Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency (NCT04686175) | Clinical Trial Compass
CompletedPhase 1/2
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency
United States, Canada, France9 participantsStarted 2021-11-21
Plain-language 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. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency.
Who can participate
Age range
18 Years – 64 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Treatment Emergent Adverse Events (TEAEs)
Timeframe: 32 days (Dose Evaluation Period)
2
Number of Treatment Emergent Adverse Events (TEAEs)
Timeframe: 52 weeks (Day 1 through Safety Follow-up Visit)
3
Incidence of Anti-Drug Antibodies (ADA)
Timeframe: 32 days (Dose Evaluation Period)
4
Incidence of Anti-Drug Antibodies (ADA)
Timeframe: 52 weeks (Baseline through Safety Follow-up Visit)
5
Area under the Plasma Concentration versus Time Curve (AUC) 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.
Exclusion criteria
. 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
. 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
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels
Timeframe: 32 days (Dose Evaluation Period)
9
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels
Timeframe: 52 weeks (Baseline through Safety Follow-up Visit)