Heterozygous Familial HypercholesterolemiaPhase 3HAUSER-RCTNCT02392559
Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
A Phase 3 study testing Evolocumab in people with Heterozygous Familial Hypercholesterolemia, run by Amgen at 66 sites in 26 countries.
Trial summary
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia. Read more about this study
- Who is it for?
- Ages 10 to 17 years
- Conditions the study is for
- Heterozygous Familial Hypercholesterolemia
- What is being tested?
- Evolocumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 158 participants
- Where
- 66 sitesin 26 countries
- Study dates
- 24 Mar 2016to 25 Nov 2019
- 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 10 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female ≥ 10 to ≤ 17 years of age (before 18th birthday)
- Diagnosis of heterozygous familial hypercholesterolemia
- On an approved statin with stable optimized dose for ≥ 4 weeks
- Other lipid-lowering therapy stable for ≥ 4 weeks (fibrates must be stable for ≥ 6 weeks)
- Fasting LDL-C ≥ 130 mg/dL (3.4 mmol/L)
- Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
Who cannot join
- Type 1 diabetes, or type 2 diabetes that is or poorly controlled
- Uncontrolled hyperthyroidism or hypothyroidism
- Cholesterylester transfer protein (CETP) inhibitor in the last 12 months, or mipomersen or lomitapide in the last 5 months
- Previously received evolocumab or any other investigational therapy to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9).
- Lipid apheresis within the last 12 weeks prior to screening.
- Homozygous familial hypercholesterolemia
Trial locations and contacts
66 sites in 26 countries.
66 sites in 26 countries
United States12 sites
Research Site
Asheville, North Carolina, 28803
Research Site
Cincinnati, Ohio, 45227
Research Site
Farmington, Connecticut, 06032
Research Site
Iowa City, Iowa, 52242
Research Site
Minneapolis, Minnesota, 55454
Research Site
Morgantown, West Virginia, 26506
Research Site
Nashville, Tennessee, 37212
Research Site
Pittsburgh, Pennsylvania, 15224
Research Site
Salt Lake City, Utah, 84113
Research Site
The Bronx, New York, 10467
Research Site
Towson, Maryland, 21204
Research Site
Wilmington, Delaware, 19803
Brazil5 sites
Research Site
Brasília, Federal District, 71625-175
Research Site
Fortaleza, Ceará, 60430-270
Research Site
São Paulo, 04040-000
Research Site
São Paulo, 05403-000
Research Site
Vitória, Espírito Santo, 29055-450
Italy5 sites
Research Site
Palermo, 90127
Research Site
Pisa, 56124
Research Site
Roma, 00161
Research Site
Roma, 00165
Research Site
Torino, 10126
Spain5 sites
Research Site
A Coruña, Galicia, 15001
Research Site
Barcelona, Catalonia, 08036
Research Site
Córdoba, Andalusia, 14004
Research Site
Lugo, Galicia, 27003
Research Site
Seville, Andalusia, 41013
Austria3 sites
Research Site
Feldkirch, 6800
Research Site
Salzburg, 5020
Research Site
Vienna, 1090
Belgium3 sites
Research Site
Brussels, 1200
Research Site
La Louvière, 7100
Research Site
Leuven, 3000
Canada3 sites
Research Site
Chicoutimi, Quebec, G7H 5H6
Research Site
Chicoutimi, Quebec, G7H 7K9
Research Site
Québec, Quebec, G1V 4W2
Czechia3 sites
Research Site
Ostrava-Poruba, 708 52
Research Site
Prague, 150 06
Research Site
Svitavy, 568 25
Colombia2 sites
Research Site
Barranquilla, Atlántico, 080020
Research Site
Floridablanca, Santander Department
Russia2 sites
Research Site
Moscow, 125412
Research Site
Saint Petersburg, 191025
South Africa2 sites
Research Site
Parow, Western Cape, 7505
Research Site
Pretoria, Gauteng, 0087
Turkey (Türkiye)2 sites
Research Site
Ankara, 06500
Research Site
Izmir, 35100
United Kingdom2 sites
Research Site
Birmingham, B4 6NH
Research Site
London, WC1N 3JH
About this study
Official title: Double-blind, Randomized, Multicenter, Placebo-Controlled Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for LDL-C Reduction in Pediatric Subjects 10 to 17 Years of Age With HeFH
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.
Read the detailed description
A study to evaluate the effect of 24 weeks of subcutaneous (SC) evolocumab compared with placebo, when added to standard of care, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in pediatric subjects 10 to 17 years of age with heterozygous familial hypercholesterolemia (HeFH).
- Being tested
- Evolocumab, Placebo
- Keywords
- Hypercholesterolemia, Elevated Cholesterol, High Cholesterol, Heterozygous Familial Hypercholesterolemia, PCSK9 mutations, Paediatric, pediatric, Childhood Familial Hypercholesterolemia
- Medical subject headings
- Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EvolocumabDrug
Also called AMG 145; EvoMab
Dose of subcutaneous evolocumab every 4 weeks
PlaceboDrug
Dose of subcutaneous placebo treatment every 4 weeks
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Matching subcutaneous injection every 4 weeks (QM) Receives: Drug: Placebo |
| EvoMab 420 mg QM | Experimental | Evolocumab subcutaneous injection QM Receives: Drug: Evolocumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline to Week 24 in LDL-C
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from interactive voice response system [IVRS]), scheduled visit and the interaction of treatment with scheduled visit as covariates. The model uses an unstructured covariance.
Measured: Baseline, Week 24
Secondary outcomes (13)
Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.
Measured: Baseline, Week 22, Week 24
Change From Baseline to Week 24 in LDL-C
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.
Measured: Baseline, Week 24
Percent Change From Baseline to Week 24 in Non-HDL-C
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates
Measured: Baseline, Week 24
Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB)
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.
Measured: Baseline, Week 24
Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.
Measured: Baseline, Week 24
Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.
Measured: Baseline, Week 24
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs
An adverse event (AE) is defined as any untoward medical occurrence that does not necessarily have a causal relationship with study treatment. An SAE is defined as an adverse event that: is fatal; is a life threatening; requires in-patient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; or other medically important serious event. Events were graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) grading scale (1=mild; 2=moderate; 3=severe; 4=life-threatening; 5=death). Events were defined as treatment emergent if they occurred after the first dose of study drug and up to and including 30 days after the last dose or the end of study date, whichever is earlier.
Measured: From first dose of study drug up to and including 30 days after the last dose or end of study date (Week 24), whichever was earlier. Mean (SD) duration on study was 5.664 (0.278) and 5.608 (0.137) months for Placebo and EvoMab arms, respectively.
Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3
Laboratory toxicity grading was based on NCI CTCAE grading. Grade 3 indicates severe toxicity and Grade 4 indicates life-threatening toxicity. Values representing a worsening from baseline are shown.
Measured: Week 24
Change From Baseline Over Time in Systolic Blood Pressure
Measured: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Change From Baseline Over Time in Diastolic Blood Pressure
Measured: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Change From Baseline Over Time in Heart Rate
Measured: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Number of Participants Testing Positive for Anti-Evolocumab Antibodies
Measured: up to Week 24
Serum Evolocumab Concentrations Over Time
Measured: Week 12, Week 22, Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2015 (estimated)
- Study start24 Mar 2016
- Primary completion25 Nov 2019
- Study completion25 Nov 2019
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.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Heterozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by AmgenThe sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 158 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02392559
- Sponsor's protocol ID
- 20120123
- Other IDs
- 2014-002277-11, 2014
- Acronym
- HAUSER-RCT
- Status last verified
- Nov 2022
- First submitted
- 25 Feb 2015
- Last updated
- 8 Nov 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
Publications
Santos RD, Ruzza A, Hovingh GK, Wiegman A, Mach F, Kurtz CE, Hamer A, Bridges I, Bartuli A, Bergeron J, Szamosi T, Santra S, Stefanutti C, Descamps OS, Greber-Platzer S, Luirink I, Kastelein JJP, Gaudet D; HAUSER-RCT Investigators. Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia. N Engl J Med. 2020 Oct 1;383(14):1317-1327. doi: 10.1056/NEJMoa2019910. Epub 2020 Aug 29.
PubMed 32865373Gaudet D, Langslet G, Gidding SS, Luirink IK, Ruzza A, Kurtz C, Lu C, Somaratne R, Raal FJ, Wiegman A. Efficacy, safety, and tolerability of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia: Rationale and design of the HAUSER-RCT study. J Clin Lipidol. 2018 Sep-Oct;12(5):1199-1207. doi: 10.1016/j.jacl.2018.05.007. Epub 2018 May 22.
PubMed 30318065Gaudet D, Ruzza A, Bridges I, Maruff P, Schembri A, Hamer A, Mach F, Bergeron J, Gaudet I, Pierre JS, Kastelein JJP, Hovingh GK, Wiegman A, Raal FJ, Santos RD. Cognitive function with evolocumab in pediatric heterozygous familial hypercholesterolemia. J Clin Lipidol. 2022 Sep-Oct;16(5):676-684. doi: 10.1016/j.jacl.2022.07.005. Epub 2022 Jul 21.
PubMed 36210291Schmidt AF, Carter JL, Pearce LS, Wilkins JT, Overington JP, Hingorani AD, Casas JP. PCSK9 monoclonal antibodies for the primary and secondary prevention of cardiovascular disease. Cochrane Database Syst Rev. 2020 Oct 20;10(10):CD011748. doi: 10.1002/14651858.CD011748.pub3.
PubMed 33078867
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-blind, Randomized, Multicenter, Placebo-Controlled Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for LDL-C Reduction in Pediatric Subjects 10 to 17 Years of Age With HeFH
- ClinicalTrials.gov ID
- NCT02392559
- Acronym / sponsor ID
- HAUSER-RCT / 20120123
- Keywords
- Hypercholesterolemia, Elevated Cholesterol, High Cholesterol, Heterozygous Familial Hypercholesterolemia, PCSK9 mutations, Paediatric, pediatric, Childhood Familial Hypercholesterolemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 158 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 24 Mar 2016 (actual)
- Primary completion
- 25 Nov 2019 (actual)
- Study completion
- 25 Nov 2019 (actual)
- First posted
- 19 Mar 2015 (estimated)
- Results
- Posted 15 Jul 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.
Detailed description
A study to evaluate the effect of 24 weeks of subcutaneous (SC) evolocumab compared with placebo, when added to standard of care, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in pediatric subjects 10 to 17 years of age with heterozygous familial hypercholesterolemia (HeFH).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboMatching subcutaneous injection every 4 weeks (QM) |
| EvoMab 420 mg QM | Experimental | Drug: EvolocumabEvolocumab subcutaneous injection QM |
Who may qualify
- Ages
- 10 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Male or female ≥ 10 to ≤ 17 years of age (before 18th birthday)
- Diagnosis of heterozygous familial hypercholesterolemia
- On an approved statin with stable optimized dose for ≥ 4 weeks
- Other lipid-lowering therapy stable for ≥ 4 weeks (fibrates must be stable for ≥ 6 weeks)
- Fasting LDL-C ≥ 130 mg/dL (3.4 mmol/L)
Key exclusion criteria (6 listed)
- Type 1 diabetes, or type 2 diabetes that is or poorly controlled
- Uncontrolled hyperthyroidism or hypothyroidism
- Cholesterylester transfer protein (CETP) inhibitor in the last 12 months, or mipomersen or lomitapide in the last 5 months
- Previously received evolocumab or any other investigational therapy to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9).
- Lipid apheresis within the last 12 weeks prior to screening.
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 |
|---|---|---|
| EvolocumabAlso: AMG 145; EvoMabDose of subcutaneous evolocumab every 4 weeks | Drug | EvoMab 420 mg QM |
| PlaceboDose of subcutaneous placebo treatment every 4 weeks | Drug | Placebo |
Primary outcome
Percent Change From Baseline to Week 24 in LDL-C
Time frame: Baseline, Week 24
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from interactive voice response system [IVRS]), scheduled visit and the interaction of treatment with scheduled visit as covariates. The model uses an unstructured covariance.
13 secondary outcomes
Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C
Time frame: Baseline, Week 22, Week 24
Change From Baseline to Week 24 in LDL-C
Time frame: Baseline, Week 24
Percent Change From Baseline to Week 24 in Non-HDL-C
Time frame: Baseline, Week 24
Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB)
Time frame: Baseline, Week 24
Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio
Time frame: Baseline, Week 24
Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio
Time frame: Baseline, Week 24
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs
Time frame: From first dose of study drug up to and including 30 days after the last dose or end of study date (Week 24), whichever was earlier. Mean (SD) duration on study was 5.664 (0.278) and 5.608 (0.137) months for Placebo and EvoMab arms, respectively.
Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3
Time frame: Week 24
Change From Baseline Over Time in Systolic Blood Pressure
Time frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Change From Baseline Over Time in Diastolic Blood Pressure
Time frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Change From Baseline Over Time in Heart Rate
Time frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Number of Participants Testing Positive for Anti-Evolocumab Antibodies
Time frame: up to Week 24
Serum Evolocumab Concentrations Over Time
Time frame: Week 12, Week 22, Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 32865373Santos RD, Ruzza A, Hovingh GK, Wiegman A, Mach F, Kurtz CE, Hamer A, Bridges I, Bartuli A, Bergeron J, Szamosi T, Santra S, Stefanutti C, Descamps OS, Greber-Platzer S, Luirink I, Kastelein JJP, Gaudet D; HAUSER-RCT Investigators. Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia. N Engl J Med. 2020 Oct 1;383(14):1317-1327. doi: 10.1056/NEJMoa2019910. Epub 2020 Aug 29.
- Background: PubMed 30318065Gaudet D, Langslet G, Gidding SS, Luirink IK, Ruzza A, Kurtz C, Lu C, Somaratne R, Raal FJ, Wiegman A. Efficacy, safety, and tolerability of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia: Rationale and design of the HAUSER-RCT study. J Clin Lipidol. 2018 Sep-Oct;12(5):1199-1207. doi: 10.1016/j.jacl.2018.05.007. Epub 2018 May 22.
- Background: PubMed 36210291Gaudet D, Ruzza A, Bridges I, Maruff P, Schembri A, Hamer A, Mach F, Bergeron J, Gaudet I, Pierre JS, Kastelein JJP, Hovingh GK, Wiegman A, Raal FJ, Santos RD. Cognitive function with evolocumab in pediatric heterozygous familial hypercholesterolemia. J Clin Lipidol. 2022 Sep-Oct;16(5):676-684. doi: 10.1016/j.jacl.2022.07.005. Epub 2022 Jul 21.
- Derived from this study: PubMed 33078867Schmidt AF, Carter JL, Pearce LS, Wilkins JT, Overington JP, Hingorani AD, Casas JP. PCSK9 monoclonal antibodies for the primary and secondary prevention of cardiovascular disease. Cochrane Database Syst Rev. 2020 Oct 20;10(10):CD011748. doi: 10.1002/14651858.CD011748.pub3.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
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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.
MD study director
Amgen
66 sites in 26 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Evolocumab in people with Heterozygous Familial Hypercholesterolemia, run by Amgen at 66 sites in 26 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 6 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: finished, as of the sponsor's last update on 8 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 66 sites in United States, Brazil, Italy, Spain, Austria and 21 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. Some participants in this study may receive a placebo. 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 15 Jul 2020). 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?
Percent Change From Baseline to Week 24 in LDL-C (time frame: Baseline, Week 24). 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 NCT02392559, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2022.