Gene MutationsPhase 2NCT06462547
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
A Phase 2 study testing INZ-701 in people with Gene Mutations and Pseudoxanthoma Elasticum, run by Inozyme Pharma at 5 sites in 4 countries.
Trial summary
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification
- What is being tested?
- INZ-701
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 19 Jun 2024to 30 Sep 2026
- Phase
- Phase 2
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Individuals eligible to participate must meet all of the following inclusion criteria:
- Provide written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP)
- Provide assent in accordance with local regulations, if <18 years of age
- Male or female, greater than 1 year of age
- Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of a previous INZ-701 clinical study in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor
- Female participants of childbearing potential who are sexually active must be using or agree to use 1 highly effective form of contraception (per CTFG 2020) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701); participants must agree to not donate ova from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701
- Male participants who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701; participants must 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, able to complete all aspects of the study
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 or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and/or may confound interpretation of study results
- Known intolerance to INZ-701 or any of its excipients
- Concurrent participation in another interventional clinical study and/or has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device
- Pregnant, trying to become pregnant, or breastfeeding
- Male participants trying to father a child
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
United States2 sites
Clinilabs Drug Development Corporation
Eatontown, New Jersey, 07724
Mayo Clinic
Rochester, Minnesota, 55905
About this study
Official title: The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
Read the detailed description
The investigational product INZ-701 is being developed as a therapeutic protein for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency and adenosine triphosphate (ATP)-binding cassette subfamily C member 6 (ABCC6) Deficiency. INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin (Ig) G1 antibody.
The ADAPT Study (INZ701-304) is an open-label study to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
The study will consist of a 30-day Screening Period, followed by an open-label Treatment Period during which all participants will receive once-weekly subcutaneous (SC) doses of INZ-701 and continue with treatment until INZ-701 is commercially available in the country/region of the participant's residence or until Inozyme chooses to discontinue development of INZ-701. Participants will complete an End of Study (EOS) safety follow-up visit approximately 30 days after their last designated study visit assigned by the Investigator and/or Sponsor.
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets Type 2
- Being tested
- INZ-701
- Keywords
- ABCC6, ATP-Binding Cassette Subfamily C Member 6 Deficiency, PXE, Pseudoxanthoma elasticum, GACI, Generalized Arterial Calcification of Infancy, ARHR2, Autosomal Recessive Hypophosphatemic Rickets Type 2, ENPP1, ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency, 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 | INZ-701 will be administered by subcutaneous injection on a once-weekly basis as follows:
* Study participants from 1 year to <13 years of age will receive a dose of 2.4 mg/kg
* Study participants ≥13 years will either receive a 1.8 mg/kg dose or a 150 mg flat 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 Treatment Emergent Adverse Events (TEAEs)
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: 6 years (long term safety assessment)
Incidence of Anti-Drug Antibodies (ADA)
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
Measured: 6 years (long term safety assessment)
Secondary outcomes (3)
Time to maximum serum concentration (Tmax)
For each subject, the time to reach maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.
Measured: 6 years (long term safety assessment)
Maximum Plasma Concentration (Cmax) of INZ-701
For each subject, the maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.collected and assessed.
Measured: 6 years (long term safety assessment)
Mean Change from Baseline in Plasma PPi Concentration
For each subject, their plasma PPi concentrations based on a validated assay will be collected and assessed throughout the study, comparing the participant's baseline value over time.
Measured: 6 years (long term safety assessment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2024
- Study start19 Jun 2024
- Primary completion30 Sep 2026
- Study completion30 Sep 2026
Why it stopped: The study was terminated by the sponsor prior to planned completion based on a review of the data across indications and overall program considerations. Study termination was not related to participant safety.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gene MutationsStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- The ADAPT Study (INZ701-304) is an open-label study to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Inozyme Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kurt Gunter, MD, Study director, Inozyme Pharma, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06462547
- Sponsor's protocol ID
- INZ701-304
- Status last verified
- Oct 2026
- First submitted
- 12 Jun 2024
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
- ClinicalTrials.gov ID
- NCT06462547
- Acronym / sponsor ID
- INZ701-304
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets Type 2
- Keywords
- ABCC6, ATP-Binding Cassette Subfamily C Member 6 Deficiency, PXE, Pseudoxanthoma elasticum, GACI, Generalized Arterial Calcification of Infancy, ARHR2, Autosomal Recessive Hypophosphatemic Rickets Type 2
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated by the sponsor prior to planned completion based on a review of the data across indications and overall program considerations. Study termination was not related to participant safety.
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Inozyme PharmaIndustry (pharma, biotech, devices)
- Study start
- 19 Jun 2024 (actual)
- Primary completion
- 30 Sep 2026 (actual)
- Study completion
- 30 Sep 2026 (actual)
- First posted
- 17 Jun 2024 (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 purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
Detailed description
The investigational product INZ-701 is being developed as a therapeutic protein for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency and adenosine triphosphate (ATP)-binding cassette subfamily C member 6 (ABCC6) Deficiency. INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin (Ig) G1 antibody.
The ADAPT Study (INZ701-304) is an open-label study to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
The study will consist of a 30-day Screening Period, followed by an open-label Treatment Period during which all participants will receive once-weekly subcutaneous (SC) doses of INZ-701 and continue with treatment until INZ-701 is commercially available in the country/region of the participant's residence or until Inozyme chooses to discontinue development of INZ-701. Participants will complete an End of Study (EOS) safety follow-up visit approximately 30 days after their last designated study visit assigned by the Investigator and/or Sponsor.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The ADAPT Study (INZ701-304) is an open-label study to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
| Arm | Type | Receives |
|---|---|---|
| INZ-701 | Experimental | Drug: INZ-701INZ-701 will be administered by subcutaneous injection on a once-weekly basis as follows: * Study participants from 1 year to <13 years of age will receive a dose of 2.4 mg/kg * Study participants ≥13 years will either receive a 1.8 mg/kg dose or a 150 mg flat dose of INZ-701 |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older 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-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 Treatment Emergent Adverse Events (TEAEs)
Time frame: 6 years (long term safety assessment)
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.
Incidence of Anti-Drug Antibodies (ADA)
Time frame: 6 years (long term safety assessment)
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
3 secondary outcomes
Time to maximum serum concentration (Tmax)
Time frame: 6 years (long term safety assessment)
Maximum Plasma Concentration (Cmax) of INZ-701
Time frame: 6 years (long term safety assessment)
Mean Change from Baseline in Plasma PPi Concentration
Time frame: 6 years (long term safety assessment)
Results and safety
The study was stopped early (reason given: The study was terminated by the sponsor prior to planned completion based on a review of the data across indications and overall program considerations. Study termination was not related to participant safety.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
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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.
Kurt Gunter, MD study director
Inozyme Pharma, Inc.
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing INZ-701 in people with Gene Mutations and Pseudoxanthoma Elasticum, run by Inozyme Pharma at 5 sites in 4 countries.
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: stopped early, as of the sponsor's last update on 19 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 5 sites in United States, France, Germany, 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 phase mean?
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?
The study was stopped early (reason given: The study was terminated by the sponsor prior to planned completion based on a review of the data across indications and overall program considerations. Study termination was not related to participant safety.). Results are not available in the source record.
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: 6 years (long term safety assessment)). 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 19 Oct 2026.