ATP-Binding Cassette Subfamily C Member 6 DeficiencyPhase 1/2NCT05030831
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
A Phase 1/2 study testing INZ-701 in people with ATP-Binding Cassette Subfamily C Member 6 Deficiency and Pseudoxanthoma Elasticum, run by Inozyme Pharma at 2 sites in 2 countries.
Trial summary
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein, for the treatment of ABCC6 Deficiency. Read more about this study
- Who is it for?
- Ages 18 to 69 years
- Conditions the study is for
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
- What is being tested?
- INZ-701
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 11 Apr 2022to 9 Jul 2024
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 to 69 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Individuals eligible to participate must meet all of the following inclusion criteria:
- Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP)
- Clinical diagnosis of PXE supported by prior genetic identification of biallelic ABCC6 mutations (ie, homozygous or compound heterozygous)
- Male or female, 18 to <70 years of age at Screening
- Plasma PPi <1300 nM at Screening
- Subjects who are being treated with statins or proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors must have been on treatment for at least 6 months prior to Screening and no new anti-lipid therapy can be introduced within 6 months of Screening
Show all 22Show fewer
- Women of child-bearing potential (WOCBP) as defined in Clinical Trials Facilitation and Coordination Group (CTFG 2020) must have a negative serum pregnancy test at Screening
- WOCBP and partners of fertile males who are WOCBP must be using or agree to use one highly effective form of contraception (per CTFG 2020) and a barrier method from at least 1 month before the first dose of INZ-701 through 30 days after the last dose of INZ-701 (greater than 5 half-lives of INZ-701). WOCBP and partners of fertile males who are WOCBP must also agree to not donate ova from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701.
- Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 30 days after the last dose of INZ-701. Males must also agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701.
- In the opinion of the Investigator, must be willing and able to complete the Dose Evaluation Period
- Agree to provide access to relevant medical records
- Individuals who meet any of the following exclusion criteria will not be eligible to participate:
- In the opinion of the Investigator, presence of any clinically significant disease (outside of those considered associated with the diagnosis of ABCC6 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled thyroid, or unrelated connective tissue, bone, mineral, lipid, ophthalmologic, or muscle disease
- Active retinal bleeding in both eyes during Screening
- Clinically significant abnormal laboratory result at Screening in the opinion of the Investigator, including but not limited to, estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2 (Chronic Kidney Disease-Epidemiology Collaboration equation) or 25-hydroxyvitamin D levels <12 ng/mL
- Known active fungal, bacterial, and/or viral infection including human immunodeficiency virus, hepatitis B virus, hepatitis C virus, or COVID-19 infection.
- Malignancy within the last 5 years, except non-melanoma skin cancers or cervical carcinoma in situ
- Known intolerance to INZ-701 or any of its excipients
- Unable to or unwilling to discontinue the use of any prohibited medication as provided in the protocol. Discontinuation should be undertaken only if considered not detrimental and indicated by the subject's treating physician.
- Concurrent participation in another non-Inozyme interventional clinical study and/or receipt of any other investigational new drug within 5 half-lives of the last dose of the other investigational drug or from 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device through completion of participation in the study
- Subjects who are pregnant, trying to become pregnant, or breastfeeding
- Subjects who are trying to father a child
Who cannot join
None listed.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United Kingdom1 site
Richmond Pharmacology Ltd (RPL)
London, London, SE1 1YR
About this study
Official title: A Phase 1/2 Open-Label Multiple Ascending Dose Study of INZ-701 With a Long-Term Extension in Adults With ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE)
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein, for the treatment of ABCC6 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ABCC6 Deficiency.
Read the detailed description
INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein in development for the treatment of ABCC6 Deficiency, a rare genetic disorder.
Study INZ701-201 is a Phase 1/2, multicenter, open-label, multiple ascending dose (MAD), dose-finding study followed by a long-term open-label Extension Period conducted in adults with ABCC6 Deficiency manifesting as Pseudoxanthoma elasticum (PXE). This study is designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701. The goal of the study is to identify a dose and dose schedule (number of doses per week) for further clinical development in ABCC6 Deficiency. No placebo will be used in this study.
Exploratory endpoints for the Extension Period of the study include evaluations of arterial and organ calcification, ophthalmologic, cardiac and renal parameters, and physical function as well as patient reported outcomes.
Subject participation consists of a Screening Period of up to 60 days, a 32-day Dose Evaluation Period, and an Extension Period during which subjects may continue to receive INZ-701 (with options for self-, caregiver-, or healthcare provider administration) until INZ-701 is approved and available in the country where the subject resides or until an alternative study for subjects to continue receiving study drug is available. During the Extension Period, follow-up visits will be conducted every 4 weeks until Week 48 of the Extension Period and then every 12 weeks (remote or in-clinic) until the subject leaves the study.
Subjects will complete a follow up visit 30 days after their last dose of INZ-701.
- Conditions
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
- Being tested
- INZ-701
- Keywords
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, ABCC6, Pseudoxanthoma elasticum, PXE, Generalized Arterial Calcification of Infancy, GACI, hypopyrophosphatemia
- Medical subject headings
- Pseudoxanthoma Elasticum, Arterial calcification of infancy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INZ-701Drug
Also called rhENPP1-Fc
INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc).
| Study group | Role | What they receive |
|---|---|---|
| INZ-701 | Experimental | The study design of the Dose Evaluation Period is a MAD 3 + 3 with 3 dose cohorts.
Based on nonclinical findings and nonclinical pharmacology modeling, the initial planned doses will be 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg, with a twice weekly dose regimen.
During the Extension Period, INZ-701 administration will initially be at the dose and dose schedule assigned in the Dose Evaluation Period; however, the Sponsor may modify the assigned dose and/or dosing regimen in the Extension Period post-Week 48 based on cumulative review of safety and PK/PD data, including population PK.
Study visits will be in-clinic every 4 weeks until Week 48 of the Extension Period and then every 12 weeks (remote or in-clinic) until the subject leaves the study. Subjects will complete an End of Study (EOS) Visit (Safety Follow-up Visit) 30 days after their last dose of INZ-701. Receives: Drug: INZ-701 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) During the Dose Evaluation Period
Number of participants experiencing at least one treatment-emergent adverse event (TEAE) during the Dose Evaluation Period. A TEAE was defined as any adverse event occurring or worsening after the first administration of INZ-701. Participants with one or more TEAEs were counted once regardless of the number of TEAEs experienced.
Measured: Day 1 to Day 32
Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) During the Extension Period
Number of participants experiencing at least one treatment-emergent adverse event (TEAE) during the Extension Period. A TEAE was defined as any adverse event occurring or worsening after the first administration of INZ-701. Participants with one or more TEAEs were counted once regardless of the number of TEAEs experienced. Treatment-emergent adverse events were summarized by NCI-CTCAE grade and relationship to INZ-701.
Measured: Week 8 to Week 48
Secondary outcomes (7)
Number of Participants With Detectable Anti-Drug Antibodies (ADAs) During the Dose Evaluation Period
Number of participants with detectable anti-drug antibodies (ADAs) during the Dose Evaluation Period. Immunogenicity testing utilized a multi-tiered approach, including screening and confirmatory testing to determine ADA specificity and subtype.
Measured: Day 1 to 32 days
Number of Participants With Detectable Anti-Drug Antibodies (ADAs) During the Extension Period
Number of participants with detectable anti-drug antibodies (ADAs) during the Extension Period. Immunogenicity testing utilized a multi-tiered approach, including screening and confirmatory testing to determine ADA specificity and subtype.
Measured: Week 8 to Week 48
Area Under the Plasma Concentration Versus Time Curve (AUC) of INZ-701 During Dose Evaluation Period
Area under the plasma concentration-time curve (AUClast) of INZ-701 calculated from serial plasma concentration measurements obtained following dosing on Day 1 and Day 29.
Measured: Day 1 and Day 29, using serial plasma samples collected from predose through 144 hours post-dose.
Maximum Plasma Concentration (Cmax) of INZ-701 During Dose Evaluation Period
Maximum plasma concentration (Cmax) of INZ-701 calculated from serial plasma concentration measurements obtained following dosing on Day 1 and Day 29.
Measured: Day 1 and Day 29 (Cmax determined from serial plasma samples collected from predose through 144 hours post-dose).
Systemic Clearance of INZ-701 During the Dose Evaluation Period
Systemic clearance of INZ-701 was planned to be estimated from pharmacokinetic assessments on Day 1 and Day 29 using serial plasma samples collected from predose through 144 hours post-dose. A valid terminal phase rate constant could not be calculated because the terminal phase did not meet all prespecified noncompartmental analysis criteria for acceptance. Therefore, systemic clearance could not be reliably estimated.
Measured: Day 1 and Day 29; serial plasma samples were planned from predose through 144 hours post-dose at each time point.
Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels During Dose Evaluation Period
Participant plasma samples were collected at screening, Day 1 to 7; Day 8, 11, 15, 18, 22; Day 29 to 31 and finally Day 32. Pre and post dose sampling was done on Day 1, Day 15 and Day 29. All plasma samples were measured using a sponsor developed assay
Measured: Day 1 to Day 32
Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels During Extension Period
Participant plasma samples were collected pre-dose on Weeks 8, 12, 24, 36 and 48. All plasma samples were measured using a sponsor developed assay
Measured: Week 8 to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2021
- Study start11 Apr 2022
- Primary completion9 Jul 2024
- Study completion9 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for ATP-Binding Cassette Subfamily C Member 6 DeficiencyStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Study INZ701-201 is a Phase 1/2, multicenter, open-label, multiple ascending dose (MAD) study followed by a long-term open-label extension period conducted in adults with ABCC6 Deficiency manifesting as PXE. The study design during the Dose Evaluation Period is a MAD 3+3 with 3 dose cohorts.
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Inozyme Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Inozyme Pharma, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05030831
- Sponsor's protocol ID
- INZ701-201
- Status last verified
- Sep 2026
- First submitted
- 26 Aug 2021
- Last updated
- 13 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Open-Label Multiple Ascending Dose Study of INZ-701 With a Long-Term Extension in Adults With ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE)
- ClinicalTrials.gov ID
- NCT05030831
- Acronym / sponsor ID
- INZ701-201
- Conditions
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
- Keywords
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, ABCC6, Pseudoxanthoma elasticum, PXE, Generalized Arterial Calcification of Infancy, GACI, hypopyrophosphatemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Inozyme PharmaIndustry (pharma, biotech, devices)
- Study start
- 11 Apr 2022 (actual)
- Primary completion
- 9 Jul 2024 (actual)
- Study completion
- 9 Jul 2024 (actual)
- First posted
- 1 Sep 2021 (actual)
- Results
- Posted 13 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein, for the treatment of ABCC6 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ABCC6 Deficiency.
Detailed description
INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein in development for the treatment of ABCC6 Deficiency, a rare genetic disorder.
Study INZ701-201 is a Phase 1/2, multicenter, open-label, multiple ascending dose (MAD), dose-finding study followed by a long-term open-label Extension Period conducted in adults with ABCC6 Deficiency manifesting as Pseudoxanthoma elasticum (PXE). This study is designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701. The goal of the study is to identify a dose and dose schedule (number of doses per week) for further clinical development in ABCC6 Deficiency. No placebo will be used in this study.
Exploratory endpoints for the Extension Period of the study include evaluations of arterial and organ calcification, ophthalmologic, cardiac and renal parameters, and physical function as well as patient reported outcomes.
Subject participation consists of a Screening Period of up to 60 days, a 32-day Dose Evaluation Period, and an Extension Period during which subjects may continue to receive INZ-701 (with options for self-, caregiver-, or healthcare provider administration) until INZ-701 is approved and available in the country where the subject resides or until an alternative study for subjects to continue receiving study drug is available. During the Extension Period, follow-up visits will be conducted every 4 weeks until Week 48 of the Extension Period and then every 12 weeks (remote or in-clinic) until the subject leaves the study.
Subjects will complete a follow up visit 30 days after their last dose of INZ-701.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Study INZ701-201 is a Phase 1/2, multicenter, open-label, multiple ascending dose (MAD) study followed by a long-term open-label extension period conducted in adults with ABCC6 Deficiency manifesting as PXE. The study design during the Dose Evaluation Period is a MAD 3+3 with 3 dose cohorts.
| Arm | Type | Receives |
|---|---|---|
| INZ-701 | Experimental | Drug: INZ-701The study design of the Dose Evaluation Period is a MAD 3 + 3 with 3 dose cohorts. Based on nonclinical findings and nonclinical pharmacology modeling, the initial planned doses will be 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg, with a twice weekly dose regimen. During the Extension Period, INZ-701 administration will initially be at the dose and dose schedule assigned in the Dose Evaluation Period; however, the Sponsor may modify the assigned dose and/or dosing regimen in the Extension Period post-Week 48 based on cumulative review of safety and PK/PD data, including population PK. Study visits will be in-clinic every 4 weeks until Week 48 of the Extension Period and then every 12 weeks (remote or in-clinic) until the subject leaves the study. Subjects will complete an End of Study (EOS) Visit (Safety Follow-up Visit) 30 days after their last dose of INZ-701. |
Who may qualify
- Ages
- 18 to 69 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP)
- Clinical diagnosis of PXE supported by prior genetic identification of biallelic ABCC6 mutations (ie, homozygous or compound heterozygous)
- Male or female, 18 to <70 years of age at Screening
- Plasma PPi <1300 nM at Screening
- Subjects who are being treated with statins or proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors must have been on treatment for at least 6 months prior to Screening and no new anti-lipid therapy can be introduced within 6 months of Screening
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| INZ-701Also: rhENPP1-FcINZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc). | Drug | INZ-701 |
Primary outcome
Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) During the Dose Evaluation Period
Time frame: Day 1 to Day 32
Number of participants experiencing at least one treatment-emergent adverse event (TEAE) during the Dose Evaluation Period. A TEAE was defined as any adverse event occurring or worsening after the first administration of INZ-701. Participants with one or more TEAEs were counted once regardless of the number of TEAEs experienced.
Number of Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) During the Extension Period
Time frame: Week 8 to Week 48
Number of participants experiencing at least one treatment-emergent adverse event (TEAE) during the Extension Period. A TEAE was defined as any adverse event occurring or worsening after the first administration of INZ-701. Participants with one or more TEAEs were counted once regardless of the number of TEAEs experienced. Treatment-emergent adverse events were summarized by NCI-CTCAE grade and relationship to INZ-701.
7 secondary outcomes
Number of Participants With Detectable Anti-Drug Antibodies (ADAs) During the Dose Evaluation Period
Time frame: Day 1 to 32 days
Number of Participants With Detectable Anti-Drug Antibodies (ADAs) During the Extension Period
Time frame: Week 8 to Week 48
Area Under the Plasma Concentration Versus Time Curve (AUC) of INZ-701 During Dose Evaluation Period
Time frame: Day 1 and Day 29, using serial plasma samples collected from predose through 144 hours post-dose.
Maximum Plasma Concentration (Cmax) of INZ-701 During Dose Evaluation Period
Time frame: Day 1 and Day 29 (Cmax determined from serial plasma samples collected from predose through 144 hours post-dose).
Systemic Clearance of INZ-701 During the Dose Evaluation Period
Time frame: Day 1 and Day 29; serial plasma samples were planned from predose through 144 hours post-dose at each time point.
Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels During Dose Evaluation Period
Time frame: Day 1 to Day 32
Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels During Extension Period
Time frame: Week 8 to Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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.
Medical Director, MD study director
Inozyme Pharma, Inc.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing INZ-701 in people with ATP-Binding Cassette Subfamily C Member 6 Deficiency and Pseudoxanthoma Elasticum, run by Inozyme Pharma at 2 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 22 inclusion and 0 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 13 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 2 sites in United States, United Kingdom. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Participants Experiencing at Least One Treatment-Emergent Adverse Event (TEAE) During the Dose Evaluation Period (time frame: Day 1 to Day 32). 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?
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 13 Oct 2026.