Lysosomal Storage DiseasesPhase 3NCT04283227

OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

A Phase 3 study testing OTL-200 in people with Lysosomal Storage Diseases and Metachromatic Leukodystrophy, run by Orchard Therapeutics at 1 site in Italy.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial summary

Who is it for?: Any age
Conditions the study is for: Lysosomal Storage Diseases, Metachromatic Leukodystrophy
What is being tested?: OTL-200
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 6 participants
Where: 1 siteItaly
Study dates: 17 Jan 2022to 31 Mar 2031
Phase: Phase 3
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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

All the following criteria need to be met:
Documented biochemical and molecular diagnosis of MLD, based on ARSA activity below the normal range and identification of two disease-causing ARSA alleles. Novel mutations will be analyzed with in silico prediction tools and excluded from being known common polymorphisms. In the case of a novel mutation(s), a 24-hour urine collection must show elevated sulfatide levels.
0/R or R/R genotype or a genotype recognized as associated with the LJ variant of MLD.
a) If symptomatic: age at disease onset between ≥7 and <17 years of age (i.e. before their 17th birthday). OR
b) If pre-symptomatic: participant must be <17 years of age at treatment (i.e. before their 17th birthday) AND must have a sibling with a diagnosis of late-juvenile MLD variant based on age at disease onset (≥7 and <17 years of age i.e. before sibling's 17th birthday), with biochemical and molecular diagnosis.
Normal cognitive function as defined by an IQ≥85 on age appropriate cognitive scales.

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a) If the participant is <7 years (i.e. before their 7th birthday): normal motor milestones achievement, normal gross motor function according to chronological age and normal neurological examination (if applicable based on the age of the subject, GMFC-MLD = 0) OR b) If participant is ≥7 years: normal gross motor function or mild gross motor function impairment, defined by a GMFC-MLD 0 or 1 (i.e. patient is able to walk independently).
NOTE: The following will not be exclusionary if present alone: 1.) Seizures 2.) Signs of the disease revealed at instrumental evaluations (Electroneurography [ENG] and brain MR)
If applicable, participant willing and capable of compliance with contraceptive use requirements.
Participant (or if applicable, parent/legal guardian) providing signed informed consent or assent as applicable

Who cannot join

Documented HIV infection (positive HIV RNA and/or anti-p24 antibodies).
Malignant neoplasia (except localised skin cancer) or a documented history of hereditary cancer syndrome. Participants with a prior successfully treated malignancy and a sufficient follow-up to exclude recurrence (based on oncologist opinion) can be included after discussion and approval by the Medical Monitor.
Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML) or other serious haematological disorders.
Patients currently enrolled in other interventional trials
Has previously undergone allogeneic HSPC gene therapy (HSPC-GT) and has evidence of residual cells of donor origin.
Previous gene therapy.

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Has symptomatic herpes zoster, not responsive to specific treatment. NOTE: Participants with a recent history of herpes zoster may be included in the study. In such cases, inclusion, additional monitoring and treatment of the condition must be discussed and approved by the Medical Monitor.
Evidence of active tuberculosis (TB) based upon medical examination, chest imaging and TB testing. Participants with latent tuberculosis, as documented by medical history and/or TB testing may be included in the study if receiving antibiotic prophylaxis (e.g. isoniazid). Inclusion, monitoring and treatment of TB in such participants must be discussed and approved by the Medical Monitor.
Acute or chronic stable Hepatitis B (HBV) as evidenced by positive Hepatitis B surface antigen (HBsAg) test result at screening or within 3 months prior to onset of conditioning and/or positive HBV DNA. NOTE: Participants with positive Hepatitis B core antibody due to prior resolved disease may be enrolled, only if a confirmatory negative HBsAg and negative Hepatitis B DNA test are obtained. Inclusion, monitoring and treatment of hepatitis in such participants must be discussed and approved by the Medical Monitor.
Presence of positive Hepatitis C RNA test result at screening. NOTE: Patients who have previously tested positive for antibodies to hepatitis C can be treated, provided they demonstrate absence of ongoing infection using a nucleic acid test with a limit of quantification of ≤15 international units/ml. Negative test results are required on at least 3 sequential occasions over a period of at least 4 weeks, after completion of treatment for Hepatitis C, with the final test conducted no more than 3 days prior to cell harvest. Inclusion, monitoring and treatment of hepatitis in such participants must be discussed and approved by the Medical Monitor.
End-organ dysfunction, severe active infection not responsive to treatment, or other severe disease or clinical condition which, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
In addition to the potential infections tested per protocol, the PI should consider testing for other transmissible infectious agents listed in the European Union (EU) Cell and Tissue Directive as clinically appropriate and results must be discussed with the Orchard medical monitor prior to stem cell harvest.
Participants with alanine transferase (ALT) >2x upper limit of normal (ULN) or total bilirubin >1.5xULN may be included only after discussed and agreed with the Orchard medical monitor and considered in the context of the criterion for excluding participants with other severe disease. Isolated elevation of total bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35% of total.

Trial locations and contacts

1 site in 1 country.

Italy1 site

Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, 20132

Other trials in Italy

About this study

Official title: An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD).

OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system. The aim of this clinical study is to assess the pharmacodynamic effect and long-term clinical efficacy and safety of OTL-200 in Late Juvenile MLD patients.

Conditions: Lysosomal Storage Diseases, Metachromatic Leukodystrophy
Being tested: OTL-200
Keywords: OTL-200, Cryopreserved formulation, Gene therapy, MLD, late juvenile, atidarsagene autotemcel
Medical subject headings: Lysosomal Storage Diseases, Leukodystrophy, Metachromatic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

OTL-200Genetic
All subjects will receive OTL-200 gene therapy and will be followed up for 8 years following treatment with OTL-200.

Study groups in NCT04283227
Study group	Role	What they receive
OTL-200 Gene Therapy	Experimental	OTL-200 is an autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. Receives: Genetic: OTL-200

What is being measured

The primary outcome is the main question the study is designed to answer.

Evaluation of OTL-200 Arylsulfatase A (ARSA) activity levels in Cerebrospinal Fluid (CSF)
Change from baseline in ARSA activity levels in CSF
Measured: 24 months after treatment
Evaluation of OTL-200 on the neuronal metabolite ratio of N-acetyl-aspartate (NAA) to creatine (Cr) in white matter regions of the brain
Change from baseline in neuronal metabolite ratio of N-acetyl-aspartate (NAA) to creatine (Cr) in white matter regions of interest of the brain
Measured: 24 months after treatment

Secondary outcomes (23)

Change in ARSA activity levels in CSF from baseline
Measured to assess the pharmacodynamic activity of OTL-200 in the Central Nervous System (CNS) post-treatment
Measured: multiple visits up to 8 years post gene-therapy
Change from baseline in neuronal metabolite ratio of NAA: Cr in white matter regions of interest of the brain
Measured to assess the pharmacodynamic activity of OTL-200 in neuronal metabolite ratio of NAA to Cr in white matter regions of interest of the brain post-treatment
Measured: multiple visits up to 8 years post gene-therapy
Change from baseline in ARSA levels in total peripheral blood mononuclear cells (PBMCs)
Measured to assess the pharmacodynamic activity of OTL-200 in circulating total PBMCs post-treatment
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Change from baseline in ARSA levels in PB CD14+
Measured to assess the pharmacodynamic activity of OTL-200 in circulating CD14+ post-treatment
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Change from baseline in ARSA levels in PB CD15+
Measured to assess the pharmacodynamic activity of OTL-200 in circulating CD15+ post-treatment
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Neuronal metabolite ratios as compared to baseline, siblings and/or untreated historical controls (may include but not limited to Cho:Cr, mIns:Cr, Lac: Cr, Cho: NAA, NAA: H2O, Cho: H2O, mIns: H2O, Lac: H2O) in white matter regions of interest
Measured to assess the pharmacodynamic activity of OTL-200 in neuronal metabolite ratios in white matter regions of interest of the brain post-treatment compared to siblings and/or untreated historical controls
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Engraftment as measured by percent lentiviral positive (%LV+) in bone marrow (BM) progenitors
Engraftment of transduced cells will be determined by measuring the percentage of hematopoietic colony-forming cells harboring the integrated vector by quantitative polymerase chain reaction (qPCR).
Measured: At D30 and multiple visits up to 8 years post gene-therapy
Vector copy number (VCN) in BM mononuclear cells
Engraftment of transduced cells will be determined by measuring the VCN per genome in BM-derived cells.
Measured: At D30 and multiple visits up to 8 years post gene-therapy
VCN in Peripheral blood PBMCs
Engraftment of transduced cells will be determined by measuring the VCN per genome in PBMCs.
Measured: At D60 and multiple visits up to 8 years post gene-therapy
Change in severity scale for brain magnetic resonance imaging (MRI)
Brain MRI will be assessed using modified Loes Score and demyelination load on T1w, T2w and FLAIR imaging.
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Change in neurocognitive function
Neurocognitive assessments will use Bayley Scale of Infant and Toddler Development (BSID), Wechsler Preschool and Primary Scale of Intelligence (WPPSI), Wechsler Intelligence Scale for Children (WISC), or Wechsler Adult Intelligence Scale (WAIS) according to the age of the participant to encompass performance, verbal, full scale IQ measures, processing speed and working memory indices
Measured: 24 months and multiple visits up to 8 years post gene-therapy as compared to baseline
Change in full neurological clinical examination (NCE)
Neurological examinations will be performed to evaluate the effect of OTL-200 on the onset or progression of MLD disease.
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Change in Gross Motor Function Classification (GMFC)-MLD
GMFC-MLD will evaluate the change in motor function according to seven clinically relevant levels of walking, sitting, locomotion, trunk and head control. The scoring range is from 0 (walking without support with quality of performance normal for age) to 6 (loss of any locomotion as well as loss of any head and trunk control).
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Change in NCV
NCV will be assessed by electroneurography which is a technique used to test and quantify the nerve conduction and impulse propagation along motor and sensory peripheral nerves.
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Vineland Adaptive Behavior Scale (VABS)
VABS will assess the individual's ability to undertake daily activities appropriate for their age group.
Measured: 24 months and multiple visits up to 8 years post gene-therapy
Conditioning regimen related toxicity and AEs
To evaluate the safety and tolerability of the HSPC GT procedure.
Measured: up to 8 years post gene-therapy
Non-conditioning related AEs
To evaluate the safety and tolerability of OTL-200.
Measured: up to 8 years post gene-therapy
Hematological reconstitution
Hematological reconstitution defined as absolute neutrophil count [ANC] ≥ 500/µL and platelet count ≥20,000 platelets/μL with associated evidence of BM recovery
Measured: By Day 60 post-gene therapy
Incidence of infusion related reactions
To evaluate the safety and tolerability of the HSPC GT procedure.
Measured: up to 8 years post gene-therapy
Immune response (e.g. anti-ARSA antibodies)
Plasma samples will be collected for anti-ARSA antibody analysis
Measured: up to 8 years post gene-therapy
Abnormal Clonal Proliferation (ACP)
Malignancy or ACP due to insertional oncogenesis will be evaluated using different tests and procedures.
Measured: up to 8 years post gene-therapy
Replication Competent Lentivirus (RCL
Molecular monitoring of RCL will be assessed via enzyme-linked immunosorbent assay (ELISA) test for serum human immunodeficiency virus (HIV) p24 antigen. A positive HIV p24 test result is subject to second level testing including: a) DNA PCR for vesicular stomatitis virus G (VSV-G) envelope (PBMC), and b) reverse transcription (RT)-PCR for serum HIV-pol ribonucleic acid (RNA) (plasma).
Measured: baseline, 1, 3, 6, and 12 months, then once a year up to 8 years post gene-therapy
Integration Site analysis findings
Detailed analysis of LV integrations will be performed on PB and BM cells, to monitor the nature and distribution of Integration Sites
Measured: 6, 12 months, then once a year up to 8 years post gene-therapy

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

17 Jan 202231 Mar 2031

First posted25 Feb 2020
Study start17 Jan 2022
Primary completion31 Jan 2026 (estimated)
Study completion31 Mar 2031 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Lysosomal Storage DiseasesStudies 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
Model description: All eligible subjects will receive intravenous (IV) infusion of OTL-200 gene therapy. Subjects will also receive conditioning regimen with busulfan.
Masking: None (open label)
Enrolment: 6 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04283227
Sponsor's protocol ID: OTL-200-07
Status last verified: Aug 2025
First submitted: 13 Feb 2020
Last updated: 5 Sep 2025
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 5 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD).
ClinicalTrials.gov ID: NCT04283227
Acronym / sponsor ID: OTL-200-07
Conditions: Lysosomal Storage Diseases, Metachromatic Leukodystrophy
Keywords: OTL-200, Cryopreserved formulation, Gene therapy, MLD, late juvenile, atidarsagene autotemcel
Phase: Phase 3
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 6 participants (actual)
Lead sponsor: Orchard TherapeuticsIndustry (pharma, biotech, devices)
Collaborators: Ospedale San Raffaele
Study start: 17 Jan 2022 (actual)
Primary completion: 31 Jan 2026 (estimated)
Study completion: 31 Mar 2031 (estimated)
First posted: 25 Feb 2020 (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.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: All eligible subjects will receive intravenous (IV) infusion of OTL-200 gene therapy. Subjects will also receive conditioning regimen with busulfan.

Arm	Type	Receives
OTL-200 Gene Therapy	Experimental	Genetic: OTL-200OTL-200 is an autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (10 listed)

Documented biochemical and molecular diagnosis of MLD, based on ARSA activity below the normal range and identification of two disease-causing ARSA alleles. Novel mutations will be analyzed with in silico prediction tools and excluded from being known common polymorphisms. In the case of a novel mutation(s), a 24-hour urine collection must show elevated sulfatide levels.
0/R or R/R genotype or a genotype recognized as associated with the LJ variant of MLD.
a) If symptomatic: age at disease onset between ≥7 and <17 years of age (i.e. before their 17th birthday). OR
b) If pre-symptomatic: participant must be <17 years of age at treatment (i.e. before their 17th birthday) AND must have a sibling with a diagnosis of late-juvenile MLD variant based on age at disease onset (≥7 and <17 years of age i.e. before sibling's 17th birthday), with biochemical and molecular diagnosis.
Normal cognitive function as defined by an IQ≥85 on age appropriate cognitive scales.

Key exclusion criteria (13 listed)

Documented HIV infection (positive HIV RNA and/or anti-p24 antibodies).
Malignant neoplasia (except localised skin cancer) or a documented history of hereditary cancer syndrome. Participants with a prior successfully treated malignancy and a sufficient follow-up to exclude recurrence (based on oncologist opinion) can be included after discussion and approval by the Medical Monitor.
Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML) or other serious haematological disorders.
Patients currently enrolled in other interventional trials
Has previously undergone allogeneic HSPC gene therapy (HSPC-GT) and has evidence of residual cells of donor origin.

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
OTL-200All subjects will receive OTL-200 gene therapy and will be followed up for 8 years following treatment with OTL-200.	Genetic	OTL-200 Gene Therapy

Primary outcome

Evaluation of OTL-200 Arylsulfatase A (ARSA) activity levels in Cerebrospinal Fluid (CSF)
Time frame: 24 months after treatment
Change from baseline in ARSA activity levels in CSF
Evaluation of OTL-200 on the neuronal metabolite ratio of N-acetyl-aspartate (NAA) to creatine (Cr) in white matter regions of the brain
Time frame: 24 months after treatment
Change from baseline in neuronal metabolite ratio of N-acetyl-aspartate (NAA) to creatine (Cr) in white matter regions of interest of the brain

23 secondary outcomes

Change in ARSA activity levels in CSF from baseline
Time frame: multiple visits up to 8 years post gene-therapy
Change from baseline in neuronal metabolite ratio of NAA: Cr in white matter regions of interest of the brain
Time frame: multiple visits up to 8 years post gene-therapy
Change from baseline in ARSA levels in total peripheral blood mononuclear cells (PBMCs)
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Change from baseline in ARSA levels in PB CD14+
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Change from baseline in ARSA levels in PB CD15+
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Neuronal metabolite ratios as compared to baseline, siblings and/or untreated historical controls (may include but not limited to Cho:Cr, mIns:Cr, Lac: Cr, Cho: NAA, NAA: H2O, Cho: H2O, mIns: H2O, Lac: H2O) in white matter regions of interest
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Engraftment as measured by percent lentiviral positive (%LV+) in bone marrow (BM) progenitors
Time frame: At D30 and multiple visits up to 8 years post gene-therapy
Vector copy number (VCN) in BM mononuclear cells
Time frame: At D30 and multiple visits up to 8 years post gene-therapy
VCN in Peripheral blood PBMCs
Time frame: At D60 and multiple visits up to 8 years post gene-therapy
Change in severity scale for brain magnetic resonance imaging (MRI)
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Change in neurocognitive function
Time frame: 24 months and multiple visits up to 8 years post gene-therapy as compared to baseline
Change in full neurological clinical examination (NCE)
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Change in Gross Motor Function Classification (GMFC)-MLD
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Change in NCV
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Vineland Adaptive Behavior Scale (VABS)
Time frame: 24 months and multiple visits up to 8 years post gene-therapy
Conditioning regimen related toxicity and AEs
Time frame: up to 8 years post gene-therapy
Non-conditioning related AEs
Time frame: up to 8 years post gene-therapy
Hematological reconstitution
Time frame: By Day 60 post-gene therapy
Incidence of infusion related reactions
Time frame: up to 8 years post gene-therapy
Immune response (e.g. anti-ARSA antibodies)
Time frame: up to 8 years post gene-therapy
Abnormal Clonal Proliferation (ACP)
Time frame: up to 8 years post gene-therapy
Replication Competent Lentivirus (RCL
Time frame: baseline, 1, 3, 6, and 12 months, then once a year up to 8 years post gene-therapy
Integration Site analysis findings
Time frame: 6, 12 months, then once a year up to 8 years post gene-therapy

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 5 Sep 2025.

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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.

Orchard Clinical Trials study director

Orchard Therapeutics

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing OTL-200 in people with Lysosomal Storage Diseases and Metachromatic Leukodystrophy, run by Orchard Therapeutics at 1 site in Italy.

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 10 inclusion and 13 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: under way, not looking for participants, as of the sponsor's last update on 5 Sep 2025. 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 Italy. 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?

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?

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?

Evaluation of OTL-200 Arylsulfatase A (ARSA) activity levels in Cerebrospinal Fluid (CSF) (time frame: 24 months after treatment). 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 NCT04283227.

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 5 Sep 2025.