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INZ-701 for ENPP1 Deficiency

Q: Are adolescents within the age range of eligibility for this experiment?

According to the criteria for this research, participants must be aged 1 year up to 12 years old. Additionally, there are 9 clinical trials available for patients below 18 and 28 studies open to persons above 65.

Q: Might I be qualified to take part in this experiment?

The specific criteria for inclusion in this trial lays out that patients must have hypophosphatemic rickets and be aged between 1 year old to 12 years old. There are 33 spots available for participants.

Q: Has INZ-701 been authorized by the FDA?

Our team at Power assigned INZ-701 a score of 3, indicating that there is enough evidence collected to support its safety. This result stems from the Phase 3 trial which has yielded both efficacy and multiple safety data points.

Q: Is this clinical trial actively recruiting participants?

Affirmative. The information available on clinicaltrials.gov attests that this trial is currently enrolling participants, with the first post dated 22nd September 2023 and the latest update made on 2nd October 2023. 33 patients are needed to complete the study at a single research centre.

Q: How many individuals are participating in the current clinical trial?

Yes, the information on clinicaltrials.gov confirms that this experiment is still actively seeking participants - it was initially posted in September of 2023 and has been updated recently as well. A total of 33 patients are needed across a single medical facility.

Phase 3

Recruiting

Research Sponsored by Inozyme Pharma

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Males and females ≥1 year and <13 years of age at Study Day 1

Female participants of childbearing potential must have a negative serum pregnancy test at Screening and must not be breastfeeding

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, day 29, week 8, week 13, week 26, week 39, week 52

Awards & highlights

Study Summary

This trial tests if a drug can help treat a rare disorder in children, focusing on safety & efficacy.

Who is the study for?

This trial is for children with ENPP1 Deficiency, showing specific bone abnormalities and growth plate activity. They must be between 1-12 years old, not pregnant or breastfeeding, willing to use contraception if applicable, and have certain vitamin D levels. Those who've had recent surgery or used certain medications like systemic corticosteroids are excluded.Check my eligibility

What is being tested?

The ENERGY 3 Study tests the safety and effectiveness of INZ-701 compared to conventional therapy in treating skeletal issues caused by ENPP1 Deficiency in children. Participants will either receive INZ-701 or stick with standard treatments to see which works better.See study design

What are the potential side effects?

While the side effects of INZ-701 are not detailed here, they may include reactions similar to other drugs targeting bone metabolism such as gastrointestinal symptoms, skin reactions, or changes in blood chemistry.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 1 and 12 years old.

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I am not pregnant or breastfeeding.

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I have a genetic diagnosis of ENPP1 Deficiency confirmed by a certified lab.

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My scans show significant bone abnormalities.

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My knee growth plates are still open.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, day 29, week 8, week 13, week 26, week 39, week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, day 29, week 8, week 13, week 26, week 39, week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, day 29, week 8, week 13, week 26, week 39, week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52

Secondary outcome measures

Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701

Change from Baseline in ENPP1 activity (µM/min) through week 52

Change from Baseline in growth Z-score (height/body length and weight) through Week 52

+3 more

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: INZ-701Experimental Treatment1 Intervention

Subjects randomized to the INZ-701 arm will be administered a 2.4 mg/kg once weekly dose by subcutaneous (SC) injection for the duration of the 52-week Randomized Treatment Period and the Open-label Extension Period.

Group II: Control Arm (Conventional Therapy)Active Control1 Intervention

Subjects randomized to the control arm will continue taking their conventional therapy as clinically indicated by their treating physician for the duration of the 52-week Randomized Treatment Period.

0 / 5Eligibility criteria met

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Who is running the clinical trial?

Inozyme PharmaLead Sponsor

7 Previous Clinical Trials

1,105 Total Patients Enrolled

Alex Lai, MDStudy DirectorInozyme Pharma, Inc.

1 Previous Clinical Trials

8 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are adolescents within the age range of eligibility for this experiment?

"According to the criteria for this research, participants must be aged 1 year up to 12 years old. Additionally, there are 9 clinical trials available for patients below 18 and 28 studies open to persons above 65."

Answered by AI

Might I be qualified to take part in this experiment?

"The specific criteria for inclusion in this trial lays out that patients must have hypophosphatemic rickets and be aged between 1 year old to 12 years old. There are 33 spots available for participants."

Answered by AI

Has INZ-701 been authorized by the FDA?

"Our team at Power assigned INZ-701 a score of 3, indicating that there is enough evidence collected to support its safety. This result stems from the Phase 3 trial which has yielded both efficacy and multiple safety data points."

Answered by AI

Is this clinical trial actively recruiting participants?

"Affirmative. The information available on clinicaltrials.gov attests that this trial is currently enrolling participants, with the first post dated 22nd September 2023 and the latest update made on 2nd October 2023. 33 patients are needed to complete the study at a single research centre."

Answered by AI

How many individuals are participating in the current clinical trial?

"Yes, the information on clinicaltrials.gov confirms that this experiment is still actively seeking participants - it was initially posted in September of 2023 and has been updated recently as well. A total of 33 patients are needed across a single medical facility."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

2023. "The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06046820.