PhenylketonuriaPhase 3NCT05166161
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
A Phase 3 study testing PTC923 in people with Phenylketonuria, run by PTC Therapeutics at 46 sites in 19 countries.
Trial summary
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Phenylketonuria
- What is being tested?
- PTC923
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 247 participants
- Where
- 46 sitesin 19 countries
- Study dates
- 14 Feb 2022to 21 Jul 2026
- Phase
- Phase 3
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by past medical history of at least 2 blood Phe measurements ≥600 μmol/L.
- Women of childbearing potential must have a negative pregnancy test at screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for up to 90 days after the last dose of the study drug.
- Males who are sexually active with women of childbearing potential who have not had a vasectomy must agree to use a barrier method of birth control during the study and for up to 90 days after the last dose of study drug. Males must also refrain from sperm donations during this time period.
- Willing to continue current diet unchanged while participating in the study (unless specifically instructed to change diet during the study by the investigator).
Who cannot join
- Inability to tolerate oral medication.
- A female who is pregnant or breastfeeding, or considering pregnancy.
- Serious neuropsychiatric illness (for example, major depression) not currently under medical control, that in the opinion of the investigator or PTC, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.
- Past medical history and/or evidence of renal impairment and/or condition including moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60 milliliters [mL]/minute [min] min as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist.
- Any other condition that in the opinion of the investigator or PTC, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.
- Requirement for concomitant treatment with any drug known to inhibit folate synthesis (for example, methotrexate).
Show all 14Show fewer
- Concomitant treatment with tetrahydrobiopterin (BH4) supplementation (for example, sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ).
- Additional criteria for non-feeder participants who did not participate in a feeder study:
- Gastrointestinal disease (such as irritable bowel syndrome, inflammatory bowel disease, chronic gastritis, and peptic ulcer disease, etc) that could affect the absorption of study drug.
- History of gastric surgery, including Roux-en-Y gastric bypass surgery or an antrectomy with vagotomy, or gastrectomy.
- History of allergies or adverse reactions to synthetic BH4 or sepiapterin.
- Any clinically significant laboratory abnormality as determined by the investigator.
- Any abnormal physical examination and/or laboratory findings indicative of signs or symptoms of renal disease, including calculated GFR <60 milliliters (mL)/minute/1.73 square meter (m^2).
- Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive GTP cyclohydrolase I, sepiapterin reductase, quinoid dihydropteridine reductase, or pterin-4-alphacarbinolamine dehydratase genes.
Trial locations and contacts
46 sites in 19 countries.
46 sites in 19 countries
United States7 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism
Gainesville, Florida, 32610
Indiana University School of Medicine Department of Medical and Molecular Genetics
Indianapolis, Indiana, 46202
Icahn School of Medicine at Mount Sinai (ISMMS)
New York, New York, 10009
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
University of Utah
Salt Lake City, Utah, 84108
Turkey (Türkiye)5 sites
Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi
Adana, 01330
Hacettepe University Medical Faculty
Altındağ, Ankara, 06230
Ege University Faculty of Medicine Children Hospital
Bornova, İzmir, 35100
İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi
Fatih, Istanbul, 34098
Gazi Üniversitesi Tıp Fakültesi
Yenimahalle, Ankara, 06500
Australia4 sites
PARC Clinical Research
Adelaide, South Australia, SA 5000
Royal Children's Hospital
Parkville, Victoria, 3052
The Queensland Children's Hospital
South Brisbane, Brisbane
Westmead Hospital
Westmead, New South Wales, 2145
Canada3 sites
Metabolics and Genetics in Calgary (MAGIC) Clinic, Ltd.
Calgary, Alberta, T2E 7Z4
McMaster Children's Hospital Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5
The Hospital for Sick Children University of Toronto Adult Clinic: The Fred A Litwin Family Centre in Genetic Medicine University Health Network & Mt. Sinai Hospital
Toronto, Ontario, M5G 1X8
Germany3 sites
University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE)
Hamburg, 20246
Universitätsklinikum Heidelberg / Zentrum für Kinder- und Jugendmedizin / Sektion für Neuropädiatrie & Stoffwechselmedizin
Heidelberg, 69120
Universitätsklinikum Münster
Münster, 48149
Japan3 sites
PTC Clinical Site 1
Multiple Locations
PTC Clinical Site 2
Multiple Locations
PTC Clinical Site 3
Multiple Locations
Portugal3 sites
CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,
Lisbon, Estremadura, 1649-035
CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria
Lisbon, Estremadura, 1649-035
Centro Hospitalar Universitário Do Porto, Epe
Porto, 4099-001
Spain3 sites
Hospital Sant Joan de Déu
Barcelona, Esplugues de Llobregat, 08950
Hospital Universitario Ramón y Cajal
Madrid, 28034
Hospital Universitario Virgen del Rocío
Seville, 41013
United Kingdom3 sites
Birmingham Children's Hospital NHS Foundation Trust
Birmingham, B4 6NH
UNIVERSITY HOSPITALS BIRMINGHAM NHS Foundation Trust
Birmingham
Great Ormond Street Hospital
London, WC1N 3JH
Brazil2 sites
Hospital de clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
Ribeirão Preto, São Paulo, 14051-140
Italy2 sites
Azienda Ospedaliera-Universita Padova
Padua, Veneto, 35129
Department of Human Neuroscience, Child and Adolescent Neuropsychiatry, Policlinico Umberto I
Rome, 00185
Czechia1 site
Fakultní nemocnice Královské Vinohrady Klinika dětí a dorostu Ambulance pro léčbu PKU a HPA
Prague, 100 34
Denmark1 site
Copenhagen University Hospital, Rigshospitalet
Copenhagen, DK-2100
France1 site
Hôpital Necker-Enfants Malades - Centre de Référence des Maladies Héréditaires du Métabolisme
Paris, 75015
Georgia1 site
Medical Genetics and Laboratory Diagnostics Center
Tbilisi, 0159
Poland1 site
Pomorski Uniwersytet Medyczny w Szczecinie Centrum Wsparcia Badań Klinicznych
Szczecin, 71-252
About this study
Official title: A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
Read the detailed description
Eligible participants are:
Feeder participants: those who have completed a Phase 3 PTC Therapeutics (PTC) sponsored feeder study.
Non-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels <360 μmol/L at study entry.
Non-feeder uncontrolled participants: those who have not completed a feeder study and have blood Phe levels ≥360 μmol/L at study entry.
- Conditions
- Phenylketonuria
- Being tested
- PTC923
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTC923Drug
Also called Sepiapterin, Sephience
PTC923 powder for oral use will be suspended in water or apple juice prior to administration.
| Study group | Role | What they receive |
|---|---|---|
| PTC923 | Experimental | Participants will receive PTC923 7.5 mg/kg (participants 0 to <6 months of age), 15 mg/kg (participants 6 to <12 months of age), 30 mg/kg (participants 12 months to <2 years of age), or 60 mg/kg (participants ≥2 years of age) orally once daily for a minimum of 12 months or until participant experiences lack of efficacy, adverse events (AEs) that lead to discontinuation, withdraws from treatment, or PTC923 is authorized and commercially available in the specific country. Receives: Drug: PTC923 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Treatment-Emergent Adverse Events (TEAEs)
A TEAE is any unfavorable or unintended sign (including an abnormal laboratory finding), symptom or disease in a study participant who is administered study drug in this study
Measured: Baseline up to end of study (up to 4 years)
Change From Baseline in Dietary Phe/Protein Consumption at Week 26, Measured During Phe Tolerance Assessment Period
Phe tolerance is defined as the total amount of dietary Phe (milligrams [mg]/kilogram [kg] per day) ingested while maintaining blood Phe levels within the range of 40 to 360 micromoles (μmol)/liter (L) (defined as ≥40 to <360 μmol/L).
Measured: Baseline, Week 26
Secondary outcomes (7)
Change From Baseline in Quality of Life (QOL) Using Phenylketonuria-Quality of Life (PKU-QOL) Questionnaire at Months 8, 14, 20, 26, 32, and 38
QOL using PKU-QOL questionnaire will be assessed in the subset of participants who are able to complete the PKU-QOL (that is, participants whose primary language is English [British or American], Turkish, Dutch, German, Spanish, Italian, Portuguese, or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to 11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL; ages ≥18 years Adult PKU-QOL).
Measured: Baseline, Months 8, 14, 20, 26, 32, and 38
Change From Baseline in QOL Using the European Quality of Life - 5 Dimensions (EQ-5D) at Months 8, 14, 20, 26, 32, and 38
QOL will be assessed using the EQ-5D (EQ-5D-Y Proxy Version 1 [3 to 7 years]; EQ-5D-Y [8 to 15 years]; EQ-5D-5L ([≥16 years]).
Measured: Baseline, Months 8, 14, 20, 26, 32, and 38
Palatability of PTC923
For participants <5 years of age, palatability will be indirectly assessed by the parent(s)/caregiver(s) of participants using the following: On the basis of the reaction/facial expression of your child, do you think that the medication is (pleasant, not sure, unpleasant)?
Measured: Month 1 Day 1
Acceptability/Ease of Administration of PTC923
For all participants <12 years of age, parent(s)/caregiver(s) will rate the acceptability/ease of administration, with the following question: Do you sometimes have problems in giving the medication to your child because he/she refuses to take it or throws it up? (Yes/No).
Measured: Month 1 Day 1
Taste/Flavor Assessment Using a Facial Hedonic Scale
For participants ≥5 to <18 years of age who are able to comply with the instructions, the participant will rate the taste/flavor using a facial hedonic scale (5=really good; 4=good; 3=not sure; 2=bad; 1=really bad).
Measured: Month 1 Day 1
Plasma Sepiapterin Concentration
Measured: Month 1 Day 1 up to Month 11 Day 1
Plasma BH4 Concentration
Measured: Month 1 Day 1 up to Month 11 Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2021
- Study start14 Feb 2022
- Primary completion21 Jul 2026
- Study completion21 Jul 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for PhenylketonuriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 247 participants (actual)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05166161
- Sponsor's protocol ID
- PTC923-MD-004-PKU
- Other IDs
- 2021-000497-28, 2023-509229-31-00, 2023
- Status last verified
- Aug 2026
- First submitted
- 8 Dec 2021
- Last updated
- 9 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
van Spronsen F, Peters H, Margvelashvili L, Agladze D, Schwartz IVD, Gizewska M, Hamazaki T, Guilder L, MacDonald A, Vijay S, Inwood A, Minami M, Fjellbirkeland O, Lund A, Lah M, Thomas JA, Longo N, Kiykim E, Ishige M, Burlina A, Belanger-Quintana A, Rutsch F, Opladen T, Mungan H, Ezgu F, Bratkovic D, Chakrapani A, Ito T, Guimas A, Zori R, Tchan M, Sacharow S, Oliveira A, Janeiro P, Guerra IC, Plana JC, Yildiz Y, Canda E, Khan A, Vockley J, Breilyn MS, Manti F, Larkin A, Hughes C, Liu E, Gao L, Ingalls K, Smith N, Muntau AC. Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study. Genet Med. 2026 Apr;28(4):101683. doi: 10.1016/j.gim.2026.101683. Epub 2026 Jan 12.
PubMed 41537382
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
- ClinicalTrials.gov ID
- NCT05166161
- Acronym / sponsor ID
- PTC923-MD-004-PKU
- Conditions
- Phenylketonuria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 247 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 14 Feb 2022 (actual)
- Primary completion
- 21 Jul 2026 (actual)
- Study completion
- 21 Jul 2026 (actual)
- First posted
- 21 Dec 2021 (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 main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
Detailed description
Eligible participants are:
Feeder participants: those who have completed a Phase 3 PTC Therapeutics (PTC) sponsored feeder study.
Non-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels <360 μmol/L at study entry.
Non-feeder uncontrolled participants: those who have not completed a feeder study and have blood Phe levels ≥360 μmol/L at study entry.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PTC923 | Experimental | Drug: PTC923Participants will receive PTC923 7.5 mg/kg (participants 0 to <6 months of age), 15 mg/kg (participants 6 to <12 months of age), 30 mg/kg (participants 12 months to <2 years of age), or 60 mg/kg (participants ≥2 years of age) orally once daily for a minimum of 12 months or until participant experiences lack of efficacy, adverse events (AEs) that lead to discontinuation, withdraws from treatment, or PTC923 is authorized and commercially available in the specific country. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by past medical history of at least 2 blood Phe measurements ≥600 μmol/L.
- Women of childbearing potential must have a negative pregnancy test at screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for up to 90 days after the last dose of the study drug.
- Males who are sexually active with women of childbearing potential who have not had a vasectomy must agree to use a barrier method of birth control during the study and for up to 90 days after the last dose of study drug. Males must also refrain from sperm donations during this time period.
- Willing to continue current diet unchanged while participating in the study (unless specifically instructed to change diet during the study by the investigator).
Key exclusion criteria (14 listed)
- Inability to tolerate oral medication.
- A female who is pregnant or breastfeeding, or considering pregnancy.
- Serious neuropsychiatric illness (for example, major depression) not currently under medical control, that in the opinion of the investigator or PTC, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.
- Past medical history and/or evidence of renal impairment and/or condition including moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60 milliliters [mL]/minute [min] min as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist.
- Any other condition that in the opinion of the investigator or PTC, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.
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 |
|---|---|---|
| PTC923Also: Sepiapterin, SephiencePTC923 powder for oral use will be suspended in water or apple juice prior to administration. | Drug | PTC923 |
Primary outcome
Number of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Baseline up to end of study (up to 4 years)
A TEAE is any unfavorable or unintended sign (including an abnormal laboratory finding), symptom or disease in a study participant who is administered study drug in this study
Change From Baseline in Dietary Phe/Protein Consumption at Week 26, Measured During Phe Tolerance Assessment Period
Time frame: Baseline, Week 26
Phe tolerance is defined as the total amount of dietary Phe (milligrams [mg]/kilogram [kg] per day) ingested while maintaining blood Phe levels within the range of 40 to 360 micromoles (μmol)/liter (L) (defined as ≥40 to <360 μmol/L).
7 secondary outcomes
Change From Baseline in Quality of Life (QOL) Using Phenylketonuria-Quality of Life (PKU-QOL) Questionnaire at Months 8, 14, 20, 26, 32, and 38
Time frame: Baseline, Months 8, 14, 20, 26, 32, and 38
Change From Baseline in QOL Using the European Quality of Life - 5 Dimensions (EQ-5D) at Months 8, 14, 20, 26, 32, and 38
Time frame: Baseline, Months 8, 14, 20, 26, 32, and 38
Palatability of PTC923
Time frame: Month 1 Day 1
Acceptability/Ease of Administration of PTC923
Time frame: Month 1 Day 1
Taste/Flavor Assessment Using a Facial Hedonic Scale
Time frame: Month 1 Day 1
Plasma Sepiapterin Concentration
Time frame: Month 1 Day 1 up to Month 11 Day 1
Plasma BH4 Concentration
Time frame: Month 1 Day 1 up to Month 11 Day 1
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 9 Sep 2026.
- Derived from this study: PubMed 41537382van Spronsen F, Peters H, Margvelashvili L, Agladze D, Schwartz IVD, Gizewska M, Hamazaki T, Guilder L, MacDonald A, Vijay S, Inwood A, Minami M, Fjellbirkeland O, Lund A, Lah M, Thomas JA, Longo N, Kiykim E, Ishige M, Burlina A, Belanger-Quintana A, Rutsch F, Opladen T, Mungan H, Ezgu F, Bratkovic D, Chakrapani A, Ito T, Guimas A, Zori R, Tchan M, Sacharow S, Oliveira A, Janeiro P, Guerra IC, Plana JC, Yildiz Y, Canda E, Khan A, Vockley J, Breilyn MS, Manti F, Larkin A, Hughes C, Liu E, Gao L, Ingalls K, Smith N, Muntau AC. Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study. Genet Med. 2026 Apr;28(4):101683. doi: 10.1016/j.gim.2026.101683. Epub 2026 Jan 12.
Related trials for Phenylketonuria
RecruitingPhase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
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
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.
The record lists no contact. Use the official record to reach the sponsor.
46 sites in 19 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing PTC923 in people with Phenylketonuria, run by PTC Therapeutics at 46 sites in 19 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 4 inclusion and 14 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 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in United States, Turkey (Türkiye), Australia, Canada, Germany and 14 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. 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?
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?
Number of Treatment-Emergent Adverse Events (TEAEs) (time frame: Baseline up to end of study (up to 4 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 NCT05166161, which also lists 1 linked publication.
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 9 Sep 2026.