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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 12 months
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
I have XLH without male-to-male transmission in my family, or I have a PHEX mutation.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 12 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 12 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from baseline in TmP/GFR in adults and children with XLH
Change from baseline in nephrocalcinosis grade
Change from baseline in serum phosphate in adults and children with XLH
+1 moreSecondary outcome measures
Growth in children with XLH
Side effects data
From 2010 Phase 4 trial • 109 Patients • NCT0126561523%
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
7%
Viral Infection
7%
Leg Cramps
7%
Hypercalcemia
7%
Allergic Infection
7%
Polydipsia
7%
Dehydration
7%
Urinary Tract Infection
3%
Chest Pain
3%
Sinusitis
3%
Headache
3%
General Infection
3%
Asthenia
3%
Fever
3%
Infection Fungal
3%
Conjuctivitis
3%
Syncope
3%
Depression
3%
Increased Cough
3%
Polyuria
3%
Abdominal Pain
3%
Photophobia
3%
Decreased libido
3%
Hypotension
3%
Nausea
3%
Esophageal ulcer
3%
Somnolence
3%
Back Pain
3%
Vomiting
3%
Pruritus
100%
80%
60%
40%
20%
0%
Study treatment Arm
Paricalcitol Treatment
Calcitriol Treatment
Cholecalciferol
Supplemental
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
Find a Location
Who is running the clinical trial?
Massachusetts General HospitalLead Sponsor
2,933 Previous Clinical Trials
13,198,471 Total Patients Enrolled
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NIH
484 Previous Clinical Trials
1,086,987 Total Patients Enrolled
2 Trials studying Hypophosphatemia
54 Patients Enrolled for Hypophosphatemia
Eva Liu, MDPrincipal Investigator - Massachusetts General Hospital and Brigham and Women's Hospital
Massachusetts General Hospital
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had cancer before, but not skin cancer.You have a problem with using drugs or alcohol, as defined by the DSM-5.Your blood calcium levels are lower than 10.0 mg/dl.I have XLH without male-to-male transmission in my family, or I have a PHEX mutation.You have a history of serious mental health issues according to the DSM-5.Your blood has too little parathyroid hormone.I am taking medication that affects how my kidneys handle minerals.I am currently using growth hormone therapy.I have used steroids for more than 2 weeks in the last year, not counting inhalers.I have not taken cinacalcet in the last two weeks.You are allergic to calcitriol.I am not using medications that affect bone health, except for birth control pills.Your vitamin D level is 20 ng/dL or higher.I do not have severe heart or lung disease.I cannot or will not stop taking calcitriol and phosphate treatments for two weeks before the study.Your kidney function is good, with an estimated glomerular filtration rate (eGFR) of 60 or higher.
Research Study Groups:
This trial has the following groups:- Group 1: Adults with XLH
- Group 2: Children with XLH
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How many participants are currently included in this clinical exploration?
"Affirmative, clinicaltrials.gov indicates that this research is still enrolling subjects. Initially posted on March 28th 2019, the study's most recent amendment was made on March 16th 2022 and it requires 20 volunteers from one location to participate in its trial."
Answered by AI
What criteria must a person fulfill in order to become involved with this experiment?
"Candidates who possess hypophosphatemic rickets, x linked dominant and fall between the age range of 3 to 70 have an opportunity to gain entry into this clinical trial. There is a maximum capacity for 20 participants in total."
Answered by AI
Are people aged thirty and above eligible for this clinical experiment?
"This medical study is open to patients who are between 3 and 70 years of age. Additionally, 12 pediatric trials have been created for those under 18 while 9 geriatric studies are offered to applicants over the age of 65."
Answered by AI
What conditions is Calcitriol commonly prescribed to treat?
"Primarily prescribed for kidney failure, calcitriol can also assist sufferers of hyperparathyroidism, secondary hypocalcemia and psoriasis vulgaris."
Answered by AI
Can you elaborate on the other experiments utilizing Calcitriol that have already been performed?
"Currently, there are 7 active trials into the effects of calcitriol with 1 in its terminal stage. Most of these researches are based out of Beijing but 72 other sites have enrolled patients for this particular study."
Answered by AI
Does this experiment currently require additional participants?
"Affirmative. Clinicaltrials.gov verifies that this trial, which was posted on March 28th 2019, is currently recruiting patients for their study. With 20 participants needed from 1 location, the trial has recently been renewed as of March 16th 2022."
Answered by AI
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