NGLY1 DeficiencyPhase 3NCT06199531
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency
A Phase 3 study testing GS-100 in people with NGLY1 Deficiency, run by Grace Science, LLC at 2 sites in United States.
Trial summary
A non-randomized, open-label, Phase 1/2/3 study of a single intracerebroventricular (ICV) administration of a gene replacement therapy (GS-100) in participants who are 2 to 18 years old with NGLY1 Deficiency. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- NGLY1 Deficiency
- What is being tested?
- GS-100
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesUnited States
- Study dates
- 13 Feb 2024to Mar 2031
- Phase
- Phase 3
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be 2 to 18 years of age, inclusive, at the time of signing the informed consent form (ICF)
- Patients with a documented diagnosis of NGLY1 Deficiency based on detection of biallelic variants in the NGLY1 gene via molecular genetic sequencing
- Elevated GNA levels may be considered alongside genetic sequencing data and other clinical data to assist with diagnosis confirmation
- Patients with two or more of the following clinical features typical of NGLY1 Deficiency:
- Global developmental delay and/or intellectual disability
- Hyperkinetic movement disorder
Show all 13Show fewer
- Transient elevation of transaminases
- (Hypo)alacrima
- Peripheral neuropathy
- For patients with epilepsy who require anti-seizure medications for seizure control: must be on a stable regimen for 28 days prior to enrollment
- Patients willing and capable per investigator opinion to comply with study procedures and requirements
- Females of childbearing potential must have a negative serum pregnancy test at screening and must agree to use an acceptable method of highly effective contraception from screening through the end of the study
- Patients or parent(s)/guardian(s) must be willing and able to provide written consent after the nature of the study has been explained and prior to performance of any research-related procedures
Who cannot join
- For Phase 1/2 only: Patients at Level 5 of both the Gross Motor Function Classification System Expanded and Revised (GMFCS E\&R) and the Communication Function Classification System (CFCS) scales as assessed by the investigator
- Contraindication to use of corticosteroids or history of a condition that could worsen with corticosteroid therapy, as assessed, and determined by the investigator
- Signs / symptoms of increased intracranial pressure (ICP), history of space occupying lesion, or ventricular shunt that would preclude ICV procedures or safety assessments
- If clinical signs or symptoms of increased ICP are present (such as headache, vomiting, altered mental status), an ophthalmology examination will be performed to assess for papilledema and/or venous pulsations
- Any comorbid medical or behavioral condition that, in the opinion of the investigator, may adversely affect the safety and well-being of the participant during the study, interfere with completion of the study procedures or follow-up, or compound interpretation of the study results
- Vital signs outside age-based normative values:
Show all 27Show fewer
- Blood pressure: values > 99th percentile as cited in the National Heart, Lung and Blood Institute (NHLBI) guidelines for blood pressure levels based on subject's age, height and sex (nhlbi.nih.gov/files/docs/guidelines/child_tbl.pdf)
- Temperature: evidence of fever such as body temperature (e.g., orally measured) of 38.0°C (100.3°F)
- Respiratory rate in breaths per minute: toddler (1-3 years old): 24-40; preschooler (4-5 years old): 22-34 breaths per minute; school-aged child (6-12 years old): 18-30 breaths per minute; adolescence (13-18 years old): 12-16.
- Oxygen saturation on room air < 92%
- Any condition that in the opinion of the investigator or the study medical monitor would prevent the patient from fully complying with the requirements of the study (including the corticosteroid treatment outline in the protocol) and/or would impact or interfere with the evaluation and interpretation of patient safety or efficacy results
- Known allergy or hypersensitivity to the GS-100 investigational product formulation
- Prior treatment with gene therapy
- Treatment with any investigational product (IP) within 30 days or 5 half-lives of the IP, whichever is longer, prior to screening period. For patients who have received a prior investigational product, all ongoing AEs experienced while receiving the investigational product must have been resolved prior to screening for this study
- Concurrent enrollment in another clinical study, unless it is an observational (non-interventional) clinical study that does not interfere with the requirements of the current protocol and does not have the potential to impact the evaluation of safety and efficacy of GS-100
- Coagulation dysfunction at screening as defined by the following:
- INR: ≥1.4 x Upper Limit of Normal (ULN)
- Any current infection with hepatitis B virus (HBV) as evidenced by as evidenced by positive HBV surface antigen (HBsAg), and/or HBV core antibody (HBcAb) at screening. Isolated HBsAb positivity for HBV vaccination in conjunction with negative confirmatory HBV DNA testing at screening is not exclusionary
- Any prior or current infection with hepatitis C virus (HCV) as evidenced by positive HCV antibody testing and confirmed by positive polymerase chain reaction (PCR) RNA testing at screening
- Any of the following abnormal laboratory values:
- Hemoglobin level: < 9 g/dL
- Absolute neutrophil count: < 1000 cells/microliter
- Platelet count: < 100,000/mm3
- Creatinine: > 1.25 x ULN
- Have a major surgery planned during the screening period through 52 weeks following GS-100 infusion, including major dental procedures (e.g., wisdom tooth extraction)
- Pregnant or breastfeeding female patient
- Patients that demonstrate elevated serum adrenocorticotropic hormone (ACTH) 1.5 times the upper limit of normal for the reference range must be referred for consultation with a pediatric endocrinologist to rule out primary adrenal insufficiency prior to being enrolled into the study. Patients may re-screen for participation in the study after medical consultation and / or possible treatment has been initiated to address any adrenal insufficiency
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Texas Children's Hospital (Baylor College of Medicine)
Houston, Texas, 77030
Oakland Children's Hospital (UCSF Benioff)
Oakland, California, 94609
About this study
Official title: A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients With NGLY1 Deficiency
A non-randomized, open-label, Phase 1/2/3 study of a single intracerebroventricular (ICV) administration of a gene replacement therapy (GS-100) in participants who are 2 to 18 years old with NGLY1 Deficiency.
Read the detailed description
This study is a first in human (FIH) open-label study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding N-glycanase 1 (NGLY1) in subjects ages 2-18 years old with NGLY1 Deficiency. The study treatment is delivered via intracerebroventricular (ICV) injection.
Phase 1/2 is the open-label dose finding part of the study designed to evaluate the safety and preliminary efficacy of GS-100 at 4 different dose levels and to select a safe and efficacious dose for the Phase 3 part of the study. Enrollment of 7 participants in the Phase 1/2 part of the study is complete.
Phase 3 is evaluating the efficacy and safety of GS-100 at the Selected Dose determined in the Phase 1/2 part of the study (Mid dose: 1e15 for 6-18-year-olds, 8.7e14 for 2-5-year-olds). Enrollment of 3 participants in the Phase 3 part of the study is complete.
- Conditions
- NGLY1 Deficiency
- Being tested
- GS-100
- Keywords
- Gene Therapy, Intervention study, Infusions, Intraventricular, Enzyme Replacement Therapy
- Medical subject headings
- NGLY1 deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GS-100Genetic
A single intracerebroventricular (ICV) dose of GS-100
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | GS-100 Low Dose Level: 4e14 total vector genomes (vg) for 6-18-year-olds (fully enrolled, 2 participants) Receives: Genetic: GS-100 |
| Cohort 2 | Experimental | GS-100 Mid Dose Level: 1e15 total vector genomes (vg) for 13-18-year-olds, 8.7e14 total vg for 2-5-year-olds (fully enrolled, 2 participants) Receives: Genetic: GS-100 |
| Cohort 3 | Experimental | GS-100 High Dose Level: 3e15 total vector genomes (vg) for 6-18-year-olds, 2.6e15 vg for 2-5-year-olds (fully enrolled, 2 participants) Receives: Genetic: GS-100 |
| Cohort 4 | Experimental | GS-100 Intermediate Dose Level: 2e15 total vector genomes (vg) for 6-18-year-olds, 1.75e15 total vg for 2-5-year-olds (fully enrolled, 1 participant) Receives: Genetic: GS-100 |
| Pivotal Cohort | Experimental | GS-100 Selected Dose Level: 1e15 total vector genomes (vg) for 6-18-year-olds, 8.7e14 total vg for 2-5-year-olds (fully enrolled, 3 participants) Receives: Genetic: GS-100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1/2 (Dose Finding): Safety and Tolerability of GS-100
Incidence of adverse events (AEs) and serious AEs (SAEs)
Measured: Baseline through Week 52
Phase 3 (Pivotal): Efficacy of GS-100 at the Selected Dose
Improvement in one or more domains of the 88-item Gross Motor Function Measure (GMFM-88) from Baseline to Week 52
Measured: Baseline through Week 52
Secondary outcomes (11)
Individual domains of the Bayley Scales of Infant and Toddler Development 4th Ed (BSID-4) for the Cognitive, Language, and Motor scales
Change in total Raw Scores and Growth Score Values (GSVs) will be analyzed. Raw Scores range 0-162; GSVs range 428-559; a higher score reflects a higher level of skill.
Measured: Baseline through Week 52
Clinical Global Impression of Change (CGI-C)
Change in value relative to Baseline; range of values is 1-7, with 1 being marked improvement
Measured: Baseline through Week 52
Clinical Global Impression of Severity (CGI-S)
Change in value; range of values is 1-7, with 1 being normal/no impairment
Measured: Baseline through Week 52
Videotaped movement disorder assessments
Change in movement disorder as captured by videotaped assessment
Measured: Baseline through Week 52
Sitting/standing assessment
Change in ability to sit, come to sitting, stand, come to standing, and walk
Measured: Baseline through Week 52
Failure to thrive
Change in weight (Z-score)
Measured: Baseline through Week 52
Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition (WPPSI-IV)
Change in standard scores; standard scores are based on a mean of 100 and a standard deviation of 15; a higher score reflects a higher level of skill
Measured: Baseline through Week 52
Caregiver Global Impression of Change (CaGI-C)
Change in value relative to Baseline; range of values is 1-7, with 1 being marked improvement
Measured: Baseline through Week 52
Caregiver Global Impression of Severity (CaGI-S)
Change in value; range from 1-7, with 1 being normal/no impairment
Measured: Baseline through Week 52
Nerve conduction velocity (NCV)
Change in peripheral nerve function
Measured: Baseline through Week 52
Seizure frequency in subjects with seizures
Change in seizure frequency as measured by seizure diaries
Measured: Baseline through Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2024
- Study start13 Feb 2024
- Primary completionMar 2027 (estimated)
- Study completionMar 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for NGLY1 DeficiencyStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- The study treatment will be delivered via intracerebroventricular (ICV) infusion.
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grace Science, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06199531
- Sponsor's protocol ID
- GS-100-A301
- Status last verified
- Apr 2026
- First submitted
- 21 Nov 2023
- Last updated
- 4 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Zhu L, Cook JW, Newton A, Dwight SS, Beahm B, Wilsey M, Mueller WF, Schweighardt B. Preclinical pharmacology and safety studies to support an AAV9 NGLY1 gene therapy clinical trial for the treatment of NGLY1 deficiency. Mol Ther Methods Clin Dev. 2025 Jun 25;33(3):101524. doi: 10.1016/j.omtm.2025.101524. eCollection 2025 Sep 11.
PubMed 40687377Tong S, Ventola P, Frater CH, Klotz J, Phillips JM, Muppidi S, Dwight SS, Mueller WF, Beahm BJ, Wilsey M, Lee KJ. NGLY1 deficiency: a prospective natural history study. Hum Mol Genet. 2023 Sep 5;32(18):2787-2796. doi: 10.1093/hmg/ddad106.
PubMed 37379343Stanclift CR, Dwight SS, Lee K, Eijkenboom QL, Wilsey M, Wilsey K, Kobayashi ES, Tong S, Bainbridge MN. NGLY1 deficiency: estimated incidence, clinical features, and genotypic spectrum from the NGLY1 Registry. Orphanet J Rare Dis. 2022 Dec 17;17(1):440. doi: 10.1186/s13023-022-02592-3.
PubMed 36528660Levy RJ, Frater CH, Gallentine WB, Phillips JM, Ruzhnikov MR. Delineating the epilepsy phenotype of NGLY1 deficiency. J Inherit Metab Dis. 2022 May;45(3):571-583. doi: 10.1002/jimd.12494. Epub 2022 Mar 11.
PubMed 35243670
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients With NGLY1 Deficiency
- ClinicalTrials.gov ID
- NCT06199531
- Acronym / sponsor ID
- GS-100-A301
- Conditions
- NGLY1 Deficiency
- Keywords
- Gene Therapy, Intervention study, Infusions, Intraventricular, Enzyme Replacement Therapy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Grace Science, LLCIndustry (pharma, biotech, devices)
- Study start
- 13 Feb 2024 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2031 (estimated)
- First posted
- 10 Jan 2024 (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.
A non-randomized, open-label, Phase 1/2/3 study of a single intracerebroventricular (ICV) administration of a gene replacement therapy (GS-100) in participants who are 2 to 18 years old with NGLY1 Deficiency.
Detailed description
This study is a first in human (FIH) open-label study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding N-glycanase 1 (NGLY1) in subjects ages 2-18 years old with NGLY1 Deficiency. The study treatment is delivered via intracerebroventricular (ICV) injection.
Phase 1/2 is the open-label dose finding part of the study designed to evaluate the safety and preliminary efficacy of GS-100 at 4 different dose levels and to select a safe and efficacious dose for the Phase 3 part of the study. Enrollment of 7 participants in the Phase 1/2 part of the study is complete.
Phase 3 is evaluating the efficacy and safety of GS-100 at the Selected Dose determined in the Phase 1/2 part of the study (Mid dose: 1e15 for 6-18-year-olds, 8.7e14 for 2-5-year-olds). Enrollment of 3 participants in the Phase 3 part of the study is complete.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
- Model description
- The study treatment will be delivered via intracerebroventricular (ICV) infusion.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: GS-100GS-100 Low Dose Level: 4e14 total vector genomes (vg) for 6-18-year-olds (fully enrolled, 2 participants) |
| Cohort 2 | Experimental | Genetic: GS-100GS-100 Mid Dose Level: 1e15 total vector genomes (vg) for 13-18-year-olds, 8.7e14 total vg for 2-5-year-olds (fully enrolled, 2 participants) |
| Cohort 3 | Experimental | Genetic: GS-100GS-100 High Dose Level: 3e15 total vector genomes (vg) for 6-18-year-olds, 2.6e15 vg for 2-5-year-olds (fully enrolled, 2 participants) |
| Cohort 4 | Experimental | Genetic: GS-100GS-100 Intermediate Dose Level: 2e15 total vector genomes (vg) for 6-18-year-olds, 1.75e15 total vg for 2-5-year-olds (fully enrolled, 1 participant) |
| Pivotal Cohort | Experimental | Genetic: GS-100GS-100 Selected Dose Level: 1e15 total vector genomes (vg) for 6-18-year-olds, 8.7e14 total vg for 2-5-year-olds (fully enrolled, 3 participants) |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Patients must be 2 to 18 years of age, inclusive, at the time of signing the informed consent form (ICF)
- Patients with a documented diagnosis of NGLY1 Deficiency based on detection of biallelic variants in the NGLY1 gene via molecular genetic sequencing
- Elevated GNA levels may be considered alongside genetic sequencing data and other clinical data to assist with diagnosis confirmation
- Patients with two or more of the following clinical features typical of NGLY1 Deficiency:
- For patients with epilepsy who require anti-seizure medications for seizure control: must be on a stable regimen for 28 days prior to enrollment
Key exclusion criteria (27 listed)
- For Phase 1/2 only: Patients at Level 5 of both the Gross Motor Function Classification System Expanded and Revised (GMFCS E\&R) and the Communication Function Classification System (CFCS) scales as assessed by the investigator
- Contraindication to use of corticosteroids or history of a condition that could worsen with corticosteroid therapy, as assessed, and determined by the investigator
- Signs / symptoms of increased intracranial pressure (ICP), history of space occupying lesion, or ventricular shunt that would preclude ICV procedures or safety assessments
- Any comorbid medical or behavioral condition that, in the opinion of the investigator, may adversely affect the safety and well-being of the participant during the study, interfere with completion of the study procedures or follow-up, or compound interpretation of the study results
- Vital signs outside age-based normative values:
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 |
|---|---|---|
| GS-100A single intracerebroventricular (ICV) dose of GS-100 | Genetic | Cohort 1, Cohort 2, Cohort 3, Cohort 4, Pivotal Cohort |
Primary outcome
Phase 1/2 (Dose Finding): Safety and Tolerability of GS-100
Time frame: Baseline through Week 52
Incidence of adverse events (AEs) and serious AEs (SAEs)
Phase 3 (Pivotal): Efficacy of GS-100 at the Selected Dose
Time frame: Baseline through Week 52
Improvement in one or more domains of the 88-item Gross Motor Function Measure (GMFM-88) from Baseline to Week 52
11 secondary outcomes
Individual domains of the Bayley Scales of Infant and Toddler Development 4th Ed (BSID-4) for the Cognitive, Language, and Motor scales
Time frame: Baseline through Week 52
Clinical Global Impression of Change (CGI-C)
Time frame: Baseline through Week 52
Clinical Global Impression of Severity (CGI-S)
Time frame: Baseline through Week 52
Videotaped movement disorder assessments
Time frame: Baseline through Week 52
Sitting/standing assessment
Time frame: Baseline through Week 52
Failure to thrive
Time frame: Baseline through Week 52
Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition (WPPSI-IV)
Time frame: Baseline through Week 52
Caregiver Global Impression of Change (CaGI-C)
Time frame: Baseline through Week 52
Caregiver Global Impression of Severity (CaGI-S)
Time frame: Baseline through Week 52
Nerve conduction velocity (NCV)
Time frame: Baseline through Week 52
Seizure frequency in subjects with seizures
Time frame: Baseline through Week 52
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 4 May 2026.
- Background: PubMed 40687377Zhu L, Cook JW, Newton A, Dwight SS, Beahm B, Wilsey M, Mueller WF, Schweighardt B. Preclinical pharmacology and safety studies to support an AAV9 NGLY1 gene therapy clinical trial for the treatment of NGLY1 deficiency. Mol Ther Methods Clin Dev. 2025 Jun 25;33(3):101524. doi: 10.1016/j.omtm.2025.101524. eCollection 2025 Sep 11.
- Background: PubMed 37379343Tong S, Ventola P, Frater CH, Klotz J, Phillips JM, Muppidi S, Dwight SS, Mueller WF, Beahm BJ, Wilsey M, Lee KJ. NGLY1 deficiency: a prospective natural history study. Hum Mol Genet. 2023 Sep 5;32(18):2787-2796. doi: 10.1093/hmg/ddad106.
- Background: PubMed 36528660Stanclift CR, Dwight SS, Lee K, Eijkenboom QL, Wilsey M, Wilsey K, Kobayashi ES, Tong S, Bainbridge MN. NGLY1 deficiency: estimated incidence, clinical features, and genotypic spectrum from the NGLY1 Registry. Orphanet J Rare Dis. 2022 Dec 17;17(1):440. doi: 10.1186/s13023-022-02592-3.
- Background: PubMed 35243670Levy RJ, Frater CH, Gallentine WB, Phillips JM, Ruzhnikov MR. Delineating the epilepsy phenotype of NGLY1 deficiency. J Inherit Metab Dis. 2022 May;45(3):571-583. doi: 10.1002/jimd.12494. Epub 2022 Mar 11.
- Individual participant dataSharing planned: No
Related trials for NGLY1 Deficiency
Active, not recruitingPhase 2
A Study of GlcNAc on Tear Production in NGLY1-CDDG
NGLY1 Deficiency
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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing GS-100 in people with NGLY1 Deficiency, run by Grace Science, LLC at 2 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 13 inclusion and 27 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: under way, not looking for participants, as of the sponsor's last update on 4 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
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?
Phase 1/2 (Dose Finding): Safety and Tolerability of GS-100 (time frame: Baseline through Week 52). The study lists 2 primary outcomes in all. 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 NCT06199531, which also lists 4 linked publications.
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 4 May 2026.