PhenylketonuriasTNSPKUNCT03820804
Nutritional Status in Phenylketonuria
An observational study of Phenylketonurias, run by Universidade do Porto at 1 site in Portugal.
Trial summary
In the era of Phenylketonuria (PKU) newborn screening, early diagnosis in the neonatal period and prompt treatment institution has protected patients from developing severe and irreversible mental retardation. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- Diet and Sapropterin
- How many people
- 94 participants
- Where
- 1 sitePortugal
- Study dates
- 7 Jan 2019to 10 Sep 2019
- 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 3 years and older
- All sexes
- Child, Adult, Older adult
Study population: Centro Hospitalar do Porto is one Portuguese Reference Centre for the treatment of inborn errors of metabolism. A cohort of patients with Phenylketonuria is under follow-up with different treatment strategies implemented. All treatment strategies are fully reimbursed by the Portuguese health system and this project aims to evaluate the longitudinal impact of different treatment options on nutritional status outcome.
Who may be able to join
- Diagnosis of PKU.
- With all the clinical data available since 2009.
- Have completed the annual routine nutritional status evaluation in the periods 2009/2010, 2011/2012, 2013/2014, 2015/2016 and 2017/2018.
- Maintaining a follow-up at Centro Hospitalar do Porto.
Who cannot join
- Lost of follow-up.
- Not have completed at least one full evaluation in each time period: 2009/2010, 2011/2012, 2013/2014, 2015/2016 and 2017/2018.
- Any other chronic medical condition which may affect diet or nutritional status.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Trends in Nutritional Status of Patients With Phenylketonuria
In the era of Phenylketonuria (PKU) newborn screening, early diagnosis in the neonatal period and prompt treatment institution has protected patients from developing severe and irreversible mental retardation. The main objective of the treatment is to prevent a chronic elevation of blood Phe concentrations, which together with reduced tyrosine concentrations may increase the risk of neurologic damage. In order to achieve this purpose, the mainstay of treatment is a special diet characterized by a natural protein restriction, supplemented with protein substitutes and special low protein foods.
The requirement to optimize growth and body composition, usually result in dietary prescriptions that are high in carbohydrate (>60% of energy intake), to promote anabolism, considering the synthetic properties of this special diet. Some studies have described a high risk of developing overweight and obesity. Although there is a tendency for a higher incidence in females, it seems that the prevalence in PKU patients follows the same trend as the general population. However, there are limited studies published so far and no longitudinal studies are available describing current practice and its impact on the prevalence of overweight and obesity; neither its consequences in terms of metabolic syndrome or cardiometabolic markers.
Read more
Recently, sapropterin dihydrochloride, which is the synthetic form of Phenylalanine Hydroxylase cofactor, is available in Portugal for patients with PKU. In practice, the sapropterin treated patients increase their natural protein intake, minimizing the synthetic characteristics of the diet. While there is a need for patient re-education about the practicalities of meeting their nutritional needs, scientific evidence about the nutritional status impact of diet liberalization is inadequate.
This study aims to test the following hypothesis:
- Global nutritional status is not significantly affected in patients with PKU under exclusive dietary treatment.
- There is a trend for increased rates of overweight and obesity in patients with PKU from 2009 and we consider this will continue to increase.
- The start of sapropterin treatment allows a higher natural protein intake in patients with PKU that significantly targets nutritional status in at least one of its components (anthropometry, body composition or biochemistry).
Read the detailed description
At the Centro Hospitalar do Porto (Reference Centre for the treatment of Inherited Metabolic Diseases) the annual nutritional status evaluation routinely generates data on anthropometry, body composition, blood pressure, nutritional intake and clinical biochemistry. Since 2009, all this information is recorded for all the patients with PKU under follow-up.
For the purpose of this project, data will be collected, from 2009 until 2018, using a minimum of 5 annual nutritional status evaluations per patient. The project will be able to compare the longitudinal evolution of nutritional status in exclusively diet treated patients (period between 2009 and 2014) with a subgroup of patients already under sapropterin treatment in the period between 2015 and 2018. Genotype is available for all patients with PKU under follow-up at Centro Hospitalar do Porto. However, the decision for starting sapropterin treatment was based on the results from a sapropterin-loading test protocol, approved in 2014 by the Portuguese Society of Metabolic Disorders (not published).
For every PKU patient under follow-up at Centro Hospitalar do Porto, the information about nutritional intake is recorded in a special file that contains all the diet details collected in every appointment. In that way, for the purpose of the annual nutritional status evaluation, every PKU patient is submitted to anthropometric and body composition evaluations, blood pressure assessment and blood samples collection for completing the hematological and biochemical measurements. Also in the same day, at the nutrition appointment, a dietary assessment is done, in order to allow further nutrition adjustments when needed. This approach allows us to know the precise nutritional ingestion in the period before all the measurements.
Data will be collected from patient databases or patient records. In the period between 2009 and 2014 there are no treatments or interventions to which the patients can be randomly assigned, as no specific treatment is the focus of the project. In the period between 2015 and 2018, patients will be divided in two groups based on treatment modalities: diet-treated only or sapropterin treatment. However, their assignment in each group does not have any influence of the project itself. The inclusion in one of the two groups will be decided based on the results of the sapropterin loading test done previously within the clinical routine protocol. Patients will be identified only by a number and only medical data will be collected. No directly or indirectly nominative data will be collected and data will be anonymous.
- Conditions
- Phenylketonurias
- Being tested
- Diet, Sapropterin
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DietOther
Also called Dietary treatment
A specific dietary treatment for patients with Phenylketonuria.
SapropterinDrug
Also called Tetrahydrobiopterin
A pharmacological treatment for patients with Phenylketonuria, used alone or in combination with dietary treatment.
| Study group | Role | What they receive |
|---|---|---|
| Diet-treated | Patients with Phenylketonuria under dietary treatment. Receives: Other: Diet | |
| Sapropterin-treated | Patients with Phenylketonuria under sapropterin treatment. Receives: Drug: Sapropterin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overweight and obesity prevalence
To determine the prevalence of overweight and obesity.
Measured: 2009-2018
Overweight and obesity incidence
To determine the incidence of overweight and obesity.
Measured: 2009-2018
Secondary outcomes (6)
Metabolic syndrome prevalence
To determine the change of the prevalence of metabolic syndrome in patients.
Measured: 2009-2018
Metabolic syndrome incidence
To determine the incidence of metabolic syndrome in patients.
Measured: 2009-2018
Body composition using bioelectrical impedance analysis
To describe the body composition evolution (fat mass, fat-free mass and phase angle) throughout the 10 years of the study.
Measured: 2009-2018
Metabolic control (blood phenylalanine concentration)
To describe the evolution of metabolic control in patients.
Measured: 2009-2018
Metabolic control (blood phenylalanine concentration) and sapropterin
To describe the evolution of metabolic control (blood phenylalanine concentrations) in patients under sapropterin treatment during the period between 2015 and 2018.
Measured: 2015-2018
Phenylalanine intake and sapropterin
To understand the impact of sapropterin treatment on phenylalanine intake (mg of phenylalanine/day) during the period between 2015 and 2018.
Measured: 2015-2018
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2019
- Study start7 Jan 2019
- Primary completion10 Sep 2019
- Study completion10 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for PhenylketonuriasStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universidade do Porto
- Sponsor type
- University, hospital or other
- Collaborators
- Centro Hospitalar do Porto, BioMarin Pharmaceutical
- Responsible party
- Sponsor
- Investigators
- Júlio C Rocha, PhD, Principal investigator, Centro Hospitalar do Porto
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03820804
- Sponsor's protocol ID
- Nutrition_PKU
- Acronym
- TNSPKU
- Status last verified
- Dec 2018
- First submitted
- 19 Oct 2018
- Last updated
- 10 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: A detailed list of the variables to be collected can be shared with other researchers.
Publications
MacDonald A, Rocha JC, van Rijn M, Feillet F. Nutrition in phenylketonuria. Mol Genet Metab. 2011;104 Suppl:S10-8. doi: 10.1016/j.ymgme.2011.08.023. Epub 2011 Sep 2.
PubMed 21944460Rocha JC, van Spronsen FJ, Almeida MF, Soares G, Quelhas D, Ramos E, Guimaraes JT, Borges N. Dietary treatment in phenylketonuria does not lead to increased risk of obesity or metabolic syndrome. Mol Genet Metab. 2012 Dec;107(4):659-63. doi: 10.1016/j.ymgme.2012.10.006. Epub 2012 Oct 16.
PubMed 23137570Dokoupil K, Gokmen-Ozel H, Lammardo AM, Motzfeldt K, Robert M, Rocha JC, van Rijn M, Ahring K, Belanger-Quintana A, MacDonald A. Optimising growth in phenylketonuria: current state of the clinical evidence base. Clin Nutr. 2012 Feb;31(1):16-21. doi: 10.1016/j.clnu.2011.09.001. Epub 2011 Sep 29.
PubMed 21959353Rocha JC, MacDonald A, Trefz F. Is overweight an issue in phenylketonuria? Mol Genet Metab. 2013;110 Suppl:S18-24. doi: 10.1016/j.ymgme.2013.08.012. Epub 2013 Aug 31.
PubMed 24055312MacDonald A, Ahring K, Dokoupil K, Gokmen-Ozel H, Lammardo AM, Motzfeldt K, Robert M, Rocha JC, van Rijn M, Belanger-Quintana A. Adjusting diet with sapropterin in phenylketonuria: what factors should be considered? Br J Nutr. 2011 Jul;106(2):175-82. doi: 10.1017/S0007114511000298.
PubMed 21466737Rocha JC, van Spronsen FJ, Almeida MF, Ramos E, Guimaraes JT, Borges N. Early dietary treated patients with phenylketonuria can achieve normal growth and body composition. Mol Genet Metab. 2013;110 Suppl:S40-3. doi: 10.1016/j.ymgme.2013.10.009. Epub 2013 Oct 22.
PubMed 24183791Gokmen Ozel H, Ahring K, Belanger-Quintana A, Dokoupil K, Lammardo AM, Robert M, Rocha JC, Almeida MF, van Rijn M, MacDonald A. Overweight and obesity in PKU: The results from 8 centres in Europe and Turkey. Mol Genet Metab Rep. 2014 Nov 16;1:483-486. doi: 10.1016/j.ymgmr.2014.11.003. eCollection 2014.
PubMed 27896128Rocha JC, van Rijn M, van Dam E, Ahring K, Belanger-Quintana A, Dokoupil K, Gokmen Ozel H, Lammardo AM, Robert M, Heidenborg C, MacDonald A. Weight Management in Phenylketonuria: What Should Be Monitored. Ann Nutr Metab. 2016;68(1):60-5. doi: 10.1159/000442304. Epub 2015 Nov 25.
PubMed 26598928
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Trends in Nutritional Status of Patients With Phenylketonuria
- ClinicalTrials.gov ID
- NCT03820804
- Acronym / sponsor ID
- TNSPKU / Nutrition_PKU
- Conditions
- Phenylketonurias
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- Universidade do PortoUniversity, hospital or other
- Collaborators
- Centro Hospitalar do Porto, BioMarin Pharmaceutical
- Study start
- 7 Jan 2019 (actual)
- Primary completion
- 10 Sep 2019 (actual)
- Study completion
- 10 Sep 2019 (actual)
- First posted
- 29 Jan 2019 (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.
In the era of Phenylketonuria (PKU) newborn screening, early diagnosis in the neonatal period and prompt treatment institution has protected patients from developing severe and irreversible mental retardation. The main objective of the treatment is to prevent a chronic elevation of blood Phe concentrations, which together with reduced tyrosine concentrations may increase the risk of neurologic damage. In order to achieve this purpose, the mainstay of treatment is a special diet characterized by a natural protein restriction, supplemented with protein substitutes and special low protein foods.
The requirement to optimize growth and body composition, usually result in dietary prescriptions that are high in carbohydrate (>60% of energy intake), to promote anabolism, considering the synthetic properties of this special diet. Some studies have described a high risk of developing overweight and obesity. Although there is a tendency for a higher incidence in females, it seems that the prevalence in PKU patients follows the same trend as the general population. However, there are limited studies published so far and no longitudinal studies are available describing current practice and its impact on the prevalence of overweight and obesity; neither its consequences in terms of metabolic syndrome or cardiometabolic markers.
Recently, sapropterin dihydrochloride, which is the synthetic form of Phenylalanine Hydroxylase cofactor, is available in Portugal for patients with PKU. In practice, the sapropterin treated patients increase their natural protein intake, minimizing the synthetic characteristics of the diet. While there is a need for patient re-education about the practicalities of meeting their nutritional needs, scientific evidence about the nutritional status impact of diet liberalization is inadequate.
This study aims to test the following hypothesis:
- Global nutritional status is not significantly affected in patients with PKU under exclusive dietary treatment.
- There is a trend for increased rates of overweight and obesity in patients with PKU from 2009 and we consider this will continue to increase.
- The start of sapropterin treatment allows a higher natural protein intake in patients with PKU that significantly targets nutritional status in at least one of its components (anthropometry, body composition or biochemistry).
Detailed description
At the Centro Hospitalar do Porto (Reference Centre for the treatment of Inherited Metabolic Diseases) the annual nutritional status evaluation routinely generates data on anthropometry, body composition, blood pressure, nutritional intake and clinical biochemistry. Since 2009, all this information is recorded for all the patients with PKU under follow-up.
For the purpose of this project, data will be collected, from 2009 until 2018, using a minimum of 5 annual nutritional status evaluations per patient. The project will be able to compare the longitudinal evolution of nutritional status in exclusively diet treated patients (period between 2009 and 2014) with a subgroup of patients already under sapropterin treatment in the period between 2015 and 2018. Genotype is available for all patients with PKU under follow-up at Centro Hospitalar do Porto. However, the decision for starting sapropterin treatment was based on the results from a sapropterin-loading test protocol, approved in 2014 by the Portuguese Society of Metabolic Disorders (not published).
For every PKU patient under follow-up at Centro Hospitalar do Porto, the information about nutritional intake is recorded in a special file that contains all the diet details collected in every appointment. In that way, for the purpose of the annual nutritional status evaluation, every PKU patient is submitted to anthropometric and body composition evaluations, blood pressure assessment and blood samples collection for completing the hematological and biochemical measurements. Also in the same day, at the nutrition appointment, a dietary assessment is done, in order to allow further nutrition adjustments when needed. This approach allows us to know the precise nutritional ingestion in the period before all the measurements.
Data will be collected from patient databases or patient records. In the period between 2009 and 2014 there are no treatments or interventions to which the patients can be randomly assigned, as no specific treatment is the focus of the project. In the period between 2015 and 2018, patients will be divided in two groups based on treatment modalities: diet-treated only or sapropterin treatment. However, their assignment in each group does not have any influence of the project itself. The inclusion in one of the two groups will be decided based on the results of the sapropterin loading test done previously within the clinical routine protocol. Patients will be identified only by a number and only medical data will be collected. No directly or indirectly nominative data will be collected and data will be anonymous.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Diet-treated | – | Other: DietPatients with Phenylketonuria under dietary treatment. |
| Sapropterin-treated | – | Drug: SapropterinPatients with Phenylketonuria under sapropterin treatment. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Centro Hospitalar do Porto is one Portuguese Reference Centre for the treatment of inborn errors of metabolism. A cohort of patients with Phenylketonuria is under follow-up with different treatment strategies implemented. All treatment strategies are fully reimbursed by the Portuguese health system and this project aims to evaluate the longitudinal impact of different treatment options on nutritional status outcome.
Key inclusion criteria (4 listed)
- Diagnosis of PKU.
- With all the clinical data available since 2009.
- Have completed the annual routine nutritional status evaluation in the periods 2009/2010, 2011/2012, 2013/2014, 2015/2016 and 2017/2018.
- Maintaining a follow-up at Centro Hospitalar do Porto.
Key exclusion criteria (3 listed)
- Lost of follow-up.
- Not have completed at least one full evaluation in each time period: 2009/2010, 2011/2012, 2013/2014, 2015/2016 and 2017/2018.
- Any other chronic medical condition which may affect diet or nutritional status.
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 |
|---|---|---|
| DietAlso: Dietary treatmentA specific dietary treatment for patients with Phenylketonuria. | Other | Diet-treated |
| SapropterinAlso: TetrahydrobiopterinA pharmacological treatment for patients with Phenylketonuria, used alone or in combination with dietary treatment. | Drug | Sapropterin-treated |
Primary outcome
Overweight and obesity prevalence
Time frame: 2009-2018
To determine the prevalence of overweight and obesity.
Overweight and obesity incidence
Time frame: 2009-2018
To determine the incidence of overweight and obesity.
6 secondary outcomes
Metabolic syndrome prevalence
Time frame: 2009-2018
Metabolic syndrome incidence
Time frame: 2009-2018
Body composition using bioelectrical impedance analysis
Time frame: 2009-2018
Metabolic control (blood phenylalanine concentration)
Time frame: 2009-2018
Metabolic control (blood phenylalanine concentration) and sapropterin
Time frame: 2015-2018
Phenylalanine intake and sapropterin
Time frame: 2015-2018
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2019.
- Background: PubMed 21944460MacDonald A, Rocha JC, van Rijn M, Feillet F. Nutrition in phenylketonuria. Mol Genet Metab. 2011;104 Suppl:S10-8. doi: 10.1016/j.ymgme.2011.08.023. Epub 2011 Sep 2.
- Background: PubMed 23137570Rocha JC, van Spronsen FJ, Almeida MF, Soares G, Quelhas D, Ramos E, Guimaraes JT, Borges N. Dietary treatment in phenylketonuria does not lead to increased risk of obesity or metabolic syndrome. Mol Genet Metab. 2012 Dec;107(4):659-63. doi: 10.1016/j.ymgme.2012.10.006. Epub 2012 Oct 16.
- Background: PubMed 21959353Dokoupil K, Gokmen-Ozel H, Lammardo AM, Motzfeldt K, Robert M, Rocha JC, van Rijn M, Ahring K, Belanger-Quintana A, MacDonald A. Optimising growth in phenylketonuria: current state of the clinical evidence base. Clin Nutr. 2012 Feb;31(1):16-21. doi: 10.1016/j.clnu.2011.09.001. Epub 2011 Sep 29.
- Background: PubMed 24055312Rocha JC, MacDonald A, Trefz F. Is overweight an issue in phenylketonuria? Mol Genet Metab. 2013;110 Suppl:S18-24. doi: 10.1016/j.ymgme.2013.08.012. Epub 2013 Aug 31.
- Background: PubMed 21466737MacDonald A, Ahring K, Dokoupil K, Gokmen-Ozel H, Lammardo AM, Motzfeldt K, Robert M, Rocha JC, van Rijn M, Belanger-Quintana A. Adjusting diet with sapropterin in phenylketonuria: what factors should be considered? Br J Nutr. 2011 Jul;106(2):175-82. doi: 10.1017/S0007114511000298.
- Background: PubMed 24183791Rocha JC, van Spronsen FJ, Almeida MF, Ramos E, Guimaraes JT, Borges N. Early dietary treated patients with phenylketonuria can achieve normal growth and body composition. Mol Genet Metab. 2013;110 Suppl:S40-3. doi: 10.1016/j.ymgme.2013.10.009. Epub 2013 Oct 22.
- Background: PubMed 27896128Gokmen Ozel H, Ahring K, Belanger-Quintana A, Dokoupil K, Lammardo AM, Robert M, Rocha JC, Almeida MF, van Rijn M, MacDonald A. Overweight and obesity in PKU: The results from 8 centres in Europe and Turkey. Mol Genet Metab Rep. 2014 Nov 16;1:483-486. doi: 10.1016/j.ymgmr.2014.11.003. eCollection 2014.
- Background: PubMed 26598928Rocha JC, van Rijn M, van Dam E, Ahring K, Belanger-Quintana A, Dokoupil K, Gokmen Ozel H, Lammardo AM, Robert M, Heidenborg C, MacDonald A. Weight Management in Phenylketonuria: What Should Be Monitored. Ann Nutr Metab. 2016;68(1):60-5. doi: 10.1159/000442304. Epub 2015 Nov 25.
- Individual participant dataSharing planned: Undecided. A detailed list of the variables to be collected can be shared with other researchers.
Related trials for Phenylketonurias
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria
Not yet recruiting
Market Research of a Slow Release Protein Substitute for the Dietary Management of PKU
Phenylketonuria (PKU) and Hyperphenylalaninemia, Brain Diseases, Metabolic, Inborn
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.
Júlio C Rocha, PhD principal investigator
Centro Hospitalar do Porto
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Phenylketonurias, run by Universidade do Porto at 1 site in Portugal.
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 4 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: finished, as of the sponsor's last update on 10 Sep 2019. 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 Portugal. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Overweight and obesity prevalence (time frame: 2009-2018). 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 NCT03820804, which also lists 8 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 10 Sep 2019; AzTrials last refreshed its copy on 27 Oct 2026.