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The Impact of Phosphate Metabolism on Healthy Aging

Primary Purpose

Hypophosphatemia, Rickets, Hypercalciuria

Status
Suspended
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
phosphate
Sponsored by
Yale University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hypophosphatemia

Eligibility Criteria

13 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Children above the age of 13 years
  • Younger and older adults with XLH and HHRH with confirmed NPT2c mutations affecting both copies of the NPT2c gene (HHRH) or one copy of the PHEX gene (XLH)
  • Be willing to provide access to prior medical records to determine eligibility including imaging, biochemical, medical, and surgical history data
  • Be willing and able to complete all aspects of the study
  • Be willing to adhere to the study visit schedule and comply with the assessments (in the opinion of the investigator).

Exclusion Criteria:

  • Subjects will be excluded, if they are children younger than age 13 years
  • Subjects that have other diseases likely to impact bone and mineral metabolism (e.g. renal, hepatic, gastrointestinal disorders, and malignancy),
  • Subjects that are currently pregnant,
  • Subjects that received medical therapy or developed any condition, which in the opinion of the investigator, could present a concern for either subject safety or difficulty with data interpretation.
  • Subjects will be excluded from Aim 2, if they are unable to tolerate supplemental phosphate.

Sites / Locations

  • Yale University School of Medicine

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Active Comparator

Active Comparator

Active Comparator

Arm Label

Patients with hereditary hypophosphatemic rickets with HHRH

Patients with X-linked Hypophosphatemia

15 Patients with X-linked Hypophosphatemia

15 Patients Hereditary hypophosphatemic rickets with HHRH

Arm Description

Hereditary hypophosphatemic rickets with hypercalciuria (HHRH)

Patients with X-linked hypophosphatemia

15 Patients with X-linked Hypophosphatemia that will receive phosphate treatment for 30 days.

15 patients withHereditary hypophosphatemic rickets with hypercalciuria (HHRH) that will receive phosphate treatment for 30 days.

Outcomes

Primary Outcome Measures

Parathyroid Hormone (PTH) levels
PTH levels are expected to increase over baseline after phosphate supplement (Pi).
Fibroblast Growth Factor 23 (FGF23) levels
FGF23 levels are expected to increase over baseline after phosphate supplement (Pi).

Secondary Outcome Measures

Full Information

First Posted
December 7, 2018
Last Updated
March 3, 2023
Sponsor
Yale University
Collaborators
National Center for Advancing Translational Sciences (NCATS)
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1. Study Identification

Unique Protocol Identification Number
NCT03771105
Brief Title
The Impact of Phosphate Metabolism on Healthy Aging
Official Title
The Impact of Phosphate Metabolism on Healthy Aging
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Suspended
Why Stopped
lack of funding
Study Start Date
January 1, 2019 (Actual)
Primary Completion Date
November 2023 (Anticipated)
Study Completion Date
November 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Yale University
Collaborators
National Center for Advancing Translational Sciences (NCATS)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Determine the association between duration and dose of chronic conventional therapy with Pi and renal (nephrocalcinosis/nephrolithiasis), vascular (endothelial function), and cardiovascular function (echo- cardiography) in patients with hereditary hypophosphatemic rickets with hypercalciuria (HHRH) and patients with X-linked hypophosphatemia (XLH).
Detailed Description
The central hypothesis of this proposal is that patients with X-linked hypophosphatemia (XLH), when matched for duration and dose of phosphate (Pi) therapy to patients with hereditary hypophosphatemic rickets with hypercalciuria (HHRH), will evidence greater cardiovascular and vascular debility than patients with HHRH. The overall objectives of this project are to utilize our existing longitudinal databases for individuals with XLH and HHRH through an interdisciplinary collaboration between pediatric and adult endocrinology to: i) quantify the impact of exposure to Pi therapy across the lifespan on cardiovascular and renal complications, which are key aging endpoints, ii) determine the acute response to Pi loading in XLH and HHRH by studying the changes in surrogate markers of cardiovascular and renal function.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypophosphatemia, Rickets, Hypercalciuria, XLH, HHRH

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Masking Description
crossover parallel
Allocation
Non-Randomized
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Patients with hereditary hypophosphatemic rickets with HHRH
Arm Type
Experimental
Arm Description
Hereditary hypophosphatemic rickets with hypercalciuria (HHRH)
Arm Title
Patients with X-linked Hypophosphatemia
Arm Type
Active Comparator
Arm Description
Patients with X-linked hypophosphatemia
Arm Title
15 Patients with X-linked Hypophosphatemia
Arm Type
Active Comparator
Arm Description
15 Patients with X-linked Hypophosphatemia that will receive phosphate treatment for 30 days.
Arm Title
15 Patients Hereditary hypophosphatemic rickets with HHRH
Arm Type
Active Comparator
Arm Description
15 patients withHereditary hypophosphatemic rickets with hypercalciuria (HHRH) that will receive phosphate treatment for 30 days.
Intervention Type
Drug
Intervention Name(s)
phosphate
Intervention Description
The target daily phosphate (Pi) intake is 3,500 mg/day (dietary intake plus our supplementation) for this study. Subjects will take a supplement of Pi (as KPhos Neutral 250 mg/tablet) to reach this target. Subjects will receive treatment for 30 days.
Primary Outcome Measure Information:
Title
Parathyroid Hormone (PTH) levels
Description
PTH levels are expected to increase over baseline after phosphate supplement (Pi).
Time Frame
30 days
Title
Fibroblast Growth Factor 23 (FGF23) levels
Description
FGF23 levels are expected to increase over baseline after phosphate supplement (Pi).
Time Frame
30 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Children above the age of 13 years Younger and older adults with XLH and HHRH with confirmed NPT2c mutations affecting both copies of the NPT2c gene (HHRH) or one copy of the PHEX gene (XLH) Be willing to provide access to prior medical records to determine eligibility including imaging, biochemical, medical, and surgical history data Be willing and able to complete all aspects of the study Be willing to adhere to the study visit schedule and comply with the assessments (in the opinion of the investigator). Exclusion Criteria: Subjects will be excluded, if they are children younger than age 13 years Subjects that have other diseases likely to impact bone and mineral metabolism (e.g. renal, hepatic, gastrointestinal disorders, and malignancy), Subjects that are currently pregnant, Subjects that received medical therapy or developed any condition, which in the opinion of the investigator, could present a concern for either subject safety or difficulty with data interpretation. Subjects will be excluded from Aim 2, if they are unable to tolerate supplemental phosphate.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clemens Bergwitz, MD
Organizational Affiliation
Yale University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Yale University School of Medicine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06511
Country
United States

12. IPD Sharing Statement

Learn more about this trial

The Impact of Phosphate Metabolism on Healthy Aging

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