A Phase 1/2 interventional study of INZ-701 in Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets and Generalized Arterial Calcification of Infancy, sponsored by Inozyme Pharma. Completed at 7 sites in 5 countries. Open to participants aged 18 Years to 64 Years. Per ClinicalTrials.gov, last updated 2026-10-27.
Sponsored by Inozyme Pharma (part of BioMarin Pharmaceutical) · Phase 1/2, Interventional, and Treatment
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. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency.
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.
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:
Clinically significant abnormal laboratory result at Screening in the opinion of the Investigator, including but not limited to screening laboratory results demonstrating
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.
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.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
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.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
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.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
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.
Drug: INZ-701
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.
Also known as: rhENPP1-Fc
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline to Week 48 of the Open-label Extension Period
| Milestone | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Started | 3 | 3 | 3 | 4 |
| Completed | 3 | 3 | 3 | 4 |
| Not completed | 0 | 0 | 0 | 0 |
| Milestone | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Started | 3 | 3 | 3 | 4 |
| Completed | 2 | 2 | 2 | 4 |
| Not completed | 1 | 1 | 1 | 0 |
| Withdrew: Withdrawal by subject | 1 | 1 | 1 | 0 |
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.
| Participants | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) During the Dose Evaluation Period | 0 | 1 | 0 | 3 |
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.
| Participants | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) During the Open-label Extension Period | 3 | 3 | 2 | 4 |
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
| Participants | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Number of Participants With Anti-Drug Antibodies (ADA) During the Dose Evaluation Period | 0 | 0 | 1 | 2 |
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.
| Participants | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Number of Participants With at Least One Positive Anti-Drug Antibody (ADA) Result During the Open-label Extension Period | 3 | 2 | 2 | 3 |
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.
| h*ng/mL | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Area Under the Plasma Concentration-Time Curve From 0 to 72 Hours (AUC0-72h) of INZ-701 Following the Day 1 Dose | 14100 ± 5720 | 28200 ± 19700 | 108000 ± 55100 | 41900 ± 36600 |
Maximum plasma concentration (Cmax) was obtained directly from the observed INZ-701 plasma concentration-time data following the Day 1 dose.
| ng/mL | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Maximum Plasma Concentration (Cmax) of INZ-701 Following the Day 1 Dose | 250 ± 84.5 | 584 ± 271 | 1950 ± 868 | 1840 ± 1770 |
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.
| L/h | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Apparent Systemic Clearance (CL/F) of INZ-701 Following the Day 1 Dose | NA ± NA | NA ± NA | NA ± NA | NA ± NA |
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.
| nM | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels at Day 32/Early Termination | 695.3 ± 1013.8 | 1063.0 ± 657.7 | 1176.7 ± 330.3 | 539.3 ± 678.6 |
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.
| nM | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels at Week 48 of the Open-label Extension Period | 359 ± 0 | 1440 ± 336 | 1050 ± 0 | 4940 ± 0 |
Collected over From first dose of INZ-701 through 30 days after the last dose of INZ-701 (up to approximately 48 weeks).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: Dose Level #1 | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Cohort 2: Dose Level #2 | 0/3 (0%) | 2/3 (66.7%) | 3/3 (100%) |
| Cohort 3: Dose Level #3 | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Cohort 4: Dose Level #4 | 0/4 (0%) | 1/4 (25%) | 4/4 (100%) |
| Event | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| patella fractureInjury, poisoning and procedural complications | 0/3 | 1/3 | 0/3 | 0/4 |
| procedure abortedSurgical and medical procedures | 0/3 | 1/3 | 0/3 | 0/4 |
| syncopeNervous system disorders | 0/3 | 0/3 | 0/3 | 1/4 |
| Event | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 |
|---|---|---|---|---|
| ArthralgiaMusculoskeletal and connective tissue disorders | 0/3 | 0/3 | 2/3 | 2/4 |
| HeadacheNervous system disorders | 1/3 | 1/3 | 2/3 | 1/4 |
| FatigueGeneral disorders | 1/3 | 0/3 | 0/3 | 1/4 |
| Injection Site BruisingGeneral disorders | 1/3 | 1/3 | 0/3 | 0/4 |
| Injection Site ErythemaGeneral disorders | 0/3 | 0/3 | 1/3 | 1/4 |
| Injection Site HaemorrhageGeneral disorders | 0/3 | 1/3 | 0/3 | 1/4 |
| Injection Site PainGeneral disorders | 0/3 | 1/3 | 0/3 | 1/4 |
| Injection Site InjuryGeneral disorders | 0/3 | 1/3 | 0/3 | 0/4 |
| Injection Site PruritusGeneral disorders | 0/3 | 1/3 | 0/3 | 0/4 |
| Injection Site SwellingGeneral disorders | 0/3 | 1/3 | 0/3 | 0/4 |
The baseline analysis population included all 13 participants who received study treatment.
| Age, Categorical(Participants) | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 | Total |
|---|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 3 | 3 | 3 | 4 | 13 |
| >=65 years | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous(Years) | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 | Total |
|---|---|---|---|---|---|
| Median | 31.0 (23 to 40) | 43.0 (30 to 58) | 25.0 (22 to 29) | 21.0 (20 to 32) | 29.0 (20 to 58) |
| Sex: Female, Male(Participants) | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 | Total |
|---|---|---|---|---|---|
| Female | 3 | 2 | 1 | 2 | 8 |
| Male | 0 | 1 | 2 | 2 | 5 |
| Race (NIH/OMB)(Participants) | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 2 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 | 0 |
| White | 3 | 3 | 2 | 1 | 9 |
| More than one race | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 1 | 1 | 2 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 | 0 | 0 |
| Not Hispanic or Latino | 3 | 3 | 2 | 3 | 11 |
| Unknown or Not Reported | 0 | 0 | 1 | 1 | 2 |
| Region of Enrollment(Participants) | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 | Total |
|---|---|---|---|---|---|
| United States | 0 | 3 | 0 | 0 | 3 |
| France | 0 | 0 | 1 | 1 | 2 |
| Germany | 0 | 0 | 2 | 3 | 5 |
| United Kingdom | 2 | 0 | 0 | 0 | 2 |
| Canada | 1 | 0 | 0 | 0 | 1 |
| Baseline Plasma Inorganic Pyrophosphate (PPi)(nM) | Cohort 1: Dose Level #1 | Cohort 2: Dose Level #2 | Cohort 3: Dose Level #3 | Cohort 4: Dose Level #4 | Total |
|---|---|---|---|---|---|
| Mean | 188.7 ± 170.9 | 639.7 ± 682.9 | 306.7 ± 36.1 | 635.3 ± 669.0 | 457.4 ± 487.1 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
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 ↗
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Studies of the drug tested here: Efmirenpase alfa
Hypophosphatemic Rickets, Autosomal Recessive, 1