- Ages 18 to 79 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- INS1202
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 7 sitesUnited States
- Study dates
- 9 Jan 2026to 31 Mar 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 to 79 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m^2).
- Participant with symptomatic ALS as diagnosed by Gold Coast diagnostic criteria.
- Sporadic ALS cohorts: Negative testing for known monogenic mutations associated with familial ALS.
- SOD1-ALS (Cohorts 2 and 3 only): Confirmed pathogenic SOD1 mutation, with negative testing for other genetic mutations associated with familial ALS.
- Any polymorphism or mutation in the coding region will require additional review by the Sponsor to determine compatibility with the study intervention.
- Baseline ALSFRS-R ≥ 24.
- ALS disease duration ≤ 42 months.
Who cannot join
- Previous treatment for ALS with cellular or gene therapies.
- Any investigational medication or treatment (for ALS or other condition).
- Note: Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
7 sites in 1 country. Contact the study team or a site near you to ask about taking part.
7 sites in 1 country
United States7 sites, 7 recruiting
- Recruiting
USA003
Baltimore, Maryland, 21205
emosmil1@jhmi.edu
- Recruiting
USA001
Columbia, Missouri, 65211
heathermchatton@health.missouri.edu
- Recruiting
USA007
Columbus, Ohio, 43221
ALSResearch@osumc.edu
- Recruiting
USA004
La Jolla, California, 92037
rprevite@health.ucsd.edu
- Recruiting
USA002
Palo Alto, California, 94304
NEUROMUSCULARRESEARCH@STANFORD.EDU
- Recruiting
USA006
Philadelphia, Pennsylvania, 19107
Alexander.McGeary@jefferson.edu
- Recruiting
USA009
Worcester, Massachusetts, 01655
danielle.kokoski@umassmed.edu
About this study
Official title: A Phase 1, Multicenter, Open-label, Dose-Finding Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection of INS1202 in Patients With Amyotrophic Lateral Sclerosis
The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to <80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- INS1202
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INS1202Genetic
Suspension for injection.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1. Receives: Genetic: INS1202 |
| Cohort 2 | Experimental | Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1. Receives: Genetic: INS1202 |
| Cohort 3 | Experimental | Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1. Receives: Genetic: INS1202 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Measured: Up to 48 weeks
Secondary outcomes (2)
Recommended Phase 2 Dose (RP2D) of INS1202-101
Measured: Up to 48 weeks
Viral Vector Shedding Following the IT Administration of INS1202 by Droplet Digital Polymerase Chain Reaction (ddPCR)
Measured: Baseline, Week 1, Week 2, Week 4, Week 16 and Week 32
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2025
- Study start9 Jan 2026
- Primary completion31 Mar 2030 (estimated)
- Study completion31 Mar 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 23 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Insmed Gene Therapy LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07290062
- Sponsor's protocol ID
- INS1202-101
- Acronym
- ARMOR
- Status last verified
- Sep 2026
- First submitted
- 4 Dec 2025
- Last updated
- 23 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Multicenter, Open-label, Dose-Finding Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection of INS1202 in Patients With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT07290062
- Acronym / sponsor ID
- ARMOR / INS1202-101
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 23 participants (estimated)
- Lead sponsor
- Insmed Gene Therapy LLCIndustry (pharma, biotech, devices)
- Study start
- 9 Jan 2026 (actual)
- Primary completion
- 31 Mar 2030 (estimated)
- Study completion
- 31 Mar 2030 (estimated)
- First posted
- 17 Dec 2025 (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.
The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to <80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: INS1202Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1. |
| Cohort 2 | Experimental | Genetic: INS1202Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1. |
| Cohort 3 | Experimental | Genetic: INS1202Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1. |
Who may qualify
- Ages
- 18 to 79 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m^2).
- Participant with symptomatic ALS as diagnosed by Gold Coast diagnostic criteria.
- Sporadic ALS cohorts: Negative testing for known monogenic mutations associated with familial ALS.
- SOD1-ALS (Cohorts 2 and 3 only): Confirmed pathogenic SOD1 mutation, with negative testing for other genetic mutations associated with familial ALS.
- Any polymorphism or mutation in the coding region will require additional review by the Sponsor to determine compatibility with the study intervention.
Key exclusion criteria (3 listed)
- Previous treatment for ALS with cellular or gene therapies.
- Any investigational medication or treatment (for ALS or other condition).
- Note: Other protocol-defined inclusion/exclusion criteria may apply.
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 |
|---|---|---|
| INS1202Suspension for injection. | Genetic | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to 48 weeks
2 secondary outcomes
Recommended Phase 2 Dose (RP2D) of INS1202-101
Time frame: Up to 48 weeks
Viral Vector Shedding Following the IT Administration of INS1202 by Droplet Digital Polymerase Chain Reaction (ddPCR)
Time frame: Baseline, Week 1, Week 2, Week 4, Week 16 and Week 32
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2026.
- Click here to access a lay person friendly information about this clinical trial.Link provided by the sponsor.
- 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.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing INS1202 in people with Amyotrophic Lateral Sclerosis, run by Insmed Gene Therapy LLC at 7 sites in United States.
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 7 inclusion and 3 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: looking for participants now, as of the sponsor's last update on 23 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. 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 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Insmed Medical Information (1-844-446-7633, medicalinformation@insmed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) (time frame: Up to 48 weeks). 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 23 Sep 2026.