Role Of Phosphorus And FGF 23 In Patients With Dent Disease

• Sponsor: Mayo Clinic (Other)
• Overall Status: Completed
• CT.gov ID: NCT02016235
• Collaborator: (none)
• Enrollment: 30
• Location: 1
• Arms: 3
• Duration (Months): 52
• Patients Per Site Per Month: 0.6

Study Details

Study Description

Brief Summary

Patients with Dent disease have suppressed levels of FGF 23 which contributes to hypercalciuria, kidney stones, nephrocalcinosis and renal failure. Supplementation with phosphorus may reduce hypercalciuria.

Condition or Disease	Intervention/Treatment	Phase
Dent Disease	Drug: Phosphorus Supplement; Other: Observation	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 30 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Role Of Phosphorus And FGF 23 In Patients With Dent Disease
• Study Start Date: Sep 1, 2014
• Actual Primary Completion Date: Jan 1, 2019
• Actual Study Completion Date: Jan 1, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Dent Disease Intervention Dent Disease subjects will receive 2 week supplementation with phosphorus	Drug: Phosphorus Supplement; 250 mg po qid; Other Names: K-phos neutral
Experimental: Kidney Stone subjects Kidney stone with or without phosphate leak subjects will receive 2 week supplementation with phosphorus	Drug: Phosphorus Supplement; 250 mg po qid; Other Names: K-phos neutral
Placebo Comparator: Dent Disease Observation Dent disease subjects will not get phosphorus	Other: Observation; Baseline blood and urine measurements only

Outcome Measures

Primary Outcome Measures

1. Change in Urine Total Protein [baseline, day 7]

   Urine protein tests detect and/or measure protein being released into the urine. Normal urine protein elimination is less than 150 mg/day

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Inclusion Criteria:

Patients will be recruited from those in the RKSC Dent Registry

1. Diagnostic criteria for Dent disease Observational arm include:

2. <18 years old

3. LMWP (at least 5 times above the upper limit of normal) and at least 1 of the following criteria: 1. Hypercalciuria, 2. Kidney stones, 3. Nephrocalcinosis, 4. Hypophosphatemia, 5. Renal phosphate leak, 6. Aminoaciduria, 7. Glucosuria without diabetes mellitus, 8. Hematuria, 9. Renal insufficiency, 10. Family history with x-linked inheritance or

4. 1 of the above criteria (1-9) and confirmed genetic mutation of CLCN5 or OCRL1.

5. Diagnostic criteria for Dent disease Intervention arm include:

6. 18 years old

7. LMWP (at least 5 times above the upper limit of normal) and at least 1 of the following criteria: 1. Hypercalciuria, 2. Kidney stones, 3. Nephrocalcinosis, 4. Hypophosphatemia, 5. Renal phosphate leak, 6. Aminoaciduria, 7. Glucosuria without diabetes mellitus, 8. Hematuria, 9. Renal insufficiency, 10. Family history with x-linked inheritance or

8. 1 of the above criteria (1-9) and confirmed genetic mutation of CLCN5 or OCRL1.

9. Idiopathic calcium nephrolithiasis with renal phosphate leak

10. Male patients > 18 years old

11. History of symptomatic calcium oxalate or calcium phosphate stone, hypercalciuria (>250 mg/24 hrs), renal phosphate leak (TMP/GFR <2.07 mg/dl)

12. Idiopathic calcium nephrolithiasis without renal phosphate leak

13. Male patients > 18 years old

14. History of symptomatic calcium oxalate or calcium phosphate stone, hypercalciuria (>250 mg/24 hrs), renal phosphate leak (TMP/GFR <2.07 mg/dl)

Exclusion Criteria:

1. Exclusion for Dent disease include: primary or secondary hyperparathyroidism, hyperthyroidism, chronic diarrhea states; intake of thiazide diuretics, glucocorticoids, or estrogens within one month of the study.

2. Exclusion criteria for calcium stone formers include: primary or secondary hyperparathyroidism, hyperthyroidism, estimated GFR <40 ml/mn/1.73m2, chronic diarrhea states; intake of thiazide diuretics, glucocorticoids, or estrogens within one month of the study.

3. Exclusion criteria include history of symptomatic or asymptomatic kidney stone disease; primary or secondary hyperparathyroidism; estimated GFR <40 ml/min/1.73m2, chronic diarrhea states; intake of thiazide diuretics, glucocorticoids, or estrogens within one month of the study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Mayo Clinic	Rochester	Minnesota	United States	55905

Sponsors and Collaborators

• Mayo Clinic

Investigators

• Principal Investigator: John C Lieske, M.D., Mayo Clinic

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Dec 15, 2016

More Information

Additional Information:

• Mayo Clinic Clinical Trials

Publications

None provided.

• Responsible Party: John Lieske, M.D., Mayo Clinic
• ClinicalTrials.gov Identifier: NCT02016235
• Other Study ID Numbers: 13-004774
• First Posted: Dec 19, 2013
• Last Update Posted: Mar 23, 2020
• Last Verified: Mar 1, 2020

• Keywords provided by John Lieske, M.D., Mayo Clinic: Dent; Dents; Dent Disease; Phosphorus Supplements; FGF 23
• Additional relevant MeSH terms: Dent Disease

Study Results

Participant Flow

Arm/Group Title	Dent Disease Intervention	Kidney Stone Subjects	Dent Disease Observation
Arm/Group Description	Dent Disease subjects will receive 2 week supplementation with phosphorus Phosphorus Supplement: 250 mg po qid	Kidney stone with or without phosphate leak subjects will receive 2 week supplementation with phosphorus Phosphorus Supplement: 250 mg po qid	Dent disease subjects will not get phosphorus Observation: Baseline blood and urine measurements only
Period Title: Overall Study
STARTED	10	10	10
COMPLETED	10	10	10
NOT COMPLETED	0	0	0

Baseline Characteristics

Arm/Group Title	Dent Disease Intervention	Kidney Stone Subjects	Dent Disease Observation	Total
Arm/Group Description	Dent Disease subjects will receive 2 week supplementation with phosphorus Phosphorus Supplement: 250 mg po qid	Kidney stone with or without phosphate leak subjects will receive 2 week supplementation with phosphorus Phosphorus Supplement: 250 mg po qid	Dent disease subjects will not get phosphorus Observation: Baseline blood and urine measurements only	Total of all reporting groups
Overall Participants	10	10	10	30
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	26.4 (12.0)	55.3 (14.6)	10.2 (1.3)	30.6 (9.3)
Sex: Female, Male (Count of Participants)
Female	0 0%	0 0%	0 0%	0 0%
Male	10 100%	10 100%	10 100%	30 100%
Race and Ethnicity Not Collected (Count of Participants)
Count of Participants [Participants]	0 0%
Region of Enrollment (participants) [Number]
United States	10 100%	10 100%	10 100%	30 100%

Outcome Measures

1. Primary Outcome

Title	Change in Urine Total Protein
Description	Urine protein tests detect and/or measure protein being released into the urine. Normal urine protein elimination is less than 150 mg/day
Time Frame	baseline, day 7

Outcome Measure Data

Analysis Population Description

Data were not collected for the Disease Dent Observation Group

Arm/Group Title	Dent Disease Intervention	Kidney Stone Subjects	Dent Disease Observation
Arm/Group Description	Dent Disease subjects will receive 2 week supplementation with phosphorus Phosphorus Supplement: 250 mg po qid	Kidney stone with or without phosphate leak subjects will receive 2 week supplementation with phosphorus Phosphorus Supplement: 250 mg po qid	Dent disease subjects will not get phosphorus Observation: Baseline blood and urine measurements only. Day 7 Urine total protein not obtained
Measure Participants	10	10	0
Mean (Standard Deviation) [mg/day]	1681 (979)	59 (38)

Adverse Events

Time Frame	Adverse Events were collected over a period of 7 days for each subjects
Arm/Group Title	Dent Disease Intervention		Kidney Stone Subjects		Dent Disease Observation
Arm/Group Description	Dent Disease subjects will receive 2 week supplementation with phosphorus Phosphorus Supplement: 250 mg po qid		Kidney stone with or without phosphate leak subjects will receive 2 week supplementation with phosphorus Phosphorus Supplement: 250 mg po qid		Dent disease subjects will not get phosphorus Observation: Baseline blood and urine measurements only. Day 7 Urine total protein not obtained
All Cause Mortality
	Dent Disease Intervention		Kidney Stone Subjects		Dent Disease Observation
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/10 (0%)		0/10 (0%)		0/10 (0%)
Serious Adverse Events
	Dent Disease Intervention		Kidney Stone Subjects		Dent Disease Observation
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/10 (0%)		0/10 (0%)		0/10 (0%)
Other (Not Including Serious) Adverse Events
	Dent Disease Intervention		Kidney Stone Subjects		Dent Disease Observation
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/10 (0%)		0/10 (0%)		0/10 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.