X Linked Hypophosphatemic RicketsPhase 1/2GH-XLHNCT02720770
Growth Hormone Treatment in Children With Hypophosphatemic Rickets
A Phase 1/2 study testing norditropine simplex in people with X Linked Hypophosphatemic Rickets, run by Bicetre Hospital.
Trial summary
This study evaluates the effect on height of a two year treatment with growth hormone in 19 children with X linked hypophosphatemic rickets. Read more about this study
- Who is it for?
- Ages 3 to 14 years
- Conditions the study is for
- X Linked Hypophosphatemic Rickets
- What is being tested?
- norditropine simplex
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- No sites listed yet
- Study dates
- Jan 2006to Jan 2011
- Phase
- Phase 1/2
- 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 3 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- clinical, biochemical and genetic diagnosis of XLH
- height SDS < 2
- at least two years of treatment with oral phosphate and calcitriol
Who cannot join
- uncontrolled rickets (ALP>600 IU)
- growth hormone deficiency
- hyperparathyroidism, nephrocalcinosis, renal insufficiency
- associated disease
- previous treatment with growth hormone
About this study
Official title: Proposition Pour un Traitement Par Hormone de Croissance Des Enfants Atteints de Rachitisme Hypophosphatemique Familial
This study evaluates the effect on height of a two year treatment with growth hormone in 19 children with X linked hypophosphatemic rickets.
- Conditions
- X Linked Hypophosphatemic Rickets
- Being tested
- norditropine simplex
- Keywords
- growth hormone
- Medical subject headings
- Familial Hypophosphatemic Rickets
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
norditropine simplexDrug
Also called growth hormone
| Study group | Role | What they receive |
|---|---|---|
| norditropine simplex | Experimental | Not described Receives: Drug: norditropine simplex |
What is being measured
The primary outcome is the main question the study is designed to answer.
change from baseline in height SDS (standard deviation score)
Measured: at year one and year two from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2016 (estimated)
- Study startJan 2006
- Primary completionJan 2009
- Study completionJan 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for X Linked 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bicetre Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alessia Usardi, Bicetre Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02720770
- Sponsor's protocol ID
- RBM 03-47
- Acronym
- GH-XLH
- Status last verified
- Mar 2016
- First submitted
- 15 Mar 2016
- Last updated
- 28 Mar 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Rothenbuhler A, Esterle L, Gueorguieva I, Salles JP, Mignot B, Colle M, Linglart A. Two-year recombinant human growth hormone (rhGH) treatment is more effective in pre-pubertal compared to pubertal short children with X-linked hypophosphatemic rickets (XLHR). Growth Horm IGF Res. 2017 Oct;36:11-15. doi: 10.1016/j.ghir.2017.08.001. Epub 2017 Aug 15.
PubMed 28822957
Study at a glance
From the registry record, last updated by the sponsor on 28 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Proposition Pour un Traitement Par Hormone de Croissance Des Enfants Atteints de Rachitisme Hypophosphatemique Familial
- ClinicalTrials.gov ID
- NCT02720770
- Acronym / sponsor ID
- GH-XLH / RBM 03-47
- Conditions
- X Linked Hypophosphatemic Rickets
- Keywords
- growth hormone
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Bicetre HospitalUniversity, hospital or other
- Study start
- Jan 2006
- Primary completion
- Jan 2009 (actual)
- Study completion
- Jan 2011 (actual)
- First posted
- 28 Mar 2016 (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.
This study evaluates the effect on height of a two year treatment with growth hormone in 19 children with X linked hypophosphatemic rickets.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| norditropine simplex | Experimental | Drug: norditropine simplex |
Who may qualify
- Ages
- 3 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- clinical, biochemical and genetic diagnosis of XLH
- height SDS < 2
- at least two years of treatment with oral phosphate and calcitriol
Key exclusion criteria (5 listed)
- uncontrolled rickets (ALP>600 IU)
- growth hormone deficiency
- hyperparathyroidism, nephrocalcinosis, renal insufficiency
- associated disease
- previous treatment with growth hormone
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 |
|---|---|---|
| norditropine simplexAlso: growth hormone | Drug | norditropine simplex |
Primary outcome
change from baseline in height SDS (standard deviation score)
Time frame: at year one and year two from baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Mar 2016.
- Derived from this study: PubMed 28822957Rothenbuhler A, Esterle L, Gueorguieva I, Salles JP, Mignot B, Colle M, Linglart A. Two-year recombinant human growth hormone (rhGH) treatment is more effective in pre-pubertal compared to pubertal short children with X-linked hypophosphatemic rickets (XLHR). Growth Horm IGF Res. 2017 Oct;36:11-15. doi: 10.1016/j.ghir.2017.08.001. Epub 2017 Aug 15.
- Individual participant dataSharing planned: No
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing norditropine simplex in people with X Linked Hypophosphatemic Rickets, run by Bicetre Hospital.
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 3 inclusion and 5 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: finished, as of the sponsor's last update on 28 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.
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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
change from baseline in height SDS (standard deviation score) (time frame: at year one and year two from baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02720770, which also lists 1 linked publication.
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 28 Mar 2016.