Tuberous Sclerosis ComplexPhase 3NCT05604170
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
A Phase 3 study testing Ganaxolone in people with Tuberous Sclerosis Complex, run by Marinus Pharmaceuticals at 54 sites in 10 countries.
Trial summary
This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001 Read more about this study
- Who is it for?
- Ages 1 year to 65 years
- Conditions the study is for
- Tuberous Sclerosis Complex
- What is being tested?
- Ganaxolone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 117 participants
- Where
- 54 sitesin 10 countries
- Study dates
- 16 May 2022to 2 Apr 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 year to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completion of Study 1042-TSC-3001 or participants who continue to meet study requirements in Study 1042-TSC-2001.
- Participant/parent(s)/LAR(s) willing and able to give written informed consent/assent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures. If the participant is not qualified or able to provide written informed consent based on age, developmental stage, intellectual capacity, or other factors, parent(s)/LAR(s) must provide assent for study participation, if appropriate.
- Parent(s)/caregiver(s) is (are) willing and able to maintain an accurate and complete daily seizure diary for the duration of the study.
- Willing and able to take Investigational product (IP) (suspension) as directed with food TID.
- Women of childbearing potential (WOCBP) must be using a medically acceptable method of birth control and have a negative quantitative serum beta-human chorionic growth hormone (β-HCG) test collected at the initial visit. Childbearing potential is defined as a female who is biologically capable of becoming pregnant. Medically acceptable methods of birth control include intrauterine devices (that have been in place for at least 1 month prior to the screening visit), hormonal contraceptives (eg, combined oral contraceptives, patch, vaginal ring, injectables, and implants), and surgical sterilization (such as oophorectomy or tubal ligation). When used consistently and correctly, "double-barrier" methods of contraception can be used as an effective alternative to highly effective contraception methods. Contraceptive measures such as Plan B™, sold for emergency use after unprotected sex, are not acceptable methods for routine use
- Male participants must agree to use highly effective contraceptive methods during the study and for 30 days after the last dose of IP. Highly effective methods of contraception include surgical sterilization (such as a vasectomy) and adequate "double-barrier" methods.
Who cannot join
- Pregnant or breastfeeding.
- An active Central nervous system (CNS) infection, demyelinating disease, or degenerative neurological disease.
- History of psychogenic nonepileptic seizures.
- Any disease or condition (other than TSC) at the initial visit that could compromise the hematologic, cardiovascular (including any cardiac conduction defect), pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the IP, or would place the participant at increased risk or interfere with the assessment of safety/efficacy. This may include any illness in the past 4 weeks which in the opinion of the investigator may affect seizure frequency.
- Unwillingness to avoid excessive alcohol use or cannabis use throughout the study.
- Have active suicidal plan/intent or have had active suicidal thoughts in the past 6 months or a suicide attempt in the past 6 months.
- Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.
- Exposed to any other investigational drug (except for GNX in Study 1042-TSC-2001 or Study 1042-TSC-3001) or investigational device within 30 days or fewer than 5 half-lives prior to Visit 1 (first visit of the OLE). For therapies in which half-life cannot be readily established, the Sponsor's medical monitor should be consulted.
Trial locations and contacts
54 sites in 10 countries.
54 sites in 10 countries
United States20 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Child Neurology Consultants of Austin (CNCA)
Austin, Texas, 78757
Boston Children's Hospital, Harvard Medical School
Boston, Massachusetts, 02115
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Atrium Health/Levine Children's Hospital
Charlotte, North Carolina, 28207
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Texas Southwestern Medical Center
Dallas, Texas, 75207
Duke University Medical Center
Durham, North Carolina, 27712
McGovern Medical School at the University of Texas Health Science Center
Houston, Texas, 77030
Children's Mercy Hospital
Kansas City, Missouri, 64108
Arkansas Children's Research Institute
Little Rock, Arkansas, 72202
UCLA Mattel Children's Hospital, TSC Center
Los Angeles, California, 90095
Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
Children's Hospital of Orange County
Orange, California, 92868
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
University of Rochester Medical Center
Rochester, New York, 14642
University of Utah Health Care-Pediatric Neurology
Salt Lake City, Utah, 84108
Rady Children's Hospital - San Diego
San Diego, California, 92123
Seattle Children's Hospital
Seattle, Washington, 98105
Nemours Children's Hospital - Delaware Valley
Wilmington, Delaware, 19803
Germany6 sites
Epilepsie-Zentrum Bethel - Krankenhaus Mara
Bielefeld, 33617
University Hospital Bonn
Bonn, 53127
ZNN - Epilepsiezentrum Frankfurt am Main
Frankfurt, 60528
Universitäts Krankenhaus Freiburg
Freiburg im Breisgau, 79106
Gemeinschaftskrankenhaus Herdecke
Herdecke, 58313
Epilepsiezentrum Kleinwachau gGmbH
Radeberg, 1454
Spain6 sites
Hospital de la Santa Creu i Sant Pau
Barcelona, 8025
Hospital Sant Joan de Déu
Barcelona, 8950
Hospital Infantil Universitario Niño Jesús
Madrid, 28009
Hospital Ruber International
Madrid, 28034
Hospital Regional Universitario de Málaga
Málaga, 29010
Hospital Universitario y Politécnico La Fe
Valencia, 46026
Canada5 sites
Hôtel Dieu de Montréal - CHUM
Montreal, H2X 0C2
CHU Sainte-Justine
Montreal, H3T 1C5
The Hospital for Sick Children
Toronto, M5G 1X8
Toronto Western Hospital
Toronto, M5T 2S8
BC Children's Hospital
Vancouver, V6H 3V4
China5 sites
Peking University First Hospital
Beijing, 100034
Beijing Children Hospital, Capital Medical University
Beijing, 100045
Chinese PLA General Hospital
Beijing, 100853
The Affiliated Hospital of Guizhou Medical University
Beijing, 550004
First Hospital of Jilin University
Jilin City, 130021
Australia4 sites
Austin Health
Heidelberg, VIC 3084
Royal Brisbane and Women's Hospital
Herston, QLD 4029
Alfred Health
Melbourne, VIC 3004
Royal Melbourne Hospital
Parkville, VIC 3050
France3 sites
University Hospital of Lyon
Bron, 69229
Hôpital Sud
Rennes, 35000
University of Strasbourg
Strasbourg, 67084
Italy2 sites
Pediatric Neurology and Muscular Diseases Unit - University of Genoa
Genova, 16147
Policlinico Umberto I
Rome, 00185
United Kingdom2 sites
Bristol Royal Hospital for Children
Bristol, BS2 8AE
Sheffield Children's Hospital
Sheffield, S10 2TH
About this study
Official title: A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC OLE)
This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001
- Conditions
- Tuberous Sclerosis Complex
- Being tested
- Ganaxolone
- Keywords
- Tuberous Sclerosis Complex-Related Epilepsy, Ganaxolone, Adjunctive
- Medical subject headings
- Tuberous Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GanaxoloneDrug
GNX will be administered.
| Study group | Role | What they receive |
|---|---|---|
| Ganaxolone (GNX) oral suspension, 3 times a day (TID) | Experimental | Not described Receives: Drug: Ganaxolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Withdrawals and Dose-Reductions Due to AEs
An adverse event (AE) was any untoward medical occurrence in a clinical study patient, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event was any untoward medical occurrence that, at any dose, results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or any other event that requires scientific judgment.
Measured: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Vital Parameters
Vital parameters including heart rate, respiration rate, blood pressure, and body temperature were measured in seated position for at least 5 minutes.
Measured: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Physical Examinations
Physical examinations included a full physical evaluation of body systems including: General appearance, head (eyes, ears, nose, and throat), cardiovascular, respiratory, gastrointestinal, genitourinary, musculoskeletal, endocrine/metabolic, hematologic/lymphatic, skin, and other systems as appropriate.
Measured: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Neurological Examinations
Neurological examinations included an evaluation of: Cranial nerves, motor exam, sensory exam, reflexes, coordination/cerebellar
Measured: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Developmental Examinations
Developmental examinations (applicable only to pediatric participants 1 to 17 years of age, inclusive) included an evaluation of speech/language, motor skills, and social skills
Measured: Up to 150 Weeks
Number of Participants With Clinically Significant 12-lead Electrocardiogram (ECG) Findings
Twelve-lead ECGs were performed by the physician using an ECG machine that automatically calculates the heart rate and measure PR, QRS, QT and QTc intervals.
Measured: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Hematology Parameters
Blood samples were collected for the analysis of hematology parameters: hemoglobin, hematocrit, erythrocytes, thrombocytes (platelet count). Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
Measured: Week 1 through Week 150
Number of Participants With Clinically Significant Changes in Chemistry Parameters
Blood samples were collected for the analysis of chemistry parameters: blood urea nitrogen (BUN), potassium, alanine aminotransferase (ALT), alkaline phosphatase (ALP)/serum glutamic pyruvic transaminase (SGPT), total bilirubin, creatinine, sodium, aspartate aminotransferase (AST)/serum glutamic oxaloacetic (SGOT), total protein, fasting blood glucose, calcium, carbon dioxide, estimated glomerular filtration rate (eGFR), and chloride
Measured: Up to 150 Weeks
Number of Participants With Clinically Significant Changes in Urinalysis
Urine samples were collected for the analysis of urinalysis parameters: Specific gravity, color, clarity, pH, glucose, protein, blood, nitrite, protein, ketones, bilirubin, urobilinogen, nitrite, leukocyte esterase by dipstick, and microscopic examination (if blood or protein was abnormal)
Measured: Up to 150 Weeks
Number of Participants (Age Greater Than 17 Years) With Abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
The C-SSRS is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 5 items for suicidal behavior (1. Preparatory acts or behavior, 2. Aborted attempt, 3. Interrupted attempt, 4. Actual attempt, 5. Completed suicide) will be reported. The score ranges from 1 to 5 and higher scores indicate worse symptoms.
Measured: Up to Week 52
Secondary outcomes (1)
Percent Change From Baseline in 28-day Seizure Frequency During Open Label Extension
Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28. Percent change from baseline in 28-day frequency was calculated for each participant by subtracting Baseline 28-day seizure frequency from post-Baseline 28-day seizure frequency, whole divided by Baseline 28-day seizure frequency and multiplied by 100.
Measured: Baseline (Day 1), Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2022
- Study start16 May 2022
- Primary completion2 Apr 2025
- Study completion2 Apr 2025
Why it stopped: Sponsor's decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Tuberous Sclerosis ComplexStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 117 participants (actual)
Sponsor and investigators
- Lead sponsor
- Marinus Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05604170
- Sponsor's protocol ID
- 1042-TSC-3002
- Status last verified
- Sep 2025
- First submitted
- 5 Oct 2022
- Last updated
- 1 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- 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 1 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC OLE)
- ClinicalTrials.gov ID
- NCT05604170
- Acronym / sponsor ID
- 1042-TSC-3002
- Conditions
- Tuberous Sclerosis Complex
- Keywords
- Tuberous Sclerosis Complex-Related Epilepsy, Ganaxolone, Adjunctive
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor's decision
- Enrolment
- 117 participants (actual)
- Lead sponsor
- Marinus PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 16 May 2022 (actual)
- Primary completion
- 2 Apr 2025 (actual)
- Study completion
- 2 Apr 2025 (actual)
- First posted
- 3 Nov 2022 (actual)
- Results
- Posted 1 Oct 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ganaxolone (GNX) oral suspension, 3 times a day (TID) | Experimental | Drug: Ganaxolone |
Who may qualify
- Ages
- 1 year to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Completion of Study 1042-TSC-3001 or participants who continue to meet study requirements in Study 1042-TSC-2001.
- Participant/parent(s)/LAR(s) willing and able to give written informed consent/assent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures. If the participant is not qualified or able to provide written informed consent based on age, developmental stage, intellectual capacity, or other factors, parent(s)/LAR(s) must provide assent for study participation, if appropriate.
- Parent(s)/caregiver(s) is (are) willing and able to maintain an accurate and complete daily seizure diary for the duration of the study.
- Willing and able to take Investigational product (IP) (suspension) as directed with food TID.
- Women of childbearing potential (WOCBP) must be using a medically acceptable method of birth control and have a negative quantitative serum beta-human chorionic growth hormone (β-HCG) test collected at the initial visit. Childbearing potential is defined as a female who is biologically capable of becoming pregnant. Medically acceptable methods of birth control include intrauterine devices (that have been in place for at least 1 month prior to the screening visit), hormonal contraceptives (eg, combined oral contraceptives, patch, vaginal ring, injectables, and implants), and surgical sterilization (such as oophorectomy or tubal ligation). When used consistently and correctly, "double-barrier" methods of contraception can be used as an effective alternative to highly effective contraception methods. Contraceptive measures such as Plan B™, sold for emergency use after unprotected sex, are not acceptable methods for routine use
Key exclusion criteria (8 listed)
- Pregnant or breastfeeding.
- An active Central nervous system (CNS) infection, demyelinating disease, or degenerative neurological disease.
- History of psychogenic nonepileptic seizures.
- Any disease or condition (other than TSC) at the initial visit that could compromise the hematologic, cardiovascular (including any cardiac conduction defect), pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the IP, or would place the participant at increased risk or interfere with the assessment of safety/efficacy. This may include any illness in the past 4 weeks which in the opinion of the investigator may affect seizure frequency.
- Unwillingness to avoid excessive alcohol use or cannabis use throughout the study.
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 |
|---|---|---|
| GanaxoloneGNX will be administered. | Drug | Ganaxolone (GNX) oral suspension, 3 times a day (TID) |
Primary outcome
Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Withdrawals and Dose-Reductions Due to AEs
Time frame: Up to 150 Weeks
An adverse event (AE) was any untoward medical occurrence in a clinical study patient, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event was any untoward medical occurrence that, at any dose, results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or any other event that requires scientific judgment.
Number of Participants With Clinically Significant Changes in Vital Parameters
Time frame: Up to 150 Weeks
Vital parameters including heart rate, respiration rate, blood pressure, and body temperature were measured in seated position for at least 5 minutes.
Number of Participants With Clinically Significant Changes in Physical Examinations
Time frame: Up to 150 Weeks
Physical examinations included a full physical evaluation of body systems including: General appearance, head (eyes, ears, nose, and throat), cardiovascular, respiratory, gastrointestinal, genitourinary, musculoskeletal, endocrine/metabolic, hematologic/lymphatic, skin, and other systems as appropriate.
Number of Participants With Clinically Significant Changes in Neurological Examinations
Time frame: Up to 150 Weeks
Neurological examinations included an evaluation of: Cranial nerves, motor exam, sensory exam, reflexes, coordination/cerebellar
Number of Participants With Clinically Significant Changes in Developmental Examinations
Time frame: Up to 150 Weeks
Developmental examinations (applicable only to pediatric participants 1 to 17 years of age, inclusive) included an evaluation of speech/language, motor skills, and social skills
Number of Participants With Clinically Significant 12-lead Electrocardiogram (ECG) Findings
Time frame: Up to 150 Weeks
Twelve-lead ECGs were performed by the physician using an ECG machine that automatically calculates the heart rate and measure PR, QRS, QT and QTc intervals.
Number of Participants With Clinically Significant Changes in Hematology Parameters
Time frame: Week 1 through Week 150
Blood samples were collected for the analysis of hematology parameters: hemoglobin, hematocrit, erythrocytes, thrombocytes (platelet count). Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
Number of Participants With Clinically Significant Changes in Chemistry Parameters
Time frame: Up to 150 Weeks
Blood samples were collected for the analysis of chemistry parameters: blood urea nitrogen (BUN), potassium, alanine aminotransferase (ALT), alkaline phosphatase (ALP)/serum glutamic pyruvic transaminase (SGPT), total bilirubin, creatinine, sodium, aspartate aminotransferase (AST)/serum glutamic oxaloacetic (SGOT), total protein, fasting blood glucose, calcium, carbon dioxide, estimated glomerular filtration rate (eGFR), and chloride
Number of Participants With Clinically Significant Changes in Urinalysis
Time frame: Up to 150 Weeks
Urine samples were collected for the analysis of urinalysis parameters: Specific gravity, color, clarity, pH, glucose, protein, blood, nitrite, protein, ketones, bilirubin, urobilinogen, nitrite, leukocyte esterase by dipstick, and microscopic examination (if blood or protein was abnormal)
Number of Participants (Age Greater Than 17 Years) With Abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
Time frame: Up to Week 52
The C-SSRS is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 5 items for suicidal behavior (1. Preparatory acts or behavior, 2. Aborted attempt, 3. Interrupted attempt, 4. Actual attempt, 5. Completed suicide) will be reported. The score ranges from 1 to 5 and higher scores indicate worse symptoms.
1 secondary outcome
Percent Change From Baseline in 28-day Seizure Frequency During Open Label Extension
Time frame: Baseline (Day 1), Week 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
54 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ganaxolone in people with Tuberous Sclerosis Complex, run by Marinus Pharmaceuticals at 54 sites in 10 countries.
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 6 inclusion and 8 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: stopped early, as of the sponsor's last update on 1 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 54 sites in United States, Germany, Spain, Canada, China and 5 more countries. 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?
Yes. Results are posted in the registry (first posted 1 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Reporting Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Withdrawals and Dose-Reductions Due to AEs (time frame: Up to 150 Weeks). The study lists 10 primary outcomes in all. 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 1 Oct 2025.