Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot Project.

November 12, 2019 updated by: Michael Econs, Indiana University

Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot

The purpose of the study is to gain a better understanding of the effect of iron on fibroblast growth factor 23 (FGF23) in the inherited disorder, autosomal dominant hypophosphatemic rickets (ADHR). ADHR is an inherited disorder in which the body makes too much FGF 23 and causes low blood phosphorus levels and bone problems such as rickets (bowed legs in children) or bone pain and weakness in adults. This study is to test whether or not giving iron helps correct the high FGF23 and there by correcting the phosphate problem.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Iron will be provided in an open label treatment to all enrolled subjects. Iron levels will be monitored in blood and doses adjusted with the target of getting the iron levels to or a little above 100 mcg/dl.

The study will look to see if there is a decrease of FGF23 level. It will also look at how long does it take to decrease the level of FGF 23 and how long it takes for the serum and urine phosphate to normalize.

Study Type

Interventional

Enrollment (Actual)

8

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • FGF Mutation in either Arginine 176 or arginine 179
  • able and willing to provide consent or have a parent that is able/willing to consent, if a minor
  • either serum iron <50mcg/dl (regardless of phosphate or intact FGF23 concentration); or iron between 500 and 100mcg/dl with serum phosphorus value below 3.0mg/dl for adults or less than or equal to 0.5 mg/dl the lower limit of normal for age in children and intact FGF23 about 30pg/ml
  • age >2 years
  • May be receiving treatment with phosphate and calcitriol, but must be willing to undergo dose adjustments by the investigators if iron resolves the phosphate wasting defect.

Exclusion Criteria:

  • malignancy within the last 5 years, except treated squamous or basal cell skin carcinoma
  • terminal illness/hospice.
  • severe end-organ disease, e.g. cardiovascular, pulmonary, etc, which may limit ability to complete study.

estimated GFR <45ml/min/1.73m2, calculated using MDRD formula for adults or modified Schwartz equation for children

  • pregnancy or plan on becoming pregnant

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: iron supplements
all subjects will receive iron supplementation based on iron levels in blood
Dietary supplement: Iron
All subjects will receive iron supplementation based on iron levels in the blood
Other Names:
  • Ferrous Sulfate
  • Ferrous Gluconate

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Does increasing serum iron concentrations above 100 mcg/dl in patients with ADHR result in a decrease in intact FGF23.
Time Frame: FGF23 will be measured at 1, 2, 3, 6, 9, and 12 months
Perform a pilot study in ADHR patients with low serum iron concentrations (defined below) to determine if increasing serum iron concentrations above 100 mcg/dl results in a decrease in intact FGF23 (primary endpoint) and C-terminal FGF23 concentrations by at least 20% and normalizes serum phosphorus and TMP/GFR (tubular maximum phosphate reabsorption/ glomerular filtration rate) within 6 months of attaining goal iron concentrations.
FGF23 will be measured at 1, 2, 3, 6, 9, and 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Indiana University

Investigators

  • Principal Investigator: Michael Econs, M.D., Indiana University School of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2014

Primary Completion (Actual)

November 12, 2019

Study Completion (Actual)

November 12, 2019

Study Registration Dates

First Submitted

August 27, 2014

First Submitted That Met QC Criteria

September 2, 2014

First Posted (Estimate)

September 8, 2014

Study Record Updates

Last Update Posted (Actual)

November 14, 2019

Last Update Submitted That Met QC Criteria

November 12, 2019

Last Verified

November 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IRON/ADHR

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Autosomal Dominant Hypophosphatemic Rickets

Clinical Trials on Iron

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Colombia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe