Familial Hypophosphatemic RicketsCHRNCT00844740
Calcimimetics in Hypophosphatemic Rickets
A study testing Cinacalcet in people with Familial Hypophosphatemic Rickets, run by Children's Mercy Hospital Kansas City at 1 site in United States.
Trial summary
Currently, large oral doses of phosphate and 1,25(OH)2D (calcitriol) are the standard treatment of patients with familial hypophosphatemic rickets (XLH). While this therapy is effective in healing the rickets, it is often limited by development of complications due to the high dose of medications required to achieve… Read more about this study
- Who is it for?
- Ages 5 to 21 yearsHealthy volunteers can take part
- Conditions the study is for
- Familial Hypophosphatemic Rickets
- What is being tested?
- Cinacalcet
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Mar 2009to 1 Mar 2012
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 21 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Patients with XLH
- Age 5-21 years old
- From the Renal and Endocrinology Clinics who have been in a stable clinical, biochemical and radiological condition for the preceding 9 months
- Who have not received Cinacalcet before
Who cannot join
- Treatment with growth hormone
- Inability to swallow pills.
- An orthopedic surgery or braces in the past 9 months, or scheduled within 9 months of start.
- Pregnancy
- History of seizure disorder
- Abnormal liver functions (which may change the drug's AUC)
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Mercy Hospital
Kansas City, Missouri, 64108
About this study
Official title: Effect of Cinacalcet on the Long-term Treatment of Familial Hypophosphatemic Rickets
Currently, large oral doses of phosphate and 1,25(OH)2D (calcitriol) are the standard treatment of patients with familial hypophosphatemic rickets (XLH). While this therapy is effective in healing the rickets, it is often limited by development of complications due to the high dose of medications required to achieve cure. Among them are the development of calcifications in the kidneys and secondary hyperparathyroidism (HPT) which in some patients may cause complications like high blood calcium level, high blood pressure and damage to the kidney. A drug to treat secondary hyperparathyroidism was just developed. In a short term study we found that it might help the treatment of XLH, by allowing the use of lower doses of the both phosphate and calcitriol. In the present study we will learn if indeed the addition of this new medicine (Cinacalcet) to the long-term treatment will allow the use of lower doses of both phosphate and calcitriol and consequently lower the risk of complications.
- Conditions
- Familial Hypophosphatemic Rickets
- Being tested
- Cinacalcet
- Keywords
- Familial hypophosphatemic rickets, Cinacalcet, hyperparathyroidism
- Medical subject headings
- Familial Hypophosphatemic Rickets, Hyperparathyroidism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CinacalcetDrug
Also called Sensipar
Stable patients with XLH already treated with Phosphate and calcitriol will add Cinacalcet to their treatment regimen. Sequential monitoring of blood nand urine biochemical variables will follow, based on which adjustments to the doses of the 3 medications will be done.
| Study group | Role | What they receive |
|---|---|---|
| Cinacalcet | Experimental | Stable patients with XLH already treated with Phosphate and calcitriol will add Cinacalcet to their treatment regimen. Sequential monitoring of blood and urine biochemical variables will follow, based on which adjustments to the doses of the 3 medications will be done. Receives: Drug: Cinacalcet |
What is being measured
The primary outcome is the main question the study is designed to answer.
doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Feb 2009 (estimated)
- Study start1 Mar 2009 (estimated)
- Primary completion1 Mar 2012 (estimated)
- Study completion1 Mar 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial Hypophosphatemic RicketsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00844740
- Sponsor's protocol ID
- 08 09-152
- Acronym
- CHR
- Status last verified
- Dec 2020
- First submitted
- 12 Feb 2009
- Last updated
- 17 Dec 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Cinacalcet on the Long-term Treatment of Familial Hypophosphatemic Rickets
- ClinicalTrials.gov ID
- NCT00844740
- Acronym / sponsor ID
- CHR / 08 09-152
- Conditions
- Familial Hypophosphatemic Rickets
- Keywords
- Familial hypophosphatemic rickets, Cinacalcet, hyperparathyroidism
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Mercy Hospital Kansas CityUniversity, hospital or other
- Study start
- 1 Mar 2009 (estimated)
- Primary completion
- 1 Mar 2012 (estimated)
- Study completion
- 1 Mar 2012 (estimated)
- First posted
- 16 Feb 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Currently, large oral doses of phosphate and 1,25(OH)2D (calcitriol) are the standard treatment of patients with familial hypophosphatemic rickets (XLH). While this therapy is effective in healing the rickets, it is often limited by development of complications due to the high dose of medications required to achieve cure. Among them are the development of calcifications in the kidneys and secondary hyperparathyroidism (HPT) which in some patients may cause complications like high blood calcium level, high blood pressure and damage to the kidney. A drug to treat secondary hyperparathyroidism was just developed. In a short term study we found that it might help the treatment of XLH, by allowing the use of lower doses of the both phosphate and calcitriol. In the present study we will learn if indeed the addition of this new medicine (Cinacalcet) to the long-term treatment will allow the use of lower doses of both phosphate and calcitriol and consequently lower the risk of complications.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cinacalcet | Experimental | Drug: CinacalcetStable patients with XLH already treated with Phosphate and calcitriol will add Cinacalcet to their treatment regimen. Sequential monitoring of blood and urine biochemical variables will follow, based on which adjustments to the doses of the 3 medications will be done. |
Who may qualify
- Ages
- 5 to 21 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Patients with XLH
- Age 5-21 years old
- From the Renal and Endocrinology Clinics who have been in a stable clinical, biochemical and radiological condition for the preceding 9 months
- Who have not received Cinacalcet before
Key exclusion criteria (6 listed)
- Treatment with growth hormone
- Inability to swallow pills.
- An orthopedic surgery or braces in the past 9 months, or scheduled within 9 months of start.
- Pregnancy
- History of seizure disorder
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CinacalcetAlso: SensiparStable patients with XLH already treated with Phosphate and calcitriol will add Cinacalcet to their treatment regimen. Sequential monitoring of blood nand urine biochemical variables will follow, based on which adjustments to the doses of the 3 medications will be done. | Drug | Cinacalcet |
Primary outcome
doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries
Time frame: 9 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Cinacalcet in people with Familial Hypophosphatemic Rickets, run by Children's Mercy Hospital Kansas City at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 17 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries (time frame: 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Dec 2020.