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Vitamin D Analog

Calcitriol for Hypophosphatemic Rickets

Phase < 1

Recruiting

Led By Eva Liu, MD

Research Sponsored by Massachusetts General Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinical diagnosis of XLH with family history excluding male-to-male transmission, or positive genotype for PHEX mutation

Must not have

History of malignancy except basal and squamous cell carcinoma of the skin

Use of diuretics or medications that alter renal handling of mineral ions

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 12 months

Awards & highlights

No Placebo-Only Group

Summary

This trial will test the effects of calcitriol (a vitamin D hormone) on people with XLH, a disorder that causes phosphate levels and skeletal mineralization to be too low.

Who is the study for?

This trial is for children and adults with X-Linked Hypophosphatemia (XLH) who have normal to slightly elevated parathyroid hormone levels, adequate kidney function, sufficient vitamin D levels, a clinical diagnosis of XLH (with specific genetic criteria or family history), and normal serum calcium. It excludes those on certain medications, with serious health conditions like unstable heart disease, pregnant or breastfeeding women, recent cancer patients (except some skin cancers), individuals with severe psychiatric disorders or substance use disorders per DSM-5 guidelines.

What is being tested?

The study tests if calcitriol alone can improve phosphate levels and bone mineralization in XLH without causing kidney problems. Participants will receive escalating doses of calcitriol over the first three months and continue treatment for one year. The effect on growth in children will also be assessed.

What are the potential side effects?

Potential side effects from calcitriol may include high blood calcium levels which could lead to confusion, bone pain, nausea; increased risk of kidney stones; frequent urination; weakness; dry mouth; muscle pain; metallic taste in mouth.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have XLH without male-to-male transmission in my family, or I have a PHEX mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had cancer before, but not skin cancer.

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I am taking medication that affects how my kidneys handle minerals.

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I am currently using growth hormone therapy.

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I do not have severe heart or lung disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from baseline in TmP/GFR in adults and children with XLH

Change from baseline in nephrocalcinosis grade

Rickets score for children with XLH

Secondary study objectives

Growth in children with XLH

Side effects data

From 2010 Phase 4 trial • 109 Patients • NCT01265615

23%

Hypophospatemia

17%

Fatigue

13%

Increased hypertension

13%

Edema

13%

Diarrhea

13%

Pain

10%

Taste perversions

10%

Arthritis

10%

Dizziness

10%

Gastroenteritis

10%

Vertigo

10%

Rhinitis

10%

Bronchitis

10%

Rash

Leg Cramps

Viral Infection

Hypercalcemia

Allergic Infection

Polydipsia

Dehydration

Urinary Tract Infection

Chest Pain

Sinusitis

Headache

General Infection

Asthenia

Fever

Infection Fungal

Conjuctivitis

Syncope

Depression

Increased Cough

Polyuria

Abdominal Pain

Photophobia

Decreased libido

Hypotension

Nausea

Esophageal ulcer

Somnolence

Back Pain

Vomiting

Pruritus

100%

80%

60%

40%

20%

Study treatment Arm

Paricalcitol Treatment

Calcitriol Treatment

Cholecalciferol

Supplemental

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Children with XLHExperimental Treatment1 Intervention

Children (age 3-17) with X-linked hypophosphatemia will be treated with optimized doses of calcitriol (without phosphate supplementation) for one year

Group II: Adults with XLHExperimental Treatment1 Intervention

Adults with X-linked hypophosphatemia will be treated with optimized doses of calcitriol (without phosphate supplementation) for one year

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Calcitriol

2014

Completed Phase 4

~1510

0 / 5Eligibility criteria met