Adenylosuccinate Synthase 1 Deficient MyopathyPhase 1(ASA-CS01)NCT07412821

A Phase 1b Study of Adenylosuccinic Acid (ASA-001) for Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.

A Phase 1 study testing adenylosuccinic acid in people with Adenylosuccinate Synthase 1 Deficient Myopathy, run by Cure ADSSL1 at 1 site in United States.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts

Official record

Search all trials

Trial summary

The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of ASA-001 in two adults diagnosed with ADSS1 deficient myopathy. The main questions it aims to answer are: Whether ASA-001 can be safely administered to ADSS1 deficient myopathy patients; Whether daily treatment with… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Adenylosuccinate Synthase 1 Deficient Myopathy
What is being tested?: adenylosuccinic acid
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 2 participants
Where: 1 siteUnited States
Study dates: 26 Mar 2026to 31 Dec 2027
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 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male and females, age 18 years and above and weighing between 60 and 85 kg
Patient(s) diagnosed with ADSS1 deficient myopathy with homozygous or compound heterozygous mutations in the ADSS1 gene.
Able to understand and comply with all the study requirements
Is willing and legally able to provide written informed consent.
Willing to use highly effective contraception

Who cannot join

Any medical condition that could, in the Investigator's opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
Any patient who, in the Investigator's opinion, seems unable/unwilling to comply with the study procedures.
Women who are pregnant or breastfeeding, or planning to become pregnant
As judged by the investigator, clinical features are present at the time of screening / baseline assessments indicating that safe travel and completion of the study and its assessments are unlikely
Other severe systemic illness or disease
Participation in another treatment clinical study within thirty (30) days or 5 half-lives of the investigational product, whichever is longer, prior to signing and dating of Informed Consent Form for this study.

Show all 13Show fewer

Known hypersensitivity to any of the components/excipients in ASA-001
Serologic evidence of hepatitis B, C, or HIV
Ongoing/active infection (including current COVID-19 infection)
Presence of clinically significant liver or renal abnormalities
Clinical chemistry and hematology outside the limits acceptable for this patient population
History of anaphylaxis or severe allergic reaction to drug therapy or foods.
Concomitant medications to manage chronic condition(s) must not interfere with the mechanism of action for ASA-001 in the opinion of the Investigator and dose(s) must not alter for at least 4 weeks before screening through to dosing (Day 1).

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

United States1 site

University of California Los Angeles (UCLA)
Los Angeles, California, 90095

Other trials in United States

About this study

Official title: A Phase 1b, Open Label, Single and Multiple Ascending Dose-escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Subcutaneous Adenylosuccinic Acid (ASA) in Two Siblings With Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.

Whether ASA-001 can be safely administered to ADSS1 deficient myopathy patients;
Whether daily treatment with ASA-001 provides benefit or slows progression of disease.

Participants will:

Take ASA-001 every day for 8 months;
Visit the clinic once every 2 weeks for check-ups and tests

Conditions: Adenylosuccinate Synthase 1 Deficient Myopathy
Being tested: adenylosuccinic acid
Keywords: ADSS1 myopathy, adenylosuccinic acid, adenylosuccinate, ASA-001, Phase 1b clinical trial, ADSSL1 myopathy, Adenylosuccinate synthase-like 1 myopathy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

adenylosuccinic acidDrug
Also called adenylosuccinate, ASA-001, succinyl adenosine monophosphate (S-AMP)
ASA-001 will be administered as a sterile solution (500-2500 mg/day) by sub-cutaneous infusion pump.

Study groups in NCT07412821
Study group	Role	What they receive
Multiple ascending dose-escalation of ASA-001	Experimental	Not described Receives: Drug: adenylosuccinic acid

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence and severity of all adverse events (AEs), treatment emergent adverse events (TAEs) and serious adverse events (SAEs).
AEs are classified as to seriousness, expectedness, and potential relationship to the investigational product.
Seriousness (SAE) criteria:
- Results in death
- Is life-threatening
- Requires in-patient hospitalization or prolongation of existing hospitalization
- Results in persistent or significant disability/incapacity
- Is a congenital anomaly/birth defect in the offspring of a participant.
Severity criteria:
- Mild: Awareness of signs or symptom, but easily tolerated
- Moderate: Discomfort sufficient to cause interference with normal activities
- Severe: Incapacitating, with inability to perform normal activities
Expectedness criteria:
- Unexpected: An AE for which the nature or severity is inconsistent with information in the protocol/consent form
- Expected: An AE known to be associated with any of the study procedures
Causality criteria:
- Unrelated
- Possibly related
- Probably related
Measured: Screening through to the last assessment at 10 months.
Observed and changes from baseline in vital signs.
Vital signs (including blood pressure (BP; mmHg), heart rate (HR; beats per minute), respiratory rate (RR; breaths per minute), and oral/tympanic/axillary temperature are measured at all visits. Height is measured at baseline only (cm); weight is measured at each visit (Kg).
Measured: Screening through to the last assessment at 10 months.
Observed and changes from baseline in 12-lead electrocardiogram (ECG).
A standard 12-lead ECG will be recorded per Schedule of Events after 5 mins rest. The ECG has little or no risk. Skin may become red or itchy in the areas where the stickers with ECG electrodes are placed. The gel that is used may cause mild skin irritation/abrasion, along with the sticky pads used to attach the electrodes.
Measured: Screening through to the last assessment at 10 months.
Observed and changes from baseline in physical examination.
A physical exam will be given at screening and per schedule of events to assess general appearance, HEENT (head, eyes, ear, nose and throat), cardiovascular, respiratory (chest), gastrointestinal (abdomen), dermatological, extremities, neurological (mental status, cranial nerves, motor examination, sensory examination, coordination, reflexes, gait), musculoskeletal and lymphatics.
Measured: Screening through to the last assessment at 10 months.
Observed and changes from baseline in hematology, comprehensive metabolic panel (CMP), hepatic tests, renal function, and serology.
Laboratory analyte samples will be collected throughout the study per Schedule of Events. Hematology (complete blood count with auto-differential), comprehensive metabolic panel (CMP) including HbA1C, with hepatic tests (to include serum transaminases, , total bilirubin and alkaline phosphatase), renal function to include creatinine, Cystatin C, urinalysis, uric acid, INR, APTT; Serology will include HIV-1, hepatitis B and C at screening.
Measured: Screening through to last assessment at 10 months.
Observed and changes from baseline in pulmonary function.
Forced Vital Capacity (FVC), Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure will be measured by spirometry to assess the strength of respiratory muscles, with MIP indicating the maximum pressure generated during a forceful inhalation against a closed airway, and MEP indicating the maximum pressure generated during a forceful exhalation against a closed airway monitor.
Measured: Screening through to the last assessment at 10 months.
Observed and changes from baseline in cardiac function.
A standard trans-thoracic echocardiogram will be recorded and read at selected study visits per Schedule of Events. Assessments include standard assessments of the anatomy (veins and atria, atrioventricular segment, ventricles, conotruncus, great arteries) as well as left ventricular and valvular function (including measurements of the left ventricular ejection fraction (LVEF)).
Measured: Screening through to the last assessment at 10 months.
Observed and changes from baseline in injection site monitoring.
Injection site(s) will be examined at each visit.
Measured: Screening through to the last assessment at 10 months.

Secondary outcomes (11)

Plasma pharmacokinetic (PK) concentration of ASA-001.
Singles doses of ASA-001 will be administered on Day 1 and Day 14 and samples will be taken at baseline (time 0), 0.5, 1, 2, 4, 6, 8 h post dose. The results from Day 1 samples are required prior to dose escalation on Day 14. The multiple dose escalation phase will commence on Day 29 and conclude on Day 85 - plasma samples will be taken pre-dose (time 0) and at 1, 2 and 4 h post-dosing.
Measured: Day 1 through to Day 85 (visit 8).
Observed and changes from baseline in serum creatinine concentration (preliminary efficacy).
Serum creatinine will be assessed within the clinical chemistry assessment, since prior clinical experience with ASA showed normalisation (from below normal range) of this measure.
Measured: Screening through to the last assessment at 10 months.
Change from baseline in Ten meter walk/run time (10MWT) (preliminary efficacy).
The time required for the participant to run or walk (fastest way to cover the distance safely) 10 meters (on a safe walkway) from a standing position will be measured (sec or min)
Measured: Screening through to the last assessment at 10 months.
Change from baseline in Timed Up-and-Go (TUG) (preliminary efficacy).
The time taken to stand from a chair, walk three metres, turn around and return and sit in the chair will be measured (sec or min).
Measured: Screening through to the last assessment at 10 months.
Change from baseline in Jamar grip strength dynamometry (preliminary efficacy).
Squeeze and grip strength will be measured using a hand held dynamometer (Kg).
Measured: Screening through to the last assessment at 10 months
Change from baseline in nerve conduction study with repetitive nerve stimulation test (preliminary efficacy).
The speed at which electrical signals travel through peripheral nerves will be assessed to identify nerve damage or dysfunction (meters/sec).
Measured: Screening through to the last assessment at 10 months
Change from baseline in clinical muscle histopathology on core needle muscle biopsy (preliminary efficacy).
Muscle core needle biopsy will be sampled at baseline and at the end of treatment (day 239) and clinical histopathology scoring will measured to assess preliminary efficacy.
Measured: Screening and at the end of treatment at Day 239.
Changes from baseline in muscle protein abundance and phosphorylation on core needle biopsy (preliminary efficacy).
Quantitative proteomics (including of phosphorylated proteins) will be used to measure muscle protein abundance and phosphorylation (preliminary efficacy).
Measured: Screening and at the end of treatment at Day 239.
Change from baseline in Quality Of Life (QOL)/patient reported outcomes measure (preliminary efficacy).
Patient reported outcomes will be assessed using Neuro-QOL Item Bank v.10 - Upper Extremity Function (Fine Motor, ADL) - Short Form (PROMIS Health Organization, National Institute for Neurological Disorders and Stroke (NINDS), 2008-13).
Measured: Screening through to the last assessment at 10 months
Changes from baseline in muscle metabolomics on core needle biopsy (preliminary efficacy).
Semi-quantitative metabolomics will be used to measure relative changes in muscle metabolite levels from baseline.
Measured: Screening and at the end of treatment at Day 239.
Change from baseline in muscle transcriptomic signature on core needle biopsy (preliminary efficacy).
RNA sequencing will be used to assess the muscle transcriptomic signature in response to treatment.
Measured: Screening and at the end of treatment at Day 239.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

26 Mar 202631 Dec 2027

First posted17 Feb 2026
Study start26 Mar 2026
Primary completion31 Dec 2027 (estimated)
Study completion31 Dec 2027 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Adenylosuccinate Synthase 1 Deficient MyopathyStudies 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: 2 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07412821
Sponsor's protocol ID: ASA-CS01
Acronym: (ASA-CS01)
Status last verified: Apr 2026
First submitted: 4 Jan 2026
Last updated: 7 Apr 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Undecided

Publications

Rybalka E, Kourakis S, Bonsett CA, Moghadaszadeh B, Beggs AH, Timpani CA. Adenylosuccinic Acid: An Orphan Drug with Untapped Potential. Pharmaceuticals (Basel). 2023 May 31;16(6):822. doi: 10.3390/ph16060822.
PubMed 37375769
Rybalka E, Park HJ, Nalini A, Baskar D, Polavarapu K, Durmus H, Xia Y, Wan L, Shieh PB, Moghadaszadeh B, Beggs AH, Mack DL, Smith AST, Hanna-Rose W, Jinnah HA, Timpani CA, Shen M, Upadhyay J, Brault JJ, Hall MD, Baweja N, Kakkar P. Current insights in ultra-rare adenylosuccinate synthetase 1 myopathy - meeting report on the First Clinical and Scientific Conference. 3 June 2024, National Centre for Advancing Translational Science, Rockville, Maryland, the United States of America. Orphanet J Rare Dis. 2024 Nov 26;19(1):438. doi: 10.1186/s13023-024-03429-x.
PubMed 39593137

Study at a glance

From the registry record, last updated by the sponsor on 7 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1b, Open Label, Single and Multiple Ascending Dose-escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Subcutaneous Adenylosuccinic Acid (ASA) in Two Siblings With Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.
ClinicalTrials.gov ID: NCT07412821
Acronym / sponsor ID: (ASA-CS01) / ASA-CS01
Conditions: Adenylosuccinate Synthase 1 Deficient Myopathy
Keywords: ADSS1 myopathy, adenylosuccinic acid, adenylosuccinate, ASA-001, Phase 1b clinical trial, ADSSL1 myopathy, Adenylosuccinate synthase-like 1 myopathy
Phase: Phase 1
Study type: Interventional
Recruitment status: Enrolling by invitation
Enrolment: 2 participants (estimated)
Lead sponsor: Cure ADSSL1University, hospital or other
Collaborators: University of California, Los Angeles
Study start: 26 Mar 2026 (actual)
Primary completion: 31 Dec 2027 (estimated)
Study completion: 31 Dec 2027 (estimated)
First posted: 17 Feb 2026 (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.

Whether ASA-001 can be safely administered to ADSS1 deficient myopathy patients;
Whether daily treatment with ASA-001 provides benefit or slows progression of disease.

Participants will:

Take ASA-001 every day for 8 months;
Visit the clinic once every 2 weeks for check-ups and tests

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Multiple ascending dose-escalation of ASA-001	Experimental	Drug: adenylosuccinic acid

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Male and females, age 18 years and above and weighing between 60 and 85 kg
Patient(s) diagnosed with ADSS1 deficient myopathy with homozygous or compound heterozygous mutations in the ADSS1 gene.
Able to understand and comply with all the study requirements
Is willing and legally able to provide written informed consent.
Willing to use highly effective contraception

Key exclusion criteria (13 listed)

Any medical condition that could, in the Investigator's opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
Any patient who, in the Investigator's opinion, seems unable/unwilling to comply with the study procedures.
Women who are pregnant or breastfeeding, or planning to become pregnant
As judged by the investigator, clinical features are present at the time of screening / baseline assessments indicating that safe travel and completion of the study and its assessments are unlikely
Other severe systemic illness or disease

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
adenylosuccinic acidAlso: adenylosuccinate, ASA-001, succinyl adenosine monophosphate (S-AMP)ASA-001 will be administered as a sterile solution (500-2500 mg/day) by sub-cutaneous infusion pump.	Drug	Multiple ascending dose-escalation of ASA-001

Primary outcome

Incidence and severity of all adverse events (AEs), treatment emergent adverse events (TAEs) and serious adverse events (SAEs).
Time frame: Screening through to the last assessment at 10 months.
AEs are classified as to seriousness, expectedness, and potential relationship to the investigational product. Seriousness (SAE) criteria: * Results in death * Is life-threatening * Requires in-patient hospitalization or prolongation of existing hospitalization * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect in the offspring of a participant. Severity criteria: * Mild: Awareness of signs or symptom, but easily tolerated * Moderate: Discomfort sufficient to cause interference with normal activities * Severe: Incapacitating, with inability to perform normal activities Expectedness criteria: * Unexpected: An AE for which the nature or severity is inconsistent with information in the protocol/consent form * Expected: An AE known to be associated with any of the study procedures Causality criteria: * Unrelated * Possibly related * Probably related
Observed and changes from baseline in vital signs.
Time frame: Screening through to the last assessment at 10 months.
Vital signs (including blood pressure (BP; mmHg), heart rate (HR; beats per minute), respiratory rate (RR; breaths per minute), and oral/tympanic/axillary temperature are measured at all visits. Height is measured at baseline only (cm); weight is measured at each visit (Kg).
Observed and changes from baseline in 12-lead electrocardiogram (ECG).
Time frame: Screening through to the last assessment at 10 months.
A standard 12-lead ECG will be recorded per Schedule of Events after 5 mins rest. The ECG has little or no risk. Skin may become red or itchy in the areas where the stickers with ECG electrodes are placed. The gel that is used may cause mild skin irritation/abrasion, along with the sticky pads used to attach the electrodes.
Observed and changes from baseline in physical examination.
Time frame: Screening through to the last assessment at 10 months.
A physical exam will be given at screening and per schedule of events to assess general appearance, HEENT (head, eyes, ear, nose and throat), cardiovascular, respiratory (chest), gastrointestinal (abdomen), dermatological, extremities, neurological (mental status, cranial nerves, motor examination, sensory examination, coordination, reflexes, gait), musculoskeletal and lymphatics.
Observed and changes from baseline in hematology, comprehensive metabolic panel (CMP), hepatic tests, renal function, and serology.
Time frame: Screening through to last assessment at 10 months.
Laboratory analyte samples will be collected throughout the study per Schedule of Events. Hematology (complete blood count with auto-differential), comprehensive metabolic panel (CMP) including HbA1C, with hepatic tests (to include serum transaminases, , total bilirubin and alkaline phosphatase), renal function to include creatinine, Cystatin C, urinalysis, uric acid, INR, APTT; Serology will include HIV-1, hepatitis B and C at screening.
Observed and changes from baseline in pulmonary function.
Time frame: Screening through to the last assessment at 10 months.
Forced Vital Capacity (FVC), Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure will be measured by spirometry to assess the strength of respiratory muscles, with MIP indicating the maximum pressure generated during a forceful inhalation against a closed airway, and MEP indicating the maximum pressure generated during a forceful exhalation against a closed airway monitor.
Observed and changes from baseline in cardiac function.
Time frame: Screening through to the last assessment at 10 months.
A standard trans-thoracic echocardiogram will be recorded and read at selected study visits per Schedule of Events. Assessments include standard assessments of the anatomy (veins and atria, atrioventricular segment, ventricles, conotruncus, great arteries) as well as left ventricular and valvular function (including measurements of the left ventricular ejection fraction (LVEF)).
Observed and changes from baseline in injection site monitoring.
Time frame: Screening through to the last assessment at 10 months.
Injection site(s) will be examined at each visit.

11 secondary outcomes

Plasma pharmacokinetic (PK) concentration of ASA-001.
Time frame: Day 1 through to Day 85 (visit 8).
Observed and changes from baseline in serum creatinine concentration (preliminary efficacy).
Time frame: Screening through to the last assessment at 10 months.
Change from baseline in Ten meter walk/run time (10MWT) (preliminary efficacy).
Time frame: Screening through to the last assessment at 10 months.
Change from baseline in Timed Up-and-Go (TUG) (preliminary efficacy).
Time frame: Screening through to the last assessment at 10 months.
Change from baseline in Jamar grip strength dynamometry (preliminary efficacy).
Time frame: Screening through to the last assessment at 10 months
Change from baseline in nerve conduction study with repetitive nerve stimulation test (preliminary efficacy).
Time frame: Screening through to the last assessment at 10 months
Change from baseline in clinical muscle histopathology on core needle muscle biopsy (preliminary efficacy).
Time frame: Screening and at the end of treatment at Day 239.
Changes from baseline in muscle protein abundance and phosphorylation on core needle biopsy (preliminary efficacy).
Time frame: Screening and at the end of treatment at Day 239.
Change from baseline in Quality Of Life (QOL)/patient reported outcomes measure (preliminary efficacy).
Time frame: Screening through to the last assessment at 10 months
Changes from baseline in muscle metabolomics on core needle biopsy (preliminary efficacy).
Time frame: Screening and at the end of treatment at Day 239.
Change from baseline in muscle transcriptomic signature on core needle biopsy (preliminary efficacy).
Time frame: Screening and at the end of treatment at Day 239.

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 7 Apr 2026.
Background: PubMed 37375769Rybalka E, Kourakis S, Bonsett CA, Moghadaszadeh B, Beggs AH, Timpani CA. Adenylosuccinic Acid: An Orphan Drug with Untapped Potential. Pharmaceuticals (Basel). 2023 May 31;16(6):822. doi: 10.3390/ph16060822.
Background: PubMed 39593137Rybalka E, Park HJ, Nalini A, Baskar D, Polavarapu K, Durmus H, Xia Y, Wan L, Shieh PB, Moghadaszadeh B, Beggs AH, Mack DL, Smith AST, Hanna-Rose W, Jinnah HA, Timpani CA, Shen M, Upadhyay J, Brault JJ, Hall MD, Baweja N, Kakkar P. Current insights in ultra-rare adenylosuccinate synthetase 1 myopathy - meeting report on the First Clinical and Scientific Conference. 3 June 2024, National Centre for Advancing Translational Science, Rockville, Maryland, the United States of America. Orphanet J Rare Dis. 2024 Nov 26;19(1):438. doi: 10.1186/s13023-024-03429-x.
Individual participant dataSharing planned: Undecided

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.

Perry B Shieh, M.D., Ph.D. principal investigator

UCLA Medical Centre

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing adenylosuccinic acid in people with Adenylosuccinate Synthase 1 Deficient Myopathy, run by Cure ADSSL1 at 1 site 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 5 inclusion and 13 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: enrolling by invitation, as of the sponsor's last update on 7 Apr 2026. 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 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?

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?

Incidence and severity of all adverse events (AEs), treatment emergent adverse events (TAEs) and serious adverse events (SAEs). (time frame: Screening through to the last assessment at 10 months.). The study lists 8 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 NCT07412821, which also lists 2 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 7 Apr 2026.