Congenital Myotonic DystrophyPhase 2/3REACH CDM XNCT05004129
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
A Phase 2/3 study testing Tideglusib in people with Congenital Myotonic Dystrophy, run by AMO Pharma Limited at 14 sites in 4 countries.
Trial summary
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve. Read more about this study
- Who is it for?
- Ages 6 to 45 years
- Conditions the study is for
- Congenital Myotonic Dystrophy
- What is being tested?
- Tideglusib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 76 participants
- Where
- 14 sitesin 4 countries
- Study dates
- 23 Aug 2021to 31 Dec 2026
- Phase
- Phase 2/3
- 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 6 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:
- Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
- Diagnosis must be genetically confirmed
- Subjects must be male or female aged ≥6 years to ≤45 years at Screening
- Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
- Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)
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- Subject's caregiver must be willing and able to support participation for duration of study
- Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
- Subjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:
- Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
- Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations)
- Subject's caregiver must be willing and able to support participation for duration of study
- Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
Who cannot join
- Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
- New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
- Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
- Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
- Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months other than the AMO-02- MD-2-003 study
- Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or respiratory disease) that may impact the interpretability of the study results
- Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry
Trial locations and contacts
14 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
14 sites in 4 countries
United States10 sites, 8 recruiting
- Recruiting
Lurie's Children's Hospital
Chicago, Illinois, 60611
Joseph Alberts, 312-227-4449, jalberts@luriechildrens.org
Caitlyn Daley, caitlyn.daley@reachcdm.com
- Recruiting
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Laura Knosp, 319-384-6277, laura-knosp@uiowa.edu
Caitlyn Daley, caitlyn.daley@reachcdm.com
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Annette Guy, 501-364-3380, GuyEA@archildrens.org
Caitlyn Daley, caitlyn.daley@reachcdm.com
- Enrolling by invitation
University of California, Los Angeles (UCLA)
Los Angeles, California, 90095
- Withdrawn
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
- Enrolling by invitation
Stanford University
Palo Alto, California, 94304
- Recruiting
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Heather DiCostanzo, 412-692-7660, follhl@upmc.edu
Caitlyn Daley, catilyn.daley@reachcdm.com
- Completed
Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program
Richmond, Virginia, 23219
- Recruiting
University of Rochester - Medical Center
Rochester, New York, 14642
James Hilbert, 585-273-5590, james_hilbert@URMC.Rochester.eud
Caitlyn Daley, caitlyn.daley@reachcdm.com
- Recruiting
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132
Domink May, 801-587-4864, dominik.may@hsc.utah.edu
Caitlyn Daley, catilyn.daley@reachcdm.com
Canada2 sites, 2 recruiting
- Enrolling by invitation
Children's Hospital London Health Sciences Centre (LHSC)
London, Ontario, N6A 4G5
- Recruiting
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Emilie Hill-Smith, 613-737-7600 x4017, ehillsmith@cheo.on.ca
Caitlyn Daley, caitlyn.daley@reachcdm.com
Australia1 site, 1 recruiting
- Recruiting
The Bright Alliance
Randwick, New South Wales, 2031
AMO Study Coordinator, 02 9382 5534, SCHN-SCHClinicalTrials@health.nsw.gov.au
Caitlyn Daley, caitlyn.daley@reachcdm.com
New Zealand1 site
- Completed
New Zealand Clinical Research (NZCR)
Auckland, 1010
About this study
Official title: An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.
Read the detailed description
This is an open-label study of either a weight-adjusted 1000 mg fixed dose or a weight banded fixed dose of tideglusib across a 52-week treatment period with an open-ended optional extended access period. The subjects are children and adolescents with Congenital DM1 who participated in the antecedent AMO-02-MD-2-003 study or individuals with either Congenital or Childhood onset DM1 who are treatment naïve.
- Conditions
- Congenital Myotonic Dystrophy
- Being tested
- Tideglusib
- Keywords
- Tideglusib, AMO-02-MD-2-004, Congenital Myotonic Dystrophy, Myotonic Dystrophy, Dystrophia Myotonica, Myotonia Atrophica, Myotonia Dystrophica, Myotonic Dystrophy, Congenital, Steinert Disease, Steinert Myotonic Dystrophy, Steinert's Disease
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TideglusibDrug
Tideglusib dosing will be weight-adjusted at 400 mg, 600 mg, or 1000 mg dose levels, or weight banded fixed doses of 400 mg, 600 mg, 800 mg or 1000 mg, with each subject starting at a weight-adjusted 400 mg dose level for 2 weeks, then up titrating to a weight-adjusted 600 mg dose level for the next 2 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Tideglusib | Experimental | Weight adjusted or weight banded tideglusib, orally, once daily Receives: Drug: Tideglusib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety (Adverse Events)
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from Screening to Enrolment (where applicable), from Enrolment to End of Treatment (52 Weeks), and End of Treatment to the End of Follow-up period.
Measured: 52 Weeks
Safety (Adverse Events) - With Optional Expanded Access
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from End of Treatment to End of Optional Extended Access, and End of Optional Extended Access to the End of Follow-up period.
Measured: Week 60 and every 8 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
The Clinician-completed Congenital DM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity of domains that are clinically relevant in Congenital DM1.
Measured: 52 Weeks
Secondary outcomes (16)
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) - With Optional Expanded Access
The Clinician-completed Congenital DM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity of domains that are clinically relevant in Congenital DM1.
Measured: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Clinical Global Impressions Improvement Scale (CGI-I)
The CGI-I requires the clinician to rate how much the subject's illness has changed (improved, worsened or stayed the same) relative to a baseline state.
Measured: 54 Weeks
Clinical Global Impressions Improvement Scale (CGI-I) - With Optional Expanded Access
The CGI-I requires the clinician to rate how much the subject's illness has changed (improved, worsened or stayed the same) relative to a baseline state.
Measured: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score
The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study.
Measured: 54 weeks
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score - With Optional Expanded Access
The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study.
Measured: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
CC-CDM1-RS provides a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 symptoms that the caregiver is asked to rate from 0 to 4 based on overall severity.
Measured: 52 weeks
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) - With Optional Expanded Access
CC-CDM1-RS provides a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 symptoms that the caregiver is asked to rate from 0 to 4 based on overall severity.
Measured: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Clinical Global Impressions Severity Scale (CGI-S)
The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis.
Measured: 54 weeks
Clinical Global Impressions Severity Scale (CGI-S) - With Optional Expanded Access
The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis.
Measured: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Autism Behavior Inventory- Clinician (ABI-C)
ABI-C is a 14 item rating scale requires the clinician to assess the core features of Autism Spectrum Disorder as well as common associated behaviors.
Measured: 52 Weeks
Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey Interview
The Vineland Adaptive Behavior Scales require the clinician to measure personal and social skills needed for everyday living. It provides a targeted assessment of adaptive behavior via a semi-structured parent or caregiver interview.
Measured: 52 Weeks
10-meter walk-run test
The 10-meter walk/run test is a performance measure used to assess walking speed in meters per second over a short distance. It can be used as an assessment of functional mobility.
Measured: 52 Weeks
Plasma Troponin T levels
Troponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.
Measured: 52 Weeks
Plasma Troponin T levels - With Optional Expanded Access
Troponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.
Measured: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Blood Leukocytes CTG Repeats
A genomic blood sample will be taken from all subjects to confirm the average number of repeats at sampling and estimate the number of leukocyte CTG repeats inherited for an individual subject.
Measured: Baseline and Week 52
Blood Leukocytes CTG Repeats - With Optional Expanded Access
A genomic blood sample will be taken from all subjects to confirm the average number of repeats at sampling and estimate the number of leukocyte CTG repeats inherited for an individual subject. For subjects that have already passed Week 52 at implementation of this endpoint, a single genomic blood sample will taken at the next in-clinic OEA visit.
Measured: Week 52 or later
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2021
- Study start23 Aug 2021
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Myotonic DystrophyStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 76 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AMO Pharma Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM, Study director, AMO Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05004129
- Sponsor's protocol ID
- AMO-02-MD-2-004
- Acronym
- REACH CDM X
- Status last verified
- May 2025
- First submitted
- 5 Aug 2021
- Last updated
- 28 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)
- ClinicalTrials.gov ID
- NCT05004129
- Acronym / sponsor ID
- REACH CDM X / AMO-02-MD-2-004
- Conditions
- Congenital Myotonic Dystrophy
- Keywords
- Tideglusib, AMO-02-MD-2-004, Congenital Myotonic Dystrophy, Myotonic Dystrophy, Dystrophia Myotonica, Myotonia Atrophica, Myotonia Dystrophica, Myotonic Dystrophy, Congenital
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 76 participants (estimated)
- Lead sponsor
- AMO Pharma LimitedIndustry (pharma, biotech, devices)
- Study start
- 23 Aug 2021 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 13 Aug 2021 (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.
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.
Detailed description
This is an open-label study of either a weight-adjusted 1000 mg fixed dose or a weight banded fixed dose of tideglusib across a 52-week treatment period with an open-ended optional extended access period. The subjects are children and adolescents with Congenital DM1 who participated in the antecedent AMO-02-MD-2-003 study or individuals with either Congenital or Childhood onset DM1 who are treatment naïve.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tideglusib | Experimental | Drug: TideglusibWeight adjusted or weight banded tideglusib, orally, once daily |
Who may qualify
- Ages
- 6 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:
- Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
- Diagnosis must be genetically confirmed
- Subjects must be male or female aged ≥6 years to ≤45 years at Screening
- Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
Key exclusion criteria (7 listed)
- Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
- New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
- Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
- Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
- Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months other than the AMO-02- MD-2-003 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 |
|---|---|---|
| TideglusibTideglusib dosing will be weight-adjusted at 400 mg, 600 mg, or 1000 mg dose levels, or weight banded fixed doses of 400 mg, 600 mg, 800 mg or 1000 mg, with each subject starting at a weight-adjusted 400 mg dose level for 2 weeks, then up titrating to a weight-adjusted 600 mg dose level for the next 2 weeks. | Drug | Tideglusib |
Primary outcome
Safety (Adverse Events)
Time frame: 52 Weeks
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from Screening to Enrolment (where applicable), from Enrolment to End of Treatment (52 Weeks), and End of Treatment to the End of Follow-up period.
Safety (Adverse Events) - With Optional Expanded Access
Time frame: Week 60 and every 8 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from End of Treatment to End of Optional Extended Access, and End of Optional Extended Access to the End of Follow-up period.
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
Time frame: 52 Weeks
The Clinician-completed Congenital DM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity of domains that are clinically relevant in Congenital DM1.
16 secondary outcomes
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) - With Optional Expanded Access
Time frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Clinical Global Impressions Improvement Scale (CGI-I)
Time frame: 54 Weeks
Clinical Global Impressions Improvement Scale (CGI-I) - With Optional Expanded Access
Time frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score
Time frame: 54 weeks
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score - With Optional Expanded Access
Time frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
Time frame: 52 weeks
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) - With Optional Expanded Access
Time frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Clinical Global Impressions Severity Scale (CGI-S)
Time frame: 54 weeks
Clinical Global Impressions Severity Scale (CGI-S) - With Optional Expanded Access
Time frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Autism Behavior Inventory- Clinician (ABI-C)
Time frame: 52 Weeks
Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey Interview
Time frame: 52 Weeks
10-meter walk-run test
Time frame: 52 Weeks
Plasma Troponin T levels
Time frame: 52 Weeks
Plasma Troponin T levels - With Optional Expanded Access
Time frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Blood Leukocytes CTG Repeats
Time frame: Baseline and Week 52
Blood Leukocytes CTG Repeats - With Optional Expanded Access
Time frame: Week 52 or later
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 28 May 2025.
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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.
Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM study director
AMO Pharma
14 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Tideglusib in people with Congenital Myotonic Dystrophy, run by AMO Pharma Limited at 14 sites in 4 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 13 inclusion and 7 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 28 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Canada, Australia, New Zealand. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (Adverse Events) (time frame: 52 Weeks). The study lists 3 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 28 May 2025.