Graves OphthalmopathyPhase 2SIRGONCT04598815
Sirolimus for Graves' Orbitopathy (GO)
A Phase 2 study testing Sirolimus in people with Graves Ophthalmopathy, run by University of Pisa at 1 site in Italy.
Trial summary
Graves' Orbitopathy (GO) is a disabling and disfiguring condition associated with Graves' Disease, due to autoimmunity against antigens expressed by the thyroid and orbital tissues, and resulting in orbital fibroblast proliferation and release of glycosaminoglycans. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Graves Ophthalmopathy
- What is being tested?
- Sirolimus
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 54 participants
- Where
- 1 siteItaly
- Study dates
- 1 Jun 2023to 31 May 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients willing and capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form
- A diagnosis of Graves' disease based on the presence of hyperthyroidism (either untreated or treated with antithyroid drugs) associated with detectable anti-thyrotropic hormone (TSH) receptor autoantibodies (TRAb). Patients must be euthyroid under control on stable medical regimen and every effort will be made to maintain the euthyroid status for the entire duration of the clinical trial
- A moderate-to-severe GO, defined as the presence of at least one of the following criteria: an exophthalmos ≥2 mm compared with normal values for sex and race; presence of inconstant to constant diplopia; a lid retraction ≥2 mm
- Active GO: CAS (4) ≥2 out of 7 points in the most affected eye
- GO duration ≤18 months
- Male and female patients of age: 18-75 years
Show all 11Show fewer
- Creatinine values within the reference range
- Indexes of liver function (AST, ALT, γGT, alkaline phosphatase, total and direct bilirubin) within the normal range
- Normal blood count, absence of diseases of hematopoiesis
- Women of childbearing potential (WOCBP, namely not in menopause or in menopause since less than two years; in all other instances women will be considered as non-WOCBP) and men who are sexually active with WOCBP must use any contraceptive method with a failure rate of less than 1% per year (as indicated in Appendix) for at least 6 and 7,5 months, respectively, after the last dose of the investigational drug (see also 2014_09_HMA_CTFG_Contraception.pdf, namely the "2014 CTFG Reccommendtions related to contraception and pregnancy testing in clinical trials")
- Compliant patients, regular follow-up possible
Who cannot join
- Optic neuropathy
- Treatment with glucocorticoids, other immunosuppressive drugs or selenium for the last three months
- Previous surgery or radiotherapy for GO
- Radioiodine treatment for hyperthyroidism over the last 3 months, as it can affect GO
- Contraindications to Sirolimus: hypersensitivity to the active substance or to any of the excipients; use of medications interfering with the pharmacokinetic and/or pharmacodynamic properties of rapamycin (e.g. CYP3A4 inhibitors or inducers; see "prohibited therapies")
- Contraindications to GC: hypersensitivity to the active substance or to any of the excipients; uncontrolled hypertension, uncontrolled diabetes; history of peptic ulcer; urinary infections, glaucoma, systemic fungal infections, systemic infections unless appropriate therapy is employed, idiopathic thrombocytopenic purpura, cerebral edema associated with malaria.
Show all 13Show fewer
- Use of medications interfering with GC or increasing the risk of GC-related adverse events (see prohibited therapies)
- Pregnant or lactating females as determined by positive serum or urine HCG test at baseline
- Acute or chronic liver disease
- Relevant malignancies
- Current and/or previous diseases of hematopoiesis
- Recent (≤1 year) history of alcoholism or drug abuse
- Mental illness that prevent patients from comprehensive, written informed consent
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II, Randomized, Superiority, Adaptive, Open-label, Single-center, Clinical Trial to Evaluate the Efficacy of Sirolimus (Rapamycin) in Patients With Graves' Disease and Moderate-to-severe and Active Graves' Orbitopathy (GO)
Graves' Orbitopathy (GO) is a disabling and disfiguring condition associated with Graves' Disease, due to autoimmunity against antigens expressed by the thyroid and orbital tissues, and resulting in orbital fibroblast proliferation and release of glycosaminoglycans. The current treatments available, especially glucocorticoids, are not effective in all patients. Two cases of patients with GO treated with Sirolimus have been reported with an excellent response to the drug.
The rationale for the use of Sirolimus lies in its mechanisms of action. Sirolimus is able to inhibit T-cell activation as well as fibroblast proliferation. In addition, acts indirectly on the Insulin-Like Growth Factor-1 (IGF-1) pathway, and recent clinical trials have shown that a monoclonal antibody against the IGF-1 receptor (Teprotumumab) is effective in patients with GO. Thus, Sirolimus could be used in GO as monotherapy in patients with GO.
Read more
The aim of the present drug vs standard treatment, open-label, randomized clinical trial is to evaluate the efficacy of Sirolimus in patients with moderately severe, active GO.
54 patients (27 per group) will be randomized into two groups, A and B. Patients in group A will receive Sirolimus for 12 weeks. Patients in group B will receive methylprendnisolone for 12 weeks.
The primary objective of the study is the response of GO at 24 weeks based on a composite evaluation. The secondary Objectives will be: 1) the response of of GO at 12, 36 and 48 weeks; 2) Relapse of GO at 36 and 48 weeks (worsening compared with the 24-week evaluation); 3) The reduction of proptosis at 12, 24, 36 and 48 weeks (proportion of patients with a reduction of proptosis of at least 2 mm); 4) Reduction of the GO clinical activity score (CAS) at 12, 24, 36 and 48 weeks; 5) Quality of life (Qol) at 12, 24, 36 and 48 weeks.
The safety objectives will be adverse events, adverse drug reactions, unexpected adverse reaction, suspected unexpected adverse reactions and death, across the study and at 12, 24, 36 and 48 weeks.
Read the detailed description
Study Design Phase II, randomized, adaptive, superiority, open-label, single-center, pilot clinical trial.
Fifty-four patients with moderate-to-severe and active GO will be randomized into two intervention groups, A and B, with a ratio of 1: 1. Subjects assigned to group A will receive Sirolimus for 12 weeks. Patients assigned to group B will receive a cumulative dose of 4.5 g of methylprednisolone divided into 12 weekly infusions. This treatment scheme is the clinical standard and patients would be treated with methylprednisolone in any case, even if they did not accept to participate to the study.
Enrollment duration: 24 months Study duration: 36 months Tentative start of trial: July 1st 2022
Study Population Fifty-four patients with Graves' disease and GO will be recruited during the routine clinical activity carried out at the Endocrinology Unit II of AOUP, which is a tertiary referral center for thyroid diseases.
Study Timeline
- Screening visit (2-6 weeks before the first visit)
- 1st (baseline) visit - (Time 0): randomization and administration of the first dose of trial agent
- Treatment period (week 1-week 12): daily administration of the trial agent (Sirolimus) or weekly administration of the standard treatment (methylprednisolone)
- Methylprednisolone treatment period and treatment visits (week 2-week 13); weekly methylprednisolone administrations (week 2-week 13)
- Safety visits (week 3, 5, 7, 9, 11)
- 2nd visit (week 12)
- 3rd visit (week 24)
- 4th visit (week 36)
- 5th visit (week 48)
- Conditions
- Graves Ophthalmopathy
- Being tested
- Sirolimus
- Medical subject headings
- Graves Ophthalmopathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamycin
Group Sirolimus: Patients will receive a first dose of Sirolimus of one 2 mg tablet on the first day, given approximately at 10 am, followed by 0.5 mg tablet per day for 12 weeks.
Group Methylprednisolone: Methylprednisolone pulse therapy will be administered for 12 weeks as follows: 500 mg iv once weekly for 6 weeks, then 250 mg iv once weekly for a further 6 weeks, for a cumulative dose 4.5 g.
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus | Experimental | Sirolimus for 12 weeks Receives: Drug: Sirolimus |
| Methylprendnisolone | Experimental | Methylprednisolone for 12 weeks Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
GO overall response
Percentage of subjects with at least two of the following compared to baseline: a) Improvement in CAS by at least 1 point ; b) Improvement in exophthalmos by at least 2 mm; c) Improvement in lid aperture by at least 2 mm; d) Improvement in eye muscle ductions ≥8 degrees; e) Improvement of visual acuity by at least 0.2/1, without worsening of any of these parameters in the contralateral eye if applicable.
Measured: 24 weeks
Secondary outcomes (18)
GO overall response
Percentage of subjects with at least two of the following compared to baseline: a) Improvement in CAS by at least 1 point ; b) Improvement in exophthalmos by at least 2 mm; c) Improvement in lid aperture by at least 2 mm; d) Improvement in eye muscle ductions ≥8 degrees; e) Improvement of visual acuity by at least 0.2/1, without worsening of any of these parameters in the contralateral eye if applicable.
Measured: 12 weeks
GO overall response
Percentage of subjects with at least two of the following compared to baseline: a) Improvement in CAS by at least 1 point ; b) Improvement in exophthalmos by at least 2 mm; c) Improvement in lid aperture by at least 2 mm; d) Improvement in eye muscle ductions ≥8 degrees; e) Improvement of visual acuity by at least 0.2/1, without worsening of any of these parameters in the contralateral eye if applicable.
Measured: 36 weeks
GO overall response
Percentage of subjects with at least two of the following compared to baseline: a) Improvement in CAS by at least 1 point ; b) Improvement in exophthalmos by at least 2 mm; c) Improvement in lid aperture by at least 2 mm; d) Improvement in eye muscle ductions ≥8 degrees; e) Improvement of visual acuity by at least 0.2/1, without worsening of any of these parameters in the contralateral eye if applicable.
Measured: 48 weeks
GO relapse
Percentage of subjects with worsening of GO in comparison with the 24 week evaluation. Worsening is defined as change in two of the following in at least one eye (compared to baseline): a) Worsening in CAS by at least 1 point; b) Worsening of exophthalmos (>2 mm); c) Worsening of lid aperture (>2 mm); d) Worsening of eye ductions ≥8 degrees; d) Worsening of visual acuity by at least 0.2/1
Measured: 36 weeks
GO relapse
Percentage of subjects with worsening of GO in comparison with the 24 week evaluation. Worsening is defined as change in two of the following in at least one eye (compared to baseline): a) Worsening in CAS by at least 1 point; b) Worsening of exophthalmos (>2 mm); c) Worsening of lid aperture (>2 mm); d) Worsening of eye ductions ≥8 degrees; d) Worsening of visual acuity by at least 0.2/1
Measured: 48 weeks
Change in exophthalmos
Percentage of subjects with a reduction greater than or equal to 2 mm compared with the baseline evaluation in the study eye, without worsening in the contralateral eye
Measured: 12 weeks
Change in exophthalmos
Percentage of subjects with a reduction greater than or equal to 2 mm compared with the baseline evaluation in the study eye, without worsening in the contralateral eye
Measured: 24 weeks
Change in exophthalmos
Percentage of subjects with a reduction greater than or equal to 2 mm compared with the baseline evaluation in the study eye, without worsening in the contralateral eye
Measured: 36 weeks
Change in exophthalmos
Percentage of subjects with a reduction greater than or equal to 2 mm compared with the baseline evaluation in the study eye, without worsening in the contralateral eye
Measured: 48 weeks
Change in the clinical activity score (CAS)
Percentage of subjects with a reduction of CAS by at least two points in the study eye, without worsening in the contralateral eye
Measured: 12 weeks
Change in the clinical activity score (CAS)
Percentage of subjects with a reduction of CAS by at least two points in the study eye, without worsening in the contralateral eye
Measured: 24 weeks
Change in the clinical activity score (CAS)
Percentage of subjects with a reduction of CAS by at least two points in the study eye, without worsening in the contralateral eye
Measured: 36 weeks
Change in the clinical activity score (CAS)
Percentage of subjects with a reduction of CAS by at least two points in the study eye, without worsening in the contralateral eye
Measured: 48 weeks
Change in quality of life
Comparison of the quality of life scores between the two groups, determined with a questionnaire specific for GO (GO-QoL)
Measured: 12 weeks
Change in quality of life
Comparison of the quality of life scores between the two groups, determined with a questionnaire specific for GO (GO-QoL)
Measured: 24 weeks
Change in quality of life
Comparison of the quality of life scores between the two groups, determined with a questionnaire specific for GO (GO-QoL)
Measured: 36 weeks
Change in quality of life
Comparison of the quality of life scores between the two groups, determined with a questionnaire specific for GO (GO-QoL)
Measured: 48 weeks
Percentage of adverse events
Percentage of adverse events, adverse drug reactions, death across the study period
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2020
- Study start1 Jun 2023 (estimated)
- Primary completion31 May 2024 (estimated)
- Study completion31 May 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graves OphthalmopathyStudies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 54 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Pisa
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Marinò Michele, University of Pisa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04598815
- Sponsor's protocol ID
- SIRGO
- Acronym
- SIRGO
- Status last verified
- Mar 2023
- First submitted
- 16 Oct 2020
- Last updated
- 15 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Randomized, Superiority, Adaptive, Open-label, Single-center, Clinical Trial to Evaluate the Efficacy of Sirolimus (Rapamycin) in Patients With Graves' Disease and Moderate-to-severe and Active Graves' Orbitopathy (GO)
- ClinicalTrials.gov ID
- NCT04598815
- Acronym / sponsor ID
- SIRGO / SIRGO
- Conditions
- Graves Ophthalmopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 54 participants (estimated)
- Lead sponsor
- University of PisaUniversity, hospital or other
- Study start
- 1 Jun 2023 (estimated)
- Primary completion
- 31 May 2024 (estimated)
- Study completion
- 31 May 2025 (estimated)
- First posted
- 22 Oct 2020 (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.
Graves' Orbitopathy (GO) is a disabling and disfiguring condition associated with Graves' Disease, due to autoimmunity against antigens expressed by the thyroid and orbital tissues, and resulting in orbital fibroblast proliferation and release of glycosaminoglycans. The current treatments available, especially glucocorticoids, are not effective in all patients. Two cases of patients with GO treated with Sirolimus have been reported with an excellent response to the drug.
The rationale for the use of Sirolimus lies in its mechanisms of action. Sirolimus is able to inhibit T-cell activation as well as fibroblast proliferation. In addition, acts indirectly on the Insulin-Like Growth Factor-1 (IGF-1) pathway, and recent clinical trials have shown that a monoclonal antibody against the IGF-1 receptor (Teprotumumab) is effective in patients with GO. Thus, Sirolimus could be used in GO as monotherapy in patients with GO.
The aim of the present drug vs standard treatment, open-label, randomized clinical trial is to evaluate the efficacy of Sirolimus in patients with moderately severe, active GO.
54 patients (27 per group) will be randomized into two groups, A and B. Patients in group A will receive Sirolimus for 12 weeks. Patients in group B will receive methylprendnisolone for 12 weeks.
The primary objective of the study is the response of GO at 24 weeks based on a composite evaluation. The secondary Objectives will be: 1) the response of of GO at 12, 36 and 48 weeks; 2) Relapse of GO at 36 and 48 weeks (worsening compared with the 24-week evaluation); 3) The reduction of proptosis at 12, 24, 36 and 48 weeks (proportion of patients with a reduction of proptosis of at least 2 mm); 4) Reduction of the GO clinical activity score (CAS) at 12, 24, 36 and 48 weeks; 5) Quality of life (Qol) at 12, 24, 36 and 48 weeks.
The safety objectives will be adverse events, adverse drug reactions, unexpected adverse reaction, suspected unexpected adverse reactions and death, across the study and at 12, 24, 36 and 48 weeks.
Detailed description
Study Design Phase II, randomized, adaptive, superiority, open-label, single-center, pilot clinical trial.
Fifty-four patients with moderate-to-severe and active GO will be randomized into two intervention groups, A and B, with a ratio of 1: 1. Subjects assigned to group A will receive Sirolimus for 12 weeks. Patients assigned to group B will receive a cumulative dose of 4.5 g of methylprednisolone divided into 12 weekly infusions. This treatment scheme is the clinical standard and patients would be treated with methylprednisolone in any case, even if they did not accept to participate to the study.
Enrollment duration: 24 months Study duration: 36 months Tentative start of trial: July 1st 2022
Study Population Fifty-four patients with Graves' disease and GO will be recruited during the routine clinical activity carried out at the Endocrinology Unit II of AOUP, which is a tertiary referral center for thyroid diseases.
Study Timeline
- Screening visit (2-6 weeks before the first visit)
- 1st (baseline) visit - (Time 0): randomization and administration of the first dose of trial agent
- Treatment period (week 1-week 12): daily administration of the trial agent (Sirolimus) or weekly administration of the standard treatment (methylprednisolone)
- Methylprednisolone treatment period and treatment visits (week 2-week 13); weekly methylprednisolone administrations (week 2-week 13)
- Safety visits (week 3, 5, 7, 9, 11)
- 2nd visit (week 12)
- 3rd visit (week 24)
- 4th visit (week 36)
- 5th visit (week 48)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sirolimus | Experimental | Drug: SirolimusSirolimus for 12 weeks |
| Methylprendnisolone | Experimental | Drug: SirolimusMethylprednisolone for 12 weeks |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Patients willing and capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form
- A diagnosis of Graves' disease based on the presence of hyperthyroidism (either untreated or treated with antithyroid drugs) associated with detectable anti-thyrotropic hormone (TSH) receptor autoantibodies (TRAb). Patients must be euthyroid under control on stable medical regimen and every effort will be made to maintain the euthyroid status for the entire duration of the clinical trial
- A moderate-to-severe GO, defined as the presence of at least one of the following criteria: an exophthalmos ≥2 mm compared with normal values for sex and race; presence of inconstant to constant diplopia; a lid retraction ≥2 mm
- Active GO: CAS (4) ≥2 out of 7 points in the most affected eye
- GO duration ≤18 months
Key exclusion criteria (13 listed)
- Optic neuropathy
- Treatment with glucocorticoids, other immunosuppressive drugs or selenium for the last three months
- Previous surgery or radiotherapy for GO
- Radioiodine treatment for hyperthyroidism over the last 3 months, as it can affect GO
- Contraindications to Sirolimus: hypersensitivity to the active substance or to any of the excipients; use of medications interfering with the pharmacokinetic and/or pharmacodynamic properties of rapamycin (e.g. CYP3A4 inhibitors or inducers; see "prohibited therapies")
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 |
|---|---|---|
| SirolimusAlso: RapamycinGroup Sirolimus: Patients will receive a first dose of Sirolimus of one 2 mg tablet on the first day, given approximately at 10 am, followed by 0.5 mg tablet per day for 12 weeks. Group Methylprednisolone: Methylprednisolone pulse therapy will be administered for 12 weeks as follows: 500 mg iv once weekly for 6 weeks, then 250 mg iv once weekly for a further 6 weeks, for a cumulative dose 4.5 g. | Drug | Methylprendnisolone, Sirolimus |
Primary outcome
GO overall response
Time frame: 24 weeks
Percentage of subjects with at least two of the following compared to baseline: a) Improvement in CAS by at least 1 point ; b) Improvement in exophthalmos by at least 2 mm; c) Improvement in lid aperture by at least 2 mm; d) Improvement in eye muscle ductions ≥8 degrees; e) Improvement of visual acuity by at least 0.2/1, without worsening of any of these parameters in the contralateral eye if applicable.
18 secondary outcomes
GO overall response
Time frame: 12 weeks
GO overall response
Time frame: 36 weeks
GO overall response
Time frame: 48 weeks
GO relapse
Time frame: 36 weeks
GO relapse
Time frame: 48 weeks
Change in exophthalmos
Time frame: 12 weeks
Change in exophthalmos
Time frame: 24 weeks
Change in exophthalmos
Time frame: 36 weeks
Change in exophthalmos
Time frame: 48 weeks
Change in the clinical activity score (CAS)
Time frame: 12 weeks
Change in the clinical activity score (CAS)
Time frame: 24 weeks
Change in the clinical activity score (CAS)
Time frame: 36 weeks
Change in the clinical activity score (CAS)
Time frame: 48 weeks
Change in quality of life
Time frame: 12 weeks
Change in quality of life
Time frame: 24 weeks
Change in quality of life
Time frame: 36 weeks
Change in quality of life
Time frame: 48 weeks
Percentage of adverse events
Time frame: 48 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2023.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sirolimus in people with Graves Ophthalmopathy, run by University of Pisa at 1 site in Italy.
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 11 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: status not confirmed, as of the sponsor's last update on 15 Mar 2023. 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 Italy. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Michele Marinò, MD (+39050997346, michele.marino@med.unipi.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
GO overall response (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Mar 2023.