Hemophilia AHUPNCT02807753
The Hemophilia Ultrasound Project
An observational study of Hemophilia A and Hemophilia B, run by University of Miami at 4 sites in United States.
Trial summary
To evaluate the prevalence of subclinical arthropathy in children with severe hemophilia undergoing a prophylaxis regimen and without evidence of target joints, using a validated ultrasound scoring method. Read more about this study
- Who is it for?
- Ages Up to 30 months
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- Ankle and Knee Ultrasound Joint Assessment and Ankle and Knee Magnetic Resonance Imaging
- How many people
- 18 participants
- Where
- 4 sitesUnited States
- Study dates
- 16 Sep 2016to 7 Dec 2020
- Phase
- Observational
- 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 Up to 30 months
- All sexes
- No healthy volunteers
- Child
Study population: Mild, Moderate and Severe Hemophilia A \& B patients.
Who may be able to join
- Severe Hemophilia Cohort:Patients from 0 up to 30 months of age with diagnosis of severe hemophilia A or B defined as a factor VIII:C/IX:C of <1% undergoing prophylaxis regimen with any factor VIII or IX concentrate and without evidence (clinical or by history) of target joint disease.
- Mild/moderate Hemophilia Cohort:Patients from 0 up to 30 months of age with diagnosis of mild hemophilia A or B defined as a factor VIII:C/IX:C of 5-50% and those with diagnosis of moderate hemophilia A or B defined a s a factor VIII:C/IX:C of 1-5% without evidence (clinical or by history) of target joint disease and no history of spontaneous joint bleeds.
Who cannot join
- Patients with concomitant Hepatitis B, Hepatitis C and HIV viral infections (because of a recognized arthritogen effect).
- Present or prior history of anti FVIII or IX inhibitors.
- Known inflammatory joint disease.
- Established target joint.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
University of Florida
Gainesville, Florida, 32610
University of Kentucky
Lexington, Kentucky, 40536
The University of Miami - Department of Pediatrics
Miami, Florida, 33136
Tulane
New Orleans, Louisiana, 70112
About this study
Official title: Evaluation of Joint Arthropathy Using Ultrasound Technique in Children With Severe Hemophilia Undergoing Prophylaxis Regimen
To evaluate the prevalence of subclinical arthropathy in children with severe hemophilia undergoing a prophylaxis regimen and without evidence of target joints, using a validated ultrasound scoring method.
Read the detailed description
Persons with Hemophilia (A or B) often experience recurrent joint bleeds, most commonly affecting the ankles, knees and elbows. These bleeds can lead to significant pain and disability over time. If recurrent joint bleeds are not managed with prompt and adequate infusions of factor concentrate, the damage caused by the presence of blood in the joint space will eventually result in a condition called debilitating chronic hemophilic arthropathy.
The initiation of and adherence to a prophylactic infusion regimen, starting with the first or second joint bleed, is essential for prevention of progression to arthropathy.
Studies have demonstrated that prophylaxis with recombinant or plasma-derived factor VIII or IX concentrates is effective in preventing clinical joint bleeds and the progression to debilitating joint disease in patients with severe hemophilia A or B respectively. However, for patients on prophylaxis, the absence of symptomatic joint bleeds and/or structural and functional abnormalities of joints on physical examination and plain radiographic images can lead to the erroneous assumption that the prophylaxis is completely effective. It has been established that patients with severe hemophilia are still at risk for subclinical bleeding ("microbleeds") despite seemingly adequate prophylaxis.
Young adults, despite a lifetime on prophylaxis and apparently normal joints are developing arthropathy in their 20's and 30's. Prophylaxis as currently practiced may only be delaying the onset of clinical joint disease.
The recent advancements in ultrasound imaging (US) have been proven to be effective in confirming a joint bleed, monitoring the evolution of a joint bleed and assessing the resolution or recurrence of a bleed. Previous studies have evaluated the prevalence of subclinical arthropathy in young hemophilic adults using both US and MRI techniques and concluded that US is as effective and sensitive as MRI identifying these subclinical joint abnormalities.
However, to the investigators' knowledge, no prior studies have used US technique over an extended period of time to monitor the natural evolution of joint arthropathy in children with hemophilia who are adherent to an established prophylaxis regimen and have no evidence of clinical joint compromise.
The seminal Joint Outcome Study, which confirmed the role of prophylaxis in preventing overt clinical joint disease, mandated a trough residual factor VIII level of 1%. While this trough level significantly decreased overt hemarthroses and joint damage, the evidence suggestive of microbleeds raised a question as to the protection afforded by such a low trough level. Intuitively it would seem as if a higher trough level should confer greater protection against microbleeds, and result in more sustainable joint health. The "ideal" protective trough, has not been established.
The investigators' hypothesis is that US is a valuable imaging technique to monitor the natural evolution of hemophilic arthropathy in children with severe hemophilia A or B who are undergoing prophylaxis regimen and do not manifest clinical evidence of hemophilic arthropathy.
Through this observational study, the investigators will provide valuable information in regards the prevalence, progression and severity of joint abnormalities. The use of US to detect microbleeds before the cumulative damaging effects demonstrable by MRI, will also allow tailoring of treatment and the implementation of new prophylaxis strategies.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- Ankle and Knee Ultrasound Joint Assessment, Ankle and Knee Magnetic Resonance Imaging
- Keywords
- Hemophilia, Arthropathy, Pediatric
- Medical subject headings
- Hemophilia A, Hemophilia B, Joint Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ankle and Knee Ultrasound Joint AssessmentOther
Ultrasound Examination of the ankles and knees
Ankle and Knee Magnetic Resonance ImagingOther
Ankle and Knee Magnetic Resonance Imaging
| Study group | Role | What they receive |
|---|---|---|
| Severe Hemophilia A or B | Medical history, physical exam, vital signs,physical therapist targeted exam and ultrasound joint assessment (ankles and knees) every 6 months.
MRI joint assessment (knees and ankles) at End of Trial Visit (Month 60). Receives: Other: Ankle and Knee Ultrasound Joint Assessment; Other: Ankle and Knee Magnetic Resonance Imaging | |
| Mild/Moderate Hemophilia A or B | Medical history, physical exam, vital signs,physical therapist targeted exam and ultrasound joint assessment (ankles and knees) every year.
MRI joint assessment (knees and ankles) at End of Trial Visit (Month 60). Receives: Other: Ankle and Knee Ultrasound Joint Assessment; Other: Ankle and Knee Magnetic Resonance Imaging |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence and natural progression of subclinical arthropathy (joint changes) in children with severe hemophilia undergoing prophylaxis using a validated ultrasound (HEAD-US) protocol.
Two independent readers will describe all the ultrasonography positive joint findings and/or changes on a standardized spreadsheet; they will also rate and score these changes based on the HEAD-US scale providing a final score for each of the evaluated joints (ankles and knees).
Measured: 5 years
Prevalence and natural progression of subclinical arthropathy (joint changes) in children with mild and/or moderate hemophilia using a validated ultrasound (HEAD-US) protocol.
Two independent readers will describe all the ultrasonography positive joint findings and/or changes on a standardized spreadsheet; they will also rate and score these changes based on the HEAD-US scale providing a final score for each of the evaluated joints (ankles and knees).
Measured: 5 years
Secondary outcomes (1)
Characteristics of subclinical joint changes (time of presentation, natural evolution, etc.) between patients with severe hemophilia and those with mild/moderate hemophilia.
The investigators will describe and compare all the ultrasonography joint findings and/or changes on a standardized spreadsheet in between the two cohort of patients.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2016 (estimated)
- Study start16 Sep 2016
- Primary completion7 Dec 2020
- Study completion7 Dec 2020
Why it stopped: Sponsor funding ended
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by University of MiamiThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Miami
- Sponsor type
- University, hospital or other
- Collaborators
- Shire
- Responsible party
- Principal investigator, Fernando F. Corrales-Medina, University of Miami
- Investigators
- Fernando F. Corrales-Medina, MD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02807753
- Sponsor's protocol ID
- 20151156
- Acronym
- HUP
- Status last verified
- Jan 2021
- First submitted
- 1 Jun 2016
- Last updated
- 22 Jan 2021
- Results posted
- No
Publications
Berntorp E. Joint outcomes in patients with haemophilia: the importance of adherence to preventive regimens. Haemophilia. 2009 Nov;15(6):1219-27. doi: 10.1111/j.1365-2516.2009.02077.x. Epub 2009 Jul 29.
PubMed 19659939Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, Ingram JD, Manco-Johnson ML, Funk S, Jacobson L, Valentino LA, Hoots WK, Buchanan GR, DiMichele D, Recht M, Brown D, Leissinger C, Bleak S, Cohen A, Mathew P, Matsunaga A, Medeiros D, Nugent D, Thomas GA, Thompson AA, McRedmond K, Soucie JM, Austin H, Evatt BL. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007 Aug 9;357(6):535-44. doi: 10.1056/NEJMoa067659.
PubMed 17687129Martinoli C, Della Casa Alberighi O, Di Minno G, Graziano E, Molinari AC, Pasta G, Russo G, Santagostino E, Tagliaferri A, Tagliafico A, Morfini M. Development and definition of a simplified scanning procedure and scoring method for Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US). Thromb Haemost. 2013 Jun;109(6):1170-9. doi: 10.1160/TH12-11-0874. Epub 2013 Apr 4.
PubMed 23571706Sierra Aisa C, Lucia Cuesta JF, Rubio Martinez A, Fernandez Mosteirin N, Iborra Munoz A, Abio Calvete M, Guillen Gomez M, Moreto Quintana A, Rubio Felix D. Comparison of ultrasound and magnetic resonance imaging for diagnosis and follow-up of joint lesions in patients with haemophilia. Haemophilia. 2014 Jan;20(1):e51-7. doi: 10.1111/hae.12268. Epub 2013 Sep 24.
PubMed 24112687Di Minno MN, Iervolino S, Soscia E, Tosetto A, Coppola A, Schiavulli M, Marrone E, Ruosi C, Salvatore M, Di Minno G. Magnetic resonance imaging and ultrasound evaluation of "healthy" joints in young subjects with severe haemophilia A. Haemophilia. 2013 May;19(3):e167-73. doi: 10.1111/hae.12107. Epub 2013 Mar 18.
PubMed 23496145Doria AS. State-of-the-art imaging techniques for the evaluation of haemophilic arthropathy: present and future. Haemophilia. 2010 Jul;16 Suppl 5:107-14. doi: 10.1111/j.1365-2516.2010.02307.x.
PubMed 20590865Muca-Perja M, Riva S, Grochowska B, Mangiafico L, Mago D, Gringeri A. Ultrasonography of haemophilic arthropathy. Haemophilia. 2012 May;18(3):364-8. doi: 10.1111/j.1365-2516.2011.02672.x. Epub 2011 Oct 17.
PubMed 21999202
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Joint Arthropathy Using Ultrasound Technique in Children With Severe Hemophilia Undergoing Prophylaxis Regimen
- ClinicalTrials.gov ID
- NCT02807753
- Acronym / sponsor ID
- HUP / 20151156
- Conditions
- Hemophilia A, Hemophilia B
- Keywords
- Hemophilia, Arthropathy, Pediatric
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor funding ended
- Enrolment
- 18 participants (actual)
- Lead sponsor
- University of MiamiUniversity, hospital or other
- Collaborators
- Shire
- Study start
- 16 Sep 2016 (actual)
- Primary completion
- 7 Dec 2020 (actual)
- Study completion
- 7 Dec 2020 (actual)
- First posted
- 21 Jun 2016 (estimated)
- 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.
To evaluate the prevalence of subclinical arthropathy in children with severe hemophilia undergoing a prophylaxis regimen and without evidence of target joints, using a validated ultrasound scoring method.
Detailed description
Persons with Hemophilia (A or B) often experience recurrent joint bleeds, most commonly affecting the ankles, knees and elbows. These bleeds can lead to significant pain and disability over time. If recurrent joint bleeds are not managed with prompt and adequate infusions of factor concentrate, the damage caused by the presence of blood in the joint space will eventually result in a condition called debilitating chronic hemophilic arthropathy.
The initiation of and adherence to a prophylactic infusion regimen, starting with the first or second joint bleed, is essential for prevention of progression to arthropathy.
Studies have demonstrated that prophylaxis with recombinant or plasma-derived factor VIII or IX concentrates is effective in preventing clinical joint bleeds and the progression to debilitating joint disease in patients with severe hemophilia A or B respectively. However, for patients on prophylaxis, the absence of symptomatic joint bleeds and/or structural and functional abnormalities of joints on physical examination and plain radiographic images can lead to the erroneous assumption that the prophylaxis is completely effective. It has been established that patients with severe hemophilia are still at risk for subclinical bleeding ("microbleeds") despite seemingly adequate prophylaxis.
Young adults, despite a lifetime on prophylaxis and apparently normal joints are developing arthropathy in their 20's and 30's. Prophylaxis as currently practiced may only be delaying the onset of clinical joint disease.
The recent advancements in ultrasound imaging (US) have been proven to be effective in confirming a joint bleed, monitoring the evolution of a joint bleed and assessing the resolution or recurrence of a bleed. Previous studies have evaluated the prevalence of subclinical arthropathy in young hemophilic adults using both US and MRI techniques and concluded that US is as effective and sensitive as MRI identifying these subclinical joint abnormalities.
However, to the investigators' knowledge, no prior studies have used US technique over an extended period of time to monitor the natural evolution of joint arthropathy in children with hemophilia who are adherent to an established prophylaxis regimen and have no evidence of clinical joint compromise.
The seminal Joint Outcome Study, which confirmed the role of prophylaxis in preventing overt clinical joint disease, mandated a trough residual factor VIII level of 1%. While this trough level significantly decreased overt hemarthroses and joint damage, the evidence suggestive of microbleeds raised a question as to the protection afforded by such a low trough level. Intuitively it would seem as if a higher trough level should confer greater protection against microbleeds, and result in more sustainable joint health. The "ideal" protective trough, has not been established.
The investigators' hypothesis is that US is a valuable imaging technique to monitor the natural evolution of hemophilic arthropathy in children with severe hemophilia A or B who are undergoing prophylaxis regimen and do not manifest clinical evidence of hemophilic arthropathy.
Through this observational study, the investigators will provide valuable information in regards the prevalence, progression and severity of joint abnormalities. The use of US to detect microbleeds before the cumulative damaging effects demonstrable by MRI, will also allow tailoring of treatment and the implementation of new prophylaxis strategies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Severe Hemophilia A or B | – | Other: Ankle and Knee Ultrasound Joint Assessment, Other: Ankle and Knee Magnetic Resonance ImagingMedical history, physical exam, vital signs,physical therapist targeted exam and ultrasound joint assessment (ankles and knees) every 6 months. MRI joint assessment (knees and ankles) at End of Trial Visit (Month 60). |
| Mild/Moderate Hemophilia A or B | – | Other: Ankle and Knee Ultrasound Joint Assessment, Other: Ankle and Knee Magnetic Resonance ImagingMedical history, physical exam, vital signs,physical therapist targeted exam and ultrasound joint assessment (ankles and knees) every year. MRI joint assessment (knees and ankles) at End of Trial Visit (Month 60). |
Who may qualify
- Ages
- Up to 30 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Mild, Moderate and Severe Hemophilia A \& B patients.
Key inclusion criteria (2 listed)
- Severe Hemophilia Cohort:Patients from 0 up to 30 months of age with diagnosis of severe hemophilia A or B defined as a factor VIII:C/IX:C of <1% undergoing prophylaxis regimen with any factor VIII or IX concentrate and without evidence (clinical or by history) of target joint disease.
- Mild/moderate Hemophilia Cohort:Patients from 0 up to 30 months of age with diagnosis of mild hemophilia A or B defined as a factor VIII:C/IX:C of 5-50% and those with diagnosis of moderate hemophilia A or B defined a s a factor VIII:C/IX:C of 1-5% without evidence (clinical or by history) of target joint disease and no history of spontaneous joint bleeds.
Key exclusion criteria (4 listed)
- Patients with concomitant Hepatitis B, Hepatitis C and HIV viral infections (because of a recognized arthritogen effect).
- Present or prior history of anti FVIII or IX inhibitors.
- Known inflammatory joint disease.
- Established target joint.
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 |
|---|---|---|
| Ankle and Knee Ultrasound Joint AssessmentUltrasound Examination of the ankles and knees | Other | Mild/Moderate Hemophilia A or B, Severe Hemophilia A or B |
| Ankle and Knee Magnetic Resonance ImagingAnkle and Knee Magnetic Resonance Imaging | Other | Mild/Moderate Hemophilia A or B, Severe Hemophilia A or B |
Primary outcome
Prevalence and natural progression of subclinical arthropathy (joint changes) in children with severe hemophilia undergoing prophylaxis using a validated ultrasound (HEAD-US) protocol.
Time frame: 5 years
Two independent readers will describe all the ultrasonography positive joint findings and/or changes on a standardized spreadsheet; they will also rate and score these changes based on the HEAD-US scale providing a final score for each of the evaluated joints (ankles and knees).
Prevalence and natural progression of subclinical arthropathy (joint changes) in children with mild and/or moderate hemophilia using a validated ultrasound (HEAD-US) protocol.
Time frame: 5 years
Two independent readers will describe all the ultrasonography positive joint findings and/or changes on a standardized spreadsheet; they will also rate and score these changes based on the HEAD-US scale providing a final score for each of the evaluated joints (ankles and knees).
1 secondary outcome
Characteristics of subclinical joint changes (time of presentation, natural evolution, etc.) between patients with severe hemophilia and those with mild/moderate hemophilia.
Time frame: 5 years
Results and safety
The study was stopped early (reason given: Sponsor funding ended). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2021.
- Background: PubMed 19659939Berntorp E. Joint outcomes in patients with haemophilia: the importance of adherence to preventive regimens. Haemophilia. 2009 Nov;15(6):1219-27. doi: 10.1111/j.1365-2516.2009.02077.x. Epub 2009 Jul 29.
- Background: PubMed 17687129Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, Ingram JD, Manco-Johnson ML, Funk S, Jacobson L, Valentino LA, Hoots WK, Buchanan GR, DiMichele D, Recht M, Brown D, Leissinger C, Bleak S, Cohen A, Mathew P, Matsunaga A, Medeiros D, Nugent D, Thomas GA, Thompson AA, McRedmond K, Soucie JM, Austin H, Evatt BL. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007 Aug 9;357(6):535-44. doi: 10.1056/NEJMoa067659.
- Background: PubMed 23571706Martinoli C, Della Casa Alberighi O, Di Minno G, Graziano E, Molinari AC, Pasta G, Russo G, Santagostino E, Tagliaferri A, Tagliafico A, Morfini M. Development and definition of a simplified scanning procedure and scoring method for Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US). Thromb Haemost. 2013 Jun;109(6):1170-9. doi: 10.1160/TH12-11-0874. Epub 2013 Apr 4.
- Background: PubMed 24112687Sierra Aisa C, Lucia Cuesta JF, Rubio Martinez A, Fernandez Mosteirin N, Iborra Munoz A, Abio Calvete M, Guillen Gomez M, Moreto Quintana A, Rubio Felix D. Comparison of ultrasound and magnetic resonance imaging for diagnosis and follow-up of joint lesions in patients with haemophilia. Haemophilia. 2014 Jan;20(1):e51-7. doi: 10.1111/hae.12268. Epub 2013 Sep 24.
- Background: PubMed 23496145Di Minno MN, Iervolino S, Soscia E, Tosetto A, Coppola A, Schiavulli M, Marrone E, Ruosi C, Salvatore M, Di Minno G. Magnetic resonance imaging and ultrasound evaluation of "healthy" joints in young subjects with severe haemophilia A. Haemophilia. 2013 May;19(3):e167-73. doi: 10.1111/hae.12107. Epub 2013 Mar 18.
- Background: PubMed 20590865Doria AS. State-of-the-art imaging techniques for the evaluation of haemophilic arthropathy: present and future. Haemophilia. 2010 Jul;16 Suppl 5:107-14. doi: 10.1111/j.1365-2516.2010.02307.x.
- Background: PubMed 21999202Muca-Perja M, Riva S, Grochowska B, Mangiafico L, Mago D, Gringeri A. Ultrasonography of haemophilic arthropathy. Haemophilia. 2012 May;18(3):364-8. doi: 10.1111/j.1365-2516.2011.02672.x. Epub 2011 Oct 17.
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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.
Fernando F. Corrales-Medina, MD principal investigator
University of Miami
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and Hemophilia B, run by University of Miami at 4 sites 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 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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.
Are results available for this study?
The study was stopped early (reason given: Sponsor funding ended). Results are not available in the source record.
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?
Prevalence and natural progression of subclinical arthropathy (joint changes) in children with severe hemophilia undergoing prophylaxis using a validated ultrasound (HEAD-US) protocol. (time frame: 5 years). The study lists 2 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 NCT02807753, which also lists 7 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 22 Jan 2021.