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Clinical Studies
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A Prospective Observational Patient Registry to Evaluate Disease Progression in Patients With ENPP1 Deficiency and Infantile-Onset ABCC6 Deficiency (GACI Type 2)
Rochester, Minn.
The purpose of this study is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette transporter protein subfamily C member 6 (ABCC6) Deficiency longitudinally. The registry will prospectively gather information about the genetic, biochemical, physiological, anatomic, radiographic, and functional manifestations (including patient reported outcomes [PROs]) of each disease during routine, standard-of-care visits, with the aim of developing a comprehensive understanding of the burden of illness and progressive nature of the disease.
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The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients with ENPP1 Deficiency and ABCC6 Deficiency
Rochester, Minn.
The purpose of this study (Study INZ701-304 [ADAPT])) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
Closed for Enrollment
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A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Adults With ENPP1 Deficiency (ENPP-INZ)
Rochester, Minn.
The purpose of this study is to assess safety and tolerability of INZ-701, a recombinant Ectonucleotide Pyrophosphatase/Phosphodiesterase (ENPP1) enzyme, for the treatment of ENPP1 deficiency. The study also aims to establish the safety and tolerability of multiple ascending subcutaneous (SC) doses of INZ-701.
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A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy and Safety of Encaleret Compared to Standard of Care in Participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Rochester, Minn.
The purpose of this study is to evaluate the effectiveness, safety, and tolerability of encaleret in comparison to standard of care (SoC) in participants with ADH1.
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Autosomal Dominant Hypocalcemia Types 1 and 2 (ADH1/2) Disease Monitoring Study (DMS) (CLARIFY)
Rochester, Minn.
The purpose iof this study is to determine the biochemical manifestations and progression of Autosomal Dominant Hypocalcemia Types 1 and 2 Disease (ADH1/2) over time.
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Denosumab Stimulated Parathyroid Scan Pilot Study in Persistent or Recurrent Primary Hyperparathyroidism
Rochester, Minn.
The purpose of this study is to determine if denosumab prior to parathyroid scan in patients with recurrent or persistent primary hyperparathyroidism and osteoporosis or osteopenia improves localization of parathyroid adenomas.
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Prevalence of Hypophosphatasia in Patients with Fibromyalgia at the Mayo Clinic
Rochester, Minn.
The overall goal of this research is to identify patients in clinical practice that have unrecognized hypophosphatasia (HPP) and select appropriate candidates for therapy. The purpose of this proposal is to identify the prevalence of adults with hypophosphatasia within a previously identified fibromyalgia cohort at our institution.
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