A Phase 2 interventional study of INZ-701 in Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets and Generalized Arterial Calcification of Infancy 1, sponsored by Inozyme Pharma. Withdrawn. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2025-05-01.
Sponsored by Inozyme Pharma (part of BioMarin Pharmaceutical) · Phase 2, Interventional, and Treatment
The purpose of Study INZ701-108 (ENABLE) is to assess the safety and efficacy of INZ-701 in patients 1 year of age and older with ENPP1 Deficiency who have not previously received INZ-701 and are not eligible for existing Inozyme-sponsored clinical studies that are still open to enrollment.
INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy in development for the treatment of the ultra-rare genetic disorder, ENPP1 Deficiency.
Study INZ701-108 (ENABLE) is an open-label study to assess the long-term safety and efficacy of INZ-701 in patients 1 year of age and older with ENPP1 Deficiency who have not previously received INZ-701 and are not eligible for an Inozyme-sponsored clinical study that is open to recruitment.
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 treatment through 104 weeks. The dose will be determined by the participant's age and weight.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Individuals eligible to participate must meet all of the following inclusion criteria:
Must have at least one of the following clinical signs and/or symptoms consistent with ENPP1 Deficiency:
Exclusion 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.
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
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.
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.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
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.
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 SC injection on a once-weekly basis as follows: * Study participants from 1 year to \<18 years of age will receive a dose of 2.4 mg/kg * Study participants ≥18 years and: weighing \<50 kg or \>100 kg will receive a 1.8 mg/kg dose, OR weighing ≥50 and ≤100 kg will receive a 150 mg dose of INZ-701
Drug: INZ-701
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
Also known as: (rhENPP1-Fc).
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52
For each subject, plasma PPi will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
Time frame: 52 weeks (Baseline through Week 52)
Change from Baseline in skeletal abnormalities as measured by the Radiographic Global Impression of Change (RGI-C) global score through Week 104 for patients 1 to <13 years
For each subject, skeletal abnormalities will be measured via RGI-C global scores obtained throughout the study, comparing the subject's baseline value over time.
Time frame: 52 weeks (Baseline through Week 52)
Change from Baseline in Rickets Severity Score (RSS) total score through Week 104 (measured on all long bones with open growth plates in the knee or wrist) for patients 1 to <13 years
For each subject, rickets severity score will be measured on all long bones with open growth plates in knees or wrist obtained throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in arterial calcification score based on low dose computed tomography (CT) through Week 104 for patients 1 to <18 years
For each subject, arterial calcification will be measured via low dose computed tomography obtained throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change over time in serum biomarkers: Fibroblast growth factor 23 for patients >1 year
For each subject, various serum biomarkers will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change over time in serum biomarkers: Procollagen type 1 N-terminal propeptide for patients >1 year
For each subject, various serum biomarkers will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change over time in serum biomarkers: Bone-specific alkaline phosphatase for patients >1 year
For each subject, various serum biomarkers will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change over time in serum biomarkers: Carboxy terminal cross-linked telopeptide of type I collagen for patients >1 year
For each subject, various serum biomarkers will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in growth parameters (body weight expressed as Z-score) through Week 104 for patients 1 to <13 years
For each subject, growth parameters will be measured throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change over time in serum biomarkers: Serum phosphate for patients >1 year
For each subject, various serum biomarkers will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in growth parameters (height/length expressed as Z-score) through Week 104 for patients 1 to <13 years
For each subject, growth parameters will be measured throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline over time in growth velocity as measured by height over time through Week 104 for patients 1 to <13 years
For each subject, growth velocity will be measured via height throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in lower extremity angle measurements from radiographic x-rays through Week 104 for patients 1 to <13 years
For each subject, lower extremity angle will be measured via radiograph x-rays throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in audiometric outcomes as measured by hearing tests through Week 104 for patients 1 to <18 years
For each subject, audiometric outcomes will be measured via hearing tests throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in 6 Minute Walk Test (6MWT) for patients >5 years
For each subject, 6-minute walk test will be measured and assessed throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change in Baseline in bone mineral content and bone mineral density Z-score via dual X-ray absorptiometry (DXA) scan through Week 104 for patients ≥13 years
For each subject, bone mineral content and density will be measured via dual x-ray absorptiometry (DXA) throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in age-appropriate motor performance-mobility assessments through Week 104 (Peabody Developmental Motor Scale - third edition [PDMS] for patients 1 to < 5 years
For each subject, motor performance-mobility assessments will be measured via the Developmental Motor Scale throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Patient Reported Outcomes (PROs) through Week 104 assessed via Patient-Reported Outcome Measurement Information System (PROMIS) Pediatric - Cognitive Function, Fatigue, Pain Intensity, Pain Interference, Mobility) for patients 1 to <18 years
For each subject, quality of life PROs will be assessed throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
PROs through Week 104 (Patient-Reported Outcome Measurement Information System [PROMIS] Pediatric - Cognitive Function, Fatigue, Pain Intensity, Pain Interference, Physical Function) for patients >17 years
For each subject, quality of life PROs will be assessed throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
PROs through Week 104 (Clinical-, Caregiver-, and Patient-reported Global Impression of Change [CGI-C, CaGI-C, and PGI-C for patients >17 years
For each subject, PROs will be assessed throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in bone histomorphometry as measured by bone biopsy through Week 104 for patients >17 years
For each subject, bone histomorphometry will be measured via bone biopsy throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Change from Baseline in pain visual analog VAS) score through Week 104 for patients >17 years
For each subject, pain will be measured via pain visual analog scale throughout the study, comparing the subject's baseline value over time.
Time frame: 104 weeks (Baseline through Week 104)
Number of Treatment Adverse Events (TEAEs) through Week 104
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: 104 weeks (Baseline through Week 104)
Incidence of Anti-Drug Antibodies (ADA) through Week 104
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
Time frame: 104 weeks (Baseline through Week 104)
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Jan 2025; primary completion for Mar 2027, 26 months later.
How these figures are worked out, and which studies are left out of them.
This study is withdrawn, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Efmirenpase alfa
Hypophosphatemic Rickets, Autosomal Recessive, 1