A Phase 2 interventional study of INZ-701 in Gene Mutations, Pseudoxanthoma Elasticum and Arterial Calcification, sponsored by Inozyme Pharma. Terminated at 5 sites in 4 countries. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2026-10-19.
Sponsored by Inozyme Pharma (part of BioMarin Pharmaceutical) · Phase 2, Interventional, and Treatment
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.
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.
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.
32 studies on the registry are indexed under Pseudoxanthoma Elasticum; 6 are open to participants now.
This study's enrollment of 22 is close to the median of 21 across 16 interventional studies indexed under Pseudoxanthoma Elasticum.
Browse Pseudoxanthoma Elasticum studies →Inozyme Pharma is the lead sponsor of 12 studies on the registry; none are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 2 (33%) have results posted.
First submitted before Inozyme Pharma became part of BioMarin Pharmaceutical.
Counted across the registry records on this site, refreshed daily.
Individuals eligible to participate must meet all of the following inclusion criteria:
Individuals who meet any of the following exclusion criteria will not be eligible to participate:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Drug: INZ-701
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).
Also known as: rhENPP1-Fc
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.
Time frame: 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.
Time frame: 6 years (long term safety assessment)
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.
Time frame: 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.
Time frame: 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.
Time frame: 6 years (long term safety assessment)
Ran from Jun 2024 to primary completion in Sep 2026, 27 months.
Since we started tracking on Sep 25, 2026, the dates moved once, on Oct 19, 2026: primary completion Oct 2026 → Sep 2026 and completion Oct 2026 → Sep 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
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.
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.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is terminated, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Efmirenpase alfa