Fibrodysplasia Ossificans ProgressivaFOPalNCT06089616
A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
An observational study of Fibrodysplasia Ossificans Progressiva, run by Ipsen at 5 sites in 2 countries.
Trial summary
The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP). FOP is an ultra-rare, severely disabling disease characterized by new bone formation in areas of the body where bone is not normally present (heterotopic ossification (HO)). Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Fibrodysplasia Ossificans Progressiva
- How many people
- 100 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 5 Dec 2024to 5 Dec 2035
- Phase
- Observational
- 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 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Adults and children with FOP who have been prescribed palovarotene, as per local label, by their treating physician for which informed consent has been obtained and maintained. Participants cannot be included if they are currently participating in a palovarotene clinical trial or an interventional clinical trial for FOP or if they have any contraindication for palovarotene (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).
Who may be able to join
- Adult or child with FOP who have been prescribed palovarotene (prior to and independently of the decision to enroll the patient in this registry study and as per local label) by their treating physician according to the locally approved product information;
- Signed informed consent as per local regulations must be obtained and maintained. Consent/assent from the participant should be obtained as appropriate before any registry study data collection are conducted. If applicable, parents or legally authorized representatives must give signed informed consent.
Who cannot join
- Currently participating in a palovarotene clinical trial;
- Currently participating in any interventional clinical trial for FOP;
- Have any contraindication to palovarotene as per the locally approved label (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).
Trial locations and contacts
5 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 2 countries
Canada3 sites, 2 recruiting
- Recruiting
Edmonton Clinic Health Academy (ECHA)- University of Alberta
Edmonton
- Active, not recruiting
Bone Research and Education Centre
Oakville
- Recruiting
University Health Network (UHN) - Toronto General
Toronto
United States2 sites, 2 recruiting
- Recruiting
The Trustees of the University of Pennsylvania, Office of Clinical Research-Legal Services, Perelman School of Medicine
Philadelphia, Pennsylvania, 19104
- Recruiting
The Regents of the University of California
San Francisco, California, 94103
About this study
Official title: An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients With Fibrodysplasia Ossificans Progressiva (FOP)
The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP).
FOP is an ultra-rare, severely disabling disease characterized by new bone formation in areas of the body where bone is not normally present (heterotopic ossification (HO)).
Read more
HO is often preceded by painful, recurrent episodes of soft tissue swelling (flare-ups).
This registry study will take place in countries where the treatment, known as palovarotene, has been approved for use. Participants will either be treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information) or untreated with palovarotene.
The main aim of this registry study will be to collect and assess real-world safety data on children and adult participants with FOP treated with palovarotene.
This registry study will also describe the effectiveness of palovarotene in exposed participants, including the effect on everyday activities and physical performance.
In addition, this registry study aims to descriptively compare key safety outcomes (i.e. flare-up episodes, growth outcomes, and bone fractures) between participants exposed and unexposed to palovarotene.
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Medical subject headings
- Myositis Ossificans
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Exposed Cohort | Participants treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information) | |
| Unexposed Cohort | Participants untreated with palovarotene |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene
Adverse events will be coded according to MedDRA and will be classified by Preferred Term (PT) and system organ class (SOC).
Measured: From Baseline up to 30 days after the last palovarotene dose.
Percentage of Participants With Serious and Non-serious treatment-related TEAEs
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Measured: From Baseline up to 30 days after the last palovarotene dose.
Percentage of Participants With all serious TEAEs, whether or not they are considered as related to the palovarotene
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Measured: From Baseline up to 30 days after the last palovarotene dose.
Percentage of Participants With nonserious TEAEs whether or not they are considered as related to the palovarotene.
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Measured: From Baseline up to 30 days after the last palovarotene dose.
Secondary outcomes (29)
Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score
The CAJIS is an objective measure of joint movement completed by the Investigator to document total joint involvement. The CAJIS total score is calculated as the sum of the scores of all joints/regions and ranges from 0 (no involvement) to 30 (maximally involved).
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in use of assistive devices
Assistive devices is a questionnaire to assess the assistive devices used by particpants and adaptations for their daily living.
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in percent of worst score for Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) total score
Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) consists of questions which are scored on a scale of 1 to 5, lower scores indicating that the participant has more difficulties.
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC)
The lung function parameters of observed FVC (liters) and percent predicted FVC are obtained by spirometry.
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Forced Expiratory Volume in one second (FEV1)
The lung function parameters of observed FEV1 (liters) and percent predicted FEV1 are obtained by spirometry.
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in absolute and percent predicted FEV1/FVC ratio
The lung function parameters of the absolute and percent predicted FEV1/FVC ratio are obtained by spirometry.
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
The lung function parameters of observed (traditional unit of mL/min/mmHg or SI unit of mmol/min/kPa) and percent predicted DLCO is obtained by the DLCO test.
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) physical and mental function (mean global physical and mental health score converted into T-scores) for participants ≥15 years old
PROMIS Global Health Scale for the adult version, the global physical health and global mental health scores will be calculated as follows:
- Global physical health scores will be calculated as the sum of scores from Questions 3, 6, 7 and 8 and will range from 4 (worse health) to 20 (better health);
- Global mental health scores will be calculated as the sum of scores from Questions 2, 4, 5 and 10 and will range from 4 (worse health) to 20 (better health).
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in PROMIS overall quality of life (QoL) (mean total score converted into T-scores) for participants <15 years old
PROMIS Global Health Scale for the paediatric version, the total score will be calculated as the sum of scores from the first 7 questions and will range from 7 (worse health) to 35 (better health).
If more than 3 questions contributing to the total score are missing, the total score will not be calculated. It will be considered as missing.
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in annualized number of Investigator-reported flare-ups by body location and overall
The annualized number of flare-ups will be derived from the number of flare-ups the participant experienced since last visit.
Measured: From Baseline and every six months up to eleven years.
Number of flare-up outcomes
Number of flare-up outcomes as measured by new bone growth, restricted movement at each visit will be reported.
Measured: From Baseline and every six months up to eleven years.
Flare-up Duration
Flare-up duration will be assessed by body location and overall, at each visit.
Measured: From Baseline and every six months up to eleven years.
Number of flare-ups
Measured: From 12 months before inclusion Baseline up to eleven years.
Movement mobility change
Assessed by key body location: better movement/the same movement/slightly worse movement/moderately worse movement/severely worse movement)
Measured: From Baseline and annually up to eleven years.
Number of locations impacted per participant
Measured: From Baseline and annually up to eleven years.
Evolution of impacted location (how many times a location is impacted) per location
Measured: From Baseline and annually up to eleven years.
Movement mobility outcomes: Percentage of participants with new bone growth overall and by flare-up status
The number of extra bone growths, location of the extra bone growth, whether it was preceded by injury, illness, vaccination and/or other events, whether it was preceded by pain, soft tissue swelling, decreased range of motion, stiffness, redness, or warmth, extra bone growth start and stop date and whether it is ongoing, will be collected.
Measured: From Baseline and every six months up to eleven years.
Percentage of participants with any fractures overall
Assessed by radiological imaging and clinical examination.
Measured: From baseline up to 30 days after the last palovarotene dose.
Change from Baseline in annualized number of fractures by body location and overall
Assessed by radiological imaging and clinical examination.
Measured: From baseline up to 30 days after the last palovarotene dose.
Incidence of pregnancy
A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy and the health status of the baby will be verified
Measured: From Baseline and every month up to 30 days after the last palovarotene dose.
Description of pregnancy characteristics and outcomes
A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy.
Measured: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of pregnancy related Adverse Events (AEs)
A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy.
Measured: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of birth and developmental outcomes through the first year after birth
A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy.
Measured: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of birth and developmental outcomes through the first year after birth
Linear height and knee height velocity will be derived for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached.
Measured: From Baseline and every six months up to eleven years.
Change from Baseline in height z-score
Linear height z-score will be derived for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached.
Measured: From Baseline and every six months up to eleven years.
Mean difference between chronological age and bone age
Bone age (assessment as per the SmPC/PI) and chronological age will be collected for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached.
Measured: From Baseline and every six months up to eleven years.
Frequency of premature physeal closure (PPC) overall
Epiphyseal status ("open" or "closed") (assessment as per the SmPC/PI) will be collected for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached.
Measured: From baseline up to 30 days after the last palovarotene dose
Mean dose/year of palovarotene for participants with FOP exposed and unexposed to palovarotene for chronic treatment
Measured: From Baseline and every six months up to eleven years.
Mean dose/cycle of palovarotene for participants with FOP exposed and unexposed to palovarotene for flare-up treatment
Measured: From Baseline and every six months up to eleven years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2023
- Study start5 Dec 2024
- Primary completion5 Dec 2035 (estimated)
- Study completion5 Dec 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fibrodysplasia Ossificans ProgressivaStudies of the same condition, open and under way.
- More trials run by IpsenThe 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
- Other
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical, Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06089616
- Sponsor's protocol ID
- CLIN-60120-453
- Acronym
- FOPal
- Status last verified
- Oct 2026
- First submitted
- 6 Oct 2023
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients With Fibrodysplasia Ossificans Progressiva (FOP)
- ClinicalTrials.gov ID
- NCT06089616
- Acronym / sponsor ID
- FOPal / CLIN-60120-453
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- 5 Dec 2024 (actual)
- Primary completion
- 5 Dec 2035 (estimated)
- Study completion
- 5 Dec 2035 (estimated)
- First posted
- 18 Oct 2023 (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.
The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP).
FOP is an ultra-rare, severely disabling disease characterized by new bone formation in areas of the body where bone is not normally present (heterotopic ossification (HO)).
HO is often preceded by painful, recurrent episodes of soft tissue swelling (flare-ups).
This registry study will take place in countries where the treatment, known as palovarotene, has been approved for use. Participants will either be treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information) or untreated with palovarotene.
The main aim of this registry study will be to collect and assess real-world safety data on children and adult participants with FOP treated with palovarotene.
This registry study will also describe the effectiveness of palovarotene in exposed participants, including the effect on everyday activities and physical performance.
In addition, this registry study aims to descriptively compare key safety outcomes (i.e. flare-up episodes, growth outcomes, and bone fractures) between participants exposed and unexposed to palovarotene.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exposed Cohort | – | –Participants treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information) |
| Unexposed Cohort | – | –Participants untreated with palovarotene |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Adults and children with FOP who have been prescribed palovarotene, as per local label, by their treating physician for which informed consent has been obtained and maintained. Participants cannot be included if they are currently participating in a palovarotene clinical trial or an interventional clinical trial for FOP or if they have any contraindication for palovarotene (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).
Key inclusion criteria (2 listed)
- Adult or child with FOP who have been prescribed palovarotene (prior to and independently of the decision to enroll the patient in this registry study and as per local label) by their treating physician according to the locally approved product information;
- Signed informed consent as per local regulations must be obtained and maintained. Consent/assent from the participant should be obtained as appropriate before any registry study data collection are conducted. If applicable, parents or legally authorized representatives must give signed informed consent.
Key exclusion criteria (3 listed)
- Currently participating in a palovarotene clinical trial;
- Currently participating in any interventional clinical trial for FOP;
- Have any contraindication to palovarotene as per the locally approved label (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene
Time frame: From Baseline up to 30 days after the last palovarotene dose.
Adverse events will be coded according to MedDRA and will be classified by Preferred Term (PT) and system organ class (SOC).
Percentage of Participants With Serious and Non-serious treatment-related TEAEs
Time frame: From Baseline up to 30 days after the last palovarotene dose.
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Percentage of Participants With all serious TEAEs, whether or not they are considered as related to the palovarotene
Time frame: From Baseline up to 30 days after the last palovarotene dose.
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Percentage of Participants With nonserious TEAEs whether or not they are considered as related to the palovarotene.
Time frame: From Baseline up to 30 days after the last palovarotene dose.
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
29 secondary outcomes
Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in use of assistive devices
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in percent of worst score for Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) total score
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC)
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Forced Expiratory Volume in one second (FEV1)
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in absolute and percent predicted FEV1/FVC ratio
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in observed and percent predicted Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) physical and mental function (mean global physical and mental health score converted into T-scores) for participants ≥15 years old
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in PROMIS overall quality of life (QoL) (mean total score converted into T-scores) for participants <15 years old
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in annualized number of Investigator-reported flare-ups by body location and overall
Time frame: From Baseline and every six months up to eleven years.
Number of flare-up outcomes
Time frame: From Baseline and every six months up to eleven years.
Flare-up Duration
Time frame: From Baseline and every six months up to eleven years.
Number of flare-ups
Time frame: From 12 months before inclusion Baseline up to eleven years.
Movement mobility change
Time frame: From Baseline and annually up to eleven years.
Number of locations impacted per participant
Time frame: From Baseline and annually up to eleven years.
Evolution of impacted location (how many times a location is impacted) per location
Time frame: From Baseline and annually up to eleven years.
Movement mobility outcomes: Percentage of participants with new bone growth overall and by flare-up status
Time frame: From Baseline and every six months up to eleven years.
Percentage of participants with any fractures overall
Time frame: From baseline up to 30 days after the last palovarotene dose.
Change from Baseline in annualized number of fractures by body location and overall
Time frame: From baseline up to 30 days after the last palovarotene dose.
Incidence of pregnancy
Time frame: From Baseline and every month up to 30 days after the last palovarotene dose.
Description of pregnancy characteristics and outcomes
Time frame: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of pregnancy related Adverse Events (AEs)
Time frame: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of birth and developmental outcomes through the first year after birth
Time frame: From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age.
Description of birth and developmental outcomes through the first year after birth
Time frame: From Baseline and every six months up to eleven years.
Change from Baseline in height z-score
Time frame: From Baseline and every six months up to eleven years.
Mean difference between chronological age and bone age
Time frame: From Baseline and every six months up to eleven years.
Frequency of premature physeal closure (PPC) overall
Time frame: From baseline up to 30 days after the last palovarotene dose
Mean dose/year of palovarotene for participants with FOP exposed and unexposed to palovarotene for chronic treatment
Time frame: From Baseline and every six months up to eleven years.
Mean dose/cycle of palovarotene for participants with FOP exposed and unexposed to palovarotene for flare-up treatment
Time frame: From Baseline and every six months up to eleven years.
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 23 Oct 2026.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
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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.
Ipsen Medical, Director study director
Ipsen
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fibrodysplasia Ossificans Progressiva, run by Ipsen at 5 sites in 2 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 2 inclusion and 3 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 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Canada, 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?
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?
Contact Ipsen Clinical Study Enquiries (see email, clinical.trials@ipsen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene (time frame: From Baseline up to 30 days after the last palovarotene dose.). The study lists 4 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 23 Oct 2026.