Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyPhase 1/2NCT04686175

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

A Phase 1/2 study testing INZ-701 in people with Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency and Autosomal Recessive Hypophosphatemic Rickets, run by Inozyme Pharma at 7 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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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) enzyme replacement therapy, for the treatment of ENPP1 Deficiency. Read more about this study

Who is it for?: Ages 18 to 64 years
Conditions the study is for: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, 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: 13 participants
Where: 7 sitesin 5 countries
Study dates: 21 Nov 2021to 13 Dec 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 64 years
All sexes
No healthy volunteers
Adult

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 ENPP1 Deficiency supported by prior identification of biallelic ENPP1 mutations (ie, homozygous or compound heterozygous)
Male or female, 18 to <65 years of age at Screening
PPi <1300 nM at Screening
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 1 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 ENPP1 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled cardiovascular, thyroid disease, or unrelated connective tissue, bone, mineral, lipid, or muscle disease
Clinically significant abnormal laboratory result at Screening in the opinion of the Investigator, including but not limited to screening laboratory results demonstrating
estimated glomerular filtration rate (eGFR) (Chronic Kidney Disease-Epidemiology Collaboration [CKD-EPI] equation) < 60 mL/min/1.73m^2,
25-hydroxyvitamin D (25[OH]D) levels <12 ng/mL, or
Intact parathyroid hormone (PTH) >40% above the upper limit of normal
Known active fungal, bacterial, and/or viral infection including human immunodeficiency virus, hepatitis B virus, hepatitis C virus, or COVID-19 virus. In Germany and France, a negative COVID-19 test result is required within 5 days prior to the first dose of INZ-701.
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 or unwilling to discontinue the use of any prohibited medication (examples include 1,25-dihydroxy vitamin D, phosphate, anti-FGF23 [eg, burosumab], calcimimetics, calcium-containing antacids, systemic corticosteroids, PTH suppressors). 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 product 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

Trial locations and contacts

7 sites in 5 countries.

Country

City, postcode or hospital

7 sites in 5 countries

United States2 sites

Clinilabs Drug Development Corporation
Eatontown, New Jersey, 07724
Mayo Clinic
Rochester, Minnesota, 55905

Other trials in United States

Germany2 sites

Parexel International GmbH
Berlin, State of Berlin, 14050
University of Hamburg (Universitatklinikum Hamburg-Eppendorf)
Hamburg, Free and Hanseatic City of Hamburg, 22529

Other trials in Germany

Canada1 site

University of Saskatchewan
Saskatoon, Saskatchewan, S7N 0W8

Other trials in Canada

France1 site

Necker University Hospital-Sick Children
Paris, Paris, 75015

Other trials in France

United Kingdom1 site

Richmond Pharmacology (RPL)
London, London Bridge, SE1 1YR

Other trials in United Kingdom

About this study

Official title: A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults With ENPP1 Deficiency

Read the detailed description

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy (ERT) in development for the treatment of ENPP1 Deficiency, an ultra-rare genetic disorder with an incidence of 1 in 64,000 pregnancies.

Study INZ701-101 is a Phase 1/2, multicenter, open-label, FIH, MAD, dose-finding study followed by a long-term open-label Extension Period conducted in adults with ENPP1 Deficiency. 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. No placebo will be used in the study. Subjects will be 18 to <65 years of age, with a confirmed genetic diagnosis of ENPP1 Deficiency and biochemical evidence of hypopyrophosphatemia (ie, PPi <1300 nM). Exploratory endpoints for the Extension Period of the study include evaluations of skeletal assessment (X-ray and DEXA), arterial and organ calcification (either Na18F-PET/CT or low dose CT [full body] without contrast, echocardiogram, and renal ultrasound), and cardiovascular function (echocardiogram) as well as patient reported outcomes.

Subject participation consists of a Screening Period of up to 30 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, followed by every 12 weeks 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.

Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy
Being tested: INZ-701
Keywords: ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency, hypopyrophosphatemia, ENPP1, Generalized Arterial Calcification of Infancy, GACI, Autosomal Recessive Hypophosphatemic Rickets Type 2, ARHR2
Medical subject headings: Hypophosphatemic Rickets, Autosomal Recessive, 1, 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
INZ701-101 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.

Study groups in NCT04686175
Study group	Role	What they receive
INZ-701	Experimental	The study design of the Dose Evaluation Period is a MAD 3 + 3 dose cohort. 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 (Cohorts 1-3) and 1.2 mg/kg once weekly dose regimen (Cohort 4). 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 With Treatment-Emergent Adverse Events (TEAEs) During the Dose Evaluation Period
Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.
Measured: 32 days
Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) During the Open-label Extension Period
Treatment-emergent adverse events (TEAEs) were defined as adverse events with an onset date on or after the first dose of INZ-701 and within 30 days after the last dose of INZ-701. For this outcome measure, participants with at least one TEAE occurring during the Open-label Extension Period through Week 48 were reported.
Measured: From the start of the Open-label Extension Period (Day 32) through Week 48
Number of Participants With Anti-Drug Antibodies (ADA) During the Dose Evaluation Period
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
Measured: 32 days
Number of Participants With at Least One Positive Anti-Drug Antibody (ADA) Result During the Open-label Extension Period
The presence of anti-drug antibodies (ADAs) was assessed in each participant. Participants with at least one positive ADA result during the Open-label Extension Period through Week 48 were reported. Samples with detected ADAs underwent further assessment, as applicable.
Measured: From entry into the Open-label Extension Period following completion of the 32-day Dose Evaluation Period through Week 48
Area Under the Plasma Concentration-Time Curve From 0 to 72 Hours (AUC0-72h) of INZ-701 Following the Day 1 Dose
Plasma samples were collected from predose through 72 hours after the Day 1 dose to determine the area under the plasma concentration-time curve from time zero to 72 hours postdose (AUC0-72h) of INZ-701.
Measured: Day 1 predose through 72 hours postdose
Maximum Plasma Concentration (Cmax) of INZ-701 Following the Day 1 Dose
Maximum plasma concentration (Cmax) was obtained directly from the observed INZ-701 plasma concentration-time data following the Day 1 dose.
Measured: Day 1 predose through Day 8 predose, up to 168 hours postdose
Apparent Systemic Clearance (CL/F) of INZ-701 Following the Day 1 Dose
Apparent systemic clearance after subcutaneous dosing (CL/F) was calculated as the administered dose divided by the area under the plasma concentration-time curve from time zero to infinity (AUCinf). AUCinf requires estimation of the terminal elimination rate constant (λz). If λz could not be reliably estimated, AUCinf and consequently CL/F could not be reliably determined.
Measured: Day 1 predose through Day 8 predose, up to 168 hours postdose
Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels at Day 32/Early Termination
Plasma inorganic pyrophosphate (PPi) concentrations were measured at Baseline and at the Day 32/Early Termination predose assessment. Change from Baseline was calculated as the Day 32/Early Termination PPi concentration minus the Baseline PPi concentration.
Measured: Baseline to Day 32/Early Termination
Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels at Week 48 of the Open-label Extension Period
Plasma inorganic pyrophosphate (PPi) concentrations were measured at Baseline and during the Open-label Extension Period. Change from Baseline at Week 48 was calculated for each participant as the Week 48 PPi concentration minus the Baseline PPi concentration.
Measured: Baseline to Week 48 of the Open-label Extension Period

Timeline

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

21 Nov 202113 Dec 2024

First posted28 Dec 2020
Study start21 Nov 2021
Primary completion29 Oct 2024
Study completion13 Dec 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 Ectonucleotide Pyrophosphatase/phosphodiesterase1 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 INZ701-101 is a Phase 1/2, multi-center, open-label, first-in-human (FIH), multiple ascending dose (MAD) study followed by a long-term open-label extension period conducted in adults with ENPP1 Deficiency. The study design during the Dose Evaluation Period is a MAD 3+3 with 3 dose cohorts.
Masking: None (open label)
Enrolment: 13 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04686175
Sponsor's protocol ID: INZ701-101
Other IDs: 2020-003716-27
Status last verified: Oct 2026
First submitted: 22 Dec 2020
Last updated: 27 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 27 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 to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults With ENPP1 Deficiency
ClinicalTrials.gov ID: NCT04686175
Acronym / sponsor ID: INZ701-101
Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy
Keywords: ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency, hypopyrophosphatemia, ENPP1, Generalized Arterial Calcification of Infancy, GACI, Autosomal Recessive Hypophosphatemic Rickets Type 2, ARHR2
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 13 participants (actual)
Lead sponsor: Inozyme PharmaIndustry (pharma, biotech, devices)
Study start: 21 Nov 2021 (actual)
Primary completion: 29 Oct 2024 (actual)
Study completion: 13 Dec 2024 (actual)
First posted: 28 Dec 2020 (actual)
Results: Posted 27 Oct 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Subjects will complete an End of Study (EOS) Visit (Safety 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 INZ701-101 is a Phase 1/2, multi-center, open-label, first-in-human (FIH), multiple ascending dose (MAD) study followed by a long-term open-label extension period conducted in adults with ENPP1 Deficiency. 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 dose cohort. 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 (Cohorts 1-3) and 1.2 mg/kg once weekly dose regimen (Cohort 4). 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 64 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

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-FcINZ701-101 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.	Drug	INZ-701

Primary outcome

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) During the Dose Evaluation Period
Time frame: 32 days
Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.
Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) During the Open-label Extension Period
Time frame: From the start of the Open-label Extension Period (Day 32) through Week 48
Treatment-emergent adverse events (TEAEs) were defined as adverse events with an onset date on or after the first dose of INZ-701 and within 30 days after the last dose of INZ-701. For this outcome measure, participants with at least one TEAE occurring during the Open-label Extension Period through Week 48 were reported.
Number of Participants With Anti-Drug Antibodies (ADA) During the Dose Evaluation Period
Time frame: 32 days
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
Number of Participants With at Least One Positive Anti-Drug Antibody (ADA) Result During the Open-label Extension Period
Time frame: From entry into the Open-label Extension Period following completion of the 32-day Dose Evaluation Period through Week 48
The presence of anti-drug antibodies (ADAs) was assessed in each participant. Participants with at least one positive ADA result during the Open-label Extension Period through Week 48 were reported. Samples with detected ADAs underwent further assessment, as applicable.
Area Under the Plasma Concentration-Time Curve From 0 to 72 Hours (AUC0-72h) of INZ-701 Following the Day 1 Dose
Time frame: Day 1 predose through 72 hours postdose
Plasma samples were collected from predose through 72 hours after the Day 1 dose to determine the area under the plasma concentration-time curve from time zero to 72 hours postdose (AUC0-72h) of INZ-701.
Maximum Plasma Concentration (Cmax) of INZ-701 Following the Day 1 Dose
Time frame: Day 1 predose through Day 8 predose, up to 168 hours postdose
Maximum plasma concentration (Cmax) was obtained directly from the observed INZ-701 plasma concentration-time data following the Day 1 dose.
Apparent Systemic Clearance (CL/F) of INZ-701 Following the Day 1 Dose
Time frame: Day 1 predose through Day 8 predose, up to 168 hours postdose
Apparent systemic clearance after subcutaneous dosing (CL/F) was calculated as the administered dose divided by the area under the plasma concentration-time curve from time zero to infinity (AUCinf). AUCinf requires estimation of the terminal elimination rate constant (λz). If λz could not be reliably estimated, AUCinf and consequently CL/F could not be reliably determined.
Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels at Day 32/Early Termination
Time frame: Baseline to Day 32/Early Termination
Plasma inorganic pyrophosphate (PPi) concentrations were measured at Baseline and at the Day 32/Early Termination predose assessment. Change from Baseline was calculated as the Day 32/Early Termination PPi concentration minus the Baseline PPi concentration.
Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels at Week 48 of the Open-label Extension Period
Time frame: Baseline to Week 48 of the Open-label Extension Period
Plasma inorganic pyrophosphate (PPi) concentrations were measured at Baseline and during the Open-label Extension Period. Change from Baseline at Week 48 was calculated for each participant as the Week 48 PPi concentration minus the Baseline PPi concentration.

Results and safety

Results posted to the registry on 27 Oct 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cohort 1: Dose Level #1	3	3
Cohort 2: Dose Level #2	3	3
Cohort 3: Dose Level #3	3	3
Cohort 4: Dose Level #4	4	4

Primary outcome: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) During the Dose Evaluation Period

Time frame: 32 days. Count of participants. Unit: Participants

Measure	Cohort 1: Dose Level #1 n = 3	Cohort 2: Dose Level #2 n = 3	Cohort 3: Dose Level #3 n = 3	Cohort 4: Dose Level #4 n = 4
Value	0	1	0	3

Population analysed: The analysis population included all participants who received at least one dose of study treatment during the respective study period.

Primary outcome: Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) During the Open-label Extension Period

Time frame: From the start of the Open-label Extension Period (Day 32) through Week 48. Count of participants. Unit: Participants

Measure	Cohort 1: Dose Level #1 n = 3	Cohort 2: Dose Level #2 n = 3	Cohort 3: Dose Level #3 n = 3	Cohort 4: Dose Level #4 n = 4
Value	3	3	2	4

Population analysed: The analysis population included all participants who received at least one dose of INZ-701 during the Open-label Extension Period.

Primary outcome: Number of Participants With Anti-Drug Antibodies (ADA) During the Dose Evaluation Period

Time frame: 32 days. Count of participants. Unit: Participants

Measure	Cohort 1: Dose Level #1 n = 3	Cohort 2: Dose Level #2 n = 3	Cohort 3: Dose Level #3 n = 3	Cohort 4: Dose Level #4 n = 4
Value	0	0	1	2

Population analysed: The analysis population included all participants who received at least one dose of study treatment and had evaluable pharmacokinetic samples during the respective study period.

Primary outcome: Number of Participants With at Least One Positive Anti-Drug Antibody (ADA) Result During the Open-label Extension Period

Time frame: From entry into the Open-label Extension Period following completion of the 32-day Dose Evaluation Period through Week 48. Count of participants. Unit: Participants

Measure	Cohort 1: Dose Level #1 n = 3	Cohort 2: Dose Level #2 n = 3	Cohort 3: Dose Level #3 n = 3	Cohort 4: Dose Level #4 n = 4
Value	3	2	2	3

Population analysed: The analysis population included all participants who received at least one dose of INZ-701 during the Open-label Extension Period through Week 48.

Adverse events

Time frame: From first dose of INZ-701 through 30 days after the last dose of INZ-701 (up to approximately 48 weeks).. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort 1: Dose Level #1	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Cohort 2: Dose Level #2	2 of 3 (66.7%)	3 of 3 (100%)	0 of 3
Cohort 3: Dose Level #3	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Cohort 4: Dose Level #4	1 of 4 (25%)	4 of 4 (100%)	0 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 27 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

7 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 27 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 7 sites in United States, Germany, Canada, France, 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 27 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 With Treatment-Emergent Adverse Events (TEAEs) During the Dose Evaluation Period (time frame: 32 days). The study lists 9 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 NCT04686175.

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 27 Oct 2026.