Philadelphia, PANCT05734196Now EnrollingIRB Ready

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Clinical Trial in Philadelphia, PA

Access cutting-edge ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency treatment through this clinical trial at a research site in Philadelphia. Study-provided care at no cost to qualified participants.

Sponsored by Inozyme Pharma

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Expert Care in Philadelphia

Access ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency treatment provided free

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Check if you qualify for this ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency clinical trial in Philadelphia, PA

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Why Participate?

  • No-Cost Study Care

  • Local to Philadelphia

    Convenient for PA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Philadelphia site if eligible
  4. 4Begin participation

About This Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Study in Philadelphia

The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.

Sponsor: Inozyme Pharma

Who Can Participate

Inclusion Criteria

Infant aged ≤ 1 year at the time of enrollment
Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
Study participants must have clinical manifestations of generalized arterial calcification of infancy (GACI) or GACI-2, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly.
Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
Written informed consent provided by a parent or legal guardian

Exclusion Criteria

In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality that precludes study participation or may confound interpretation of study result
Receiving end of life or hospice care
Known malignancy
Concurrent participation in another non-Inozyme interventional study
Treatment with any non-Inozyme product or investigational device during study participation

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Philadelphia?

Yes, this clinical trial (NCT05734196) has an active research site in Philadelphia, PA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Treatment Options in Philadelphia, PA

If you're searching for ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency treatment options in Philadelphia, PA, this clinical trial (NCT05734196) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Philadelphia research site is actively enrolling participants for this clinical trial. You'll receive care from experienced ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency clinical trials near you to find additional studies recruiting in your area.

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