Hypophosphatasia (HPP)BABSNCT04181164
Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)
An observational study of Hypophosphatasia (HPP), run by Hvidovre University Hospital at 1 site in Denmark.
Trial summary
The study aims to evaluate the bone architecture and bone strength in adults with Hypophosphatasia (HPP). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Hypophosphatasia (HPP)
- What is being tested?
- Microindentation, High resolution peripheral quantitative computed tomography (HRpQCT) and Biochemical analysis of different bone markers.
- How many people
- 30 participants
- Where
- 1 siteDenmark
- Study dates
- 1 Oct 2019to 30 Dec 2021
- Phase
- Observational
- 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 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Adults, diagnosed with HPP (genetic verified) (n=15), matched 1:1 in case of gender, age (± 5 years), BMI (± 3 kg/m2), postmenopausal status (± 2 years) with healthy controls.
Who may be able to join
- Genetically verified HPP
- Age: ≥ 18 years
- Persistently low levels of alkaline phosphatase (ALP) ≤ 35 U/L (normal range 35-105 U/L)
- At least one of the following symptoms: a) dental manifestations; b) musculoskeletal pain; c) history of fracture(s)
- Submitted informed consent
- No ALP measurements ≤ 45 U/l and ≥ 50% of all ALP measurements, registered in the electronical clinical journal ≥ 55 U/l
Show all 9Show fewer
- Normal parathyroid hormone (PTH) and Pyridoxal-5´-phosphate (PLP)
- Vitamin D3 ≥ 25 nmol/L
- Submitted informed consent
Who cannot join
- Pregnancy
- Skin infection or severe skin affection in the measurement area of microindentation
- Known allergy to Lidocain
- Former or current medical treatment influencing bone metabolism (oral corticosteroid > 12 weeks, former or current anti-osteoporosis treatment at any time (regardless drug holiday), all kind of sex steroids (excluding oral contraception), anti-convulsants)
- Current malignant disorders
- Family history of a genetic metabolic bone disease (HPP, Osteogenesis imperfecta)
Show all 17Show fewer
- Rickets in childhood
- Former or current Osteoporosis
- Former or current Osteomalacia
- Known diabetes
- Former or current medical treatment influencing bone metabolism (oral corticosteroid > 12 weeks, former or current anti-osteoporosis treatment at any time (regardless drug holiday), all kind of sex steroids (excluding oral contraception), anti- convulsants)
- Skin infection or severe skin affection in the measurement area of microindentation
- Chronic liver or gallbladder disease
- Current malignant disorders
- Former or current thyrotoxicosis (T4 over normal range ≥ 6 months)
- Cushing disease
- Pregnancy
Trial locations and contacts
1 site in 1 country.
About this study
The study aims to evaluate the bone architecture and bone strength in adults with Hypophosphatasia (HPP).
- Conditions
- Hypophosphatasia (HPP)
- Being tested
- Microindentation, High resolution peripheral quantitative computed tomography (HRpQCT), Biochemical analysis of different bone markers.
- Medical subject headings
- Hypophosphatasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MicroindentationOther
Microindentation is a new technology directly measuring bone strength by a minimal invasive technique.
High resolution peripheral quantitative computed tomography (HRpQCT)Other
HRpQCT scan can assess the cross-sectional geometry of the bone and is an appropriate investigation to evaluate bone quality.
Biochemical analysis of different bone markers.Biological
Measurement of different bone markers by biochemical analysis of blood samples.
| Study group | Role | What they receive |
|---|---|---|
| HPP-Group | Adults with hypophosphatasia. Receives: Other: Microindentation; Other: High resolution peripheral quantitative computed tomography (HRpQCT); Biological: Biochemical analysis of different bone markers. | |
| Control-Group | Healthy control subjects. Receives: Other: Microindentation; Other: High resolution peripheral quantitative computed tomography (HRpQCT); Biological: Biochemical analysis of different bone markers. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Differences in Bone Mineral Strength Index (BMSi) between the two groups, assessed by microindentation (OsteoProbe®).
Differences in BMSi between the HPP- and Control-Group will be evaluated by microindentation (OsteoProbe®).
Microindentation is a technology directly measuring bone strength by a minimal invasive technique. By applying a standardized pressure with a probe, which at the same time measures the indentation depth in the tibia bone, a measure of bone strength is obtained and calculated as Bone Mineral Strength Index (BMSi) [1].
Measured: 1. October 2019 - 31.July 2020
Secondary outcomes (3)
Correlation between BMSi and fracture prevalence in the HPP-Group and the Control-Group.
BMSi will be evaluated by microindentation (described above). In addition, information about the occurrence of fractures in the HPP- and Control-Group will be obtained by data from the Danish National Patient Register and a structured clinical interview.
Measured: 1. October 2019 - 31.July 2020
Evaluation of differences in bone microarchitecture between the HPP- and Control-Group by high resolution peripheral quantitative computed tomography (HRpQCT).
To asses differences in bone architecture between the two groups, the non-dominant distal radius and non-dominant distal tibia will be examined by HRpQCT, which will provide data about total, trabecular and cortical BMD, trabecular thickness, cortical thickness, trabecular number, stiffness and finite element failure load of the radius and tibia.
Measured: 1. October 2019 - 31.July 2020
Evaluation of differences in bone homeostasis between the two groups by biochemical analysis of different bone markers (P1NP, CTx, BALP, Trab-5, Sclerostin, Osteocalcin and FGF23)
Blood samples will be collected for biochemical analysis of different bone markers (P1NP, CTx, BALP, Trab-5, Sclerostin, Osteocalcin and FGF23).
BALP = Bone specific alkaline phosphatase CTx = Carboxy-terminal collagen crosslinks FGF-23 = Fibroblast growth factor 23 P1NP = Procollagen type 1 amino-terminal propeptide Trab-5 = Tartrate-resistant acid phosphatase-5
Measured: 1. October 2019 - 31.July 2020
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2019
- Study start1 Oct 2019
- Primary completion30 Sep 2021 (estimated)
- Study completion30 Dec 2021 (estimated)
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 Hypophosphatasia (HPP)Studies 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
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hvidovre University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Odense University Hospital
- Responsible party
- Principal investigator, Nicola Hepp, Hvidovre University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04181164
- Sponsor's protocol ID
- HvH-BABS-Study
- Acronym
- BABS
- Status last verified
- Aug 2021
- First submitted
- 18 Nov 2019
- Last updated
- 1 Sep 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Herrera S, Diez-Perez A. Clinical experience with microindentation in vivo in humans. Bone. 2017 Feb;95:175-182. doi: 10.1016/j.bone.2016.11.003. Epub 2016 Nov 11.
PubMed 27840302
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)
- ClinicalTrials.gov ID
- NCT04181164
- Acronym / sponsor ID
- BABS / HvH-BABS-Study
- Conditions
- Hypophosphatasia (HPP)
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Hvidovre University HospitalUniversity, hospital or other
- Collaborators
- Odense University Hospital
- Study start
- 1 Oct 2019 (actual)
- Primary completion
- 30 Sep 2021 (estimated)
- Study completion
- 30 Dec 2021 (estimated)
- First posted
- 29 Nov 2019 (actual)
- 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.
The study aims to evaluate the bone architecture and bone strength in adults with Hypophosphatasia (HPP).
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HPP-Group | – | Other: Microindentation, Other: High resolution peripheral quantitative computed tomography (HRpQCT), Biological: Biochemical analysis of different bone markers.Adults with hypophosphatasia. |
| Control-Group | – | Other: Microindentation, Other: High resolution peripheral quantitative computed tomography (HRpQCT), Biological: Biochemical analysis of different bone markers.Healthy control subjects. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Adults, diagnosed with HPP (genetic verified) (n=15), matched 1:1 in case of gender, age (± 5 years), BMI (± 3 kg/m2), postmenopausal status (± 2 years) with healthy controls.
Key inclusion criteria (9 listed)
- Genetically verified HPP
- Age: ≥ 18 years
- Persistently low levels of alkaline phosphatase (ALP) ≤ 35 U/L (normal range 35-105 U/L)
- At least one of the following symptoms: a) dental manifestations; b) musculoskeletal pain; c) history of fracture(s)
- Submitted informed consent
Key exclusion criteria (17 listed)
- Pregnancy
- Skin infection or severe skin affection in the measurement area of microindentation
- Known allergy to Lidocain
- Former or current medical treatment influencing bone metabolism (oral corticosteroid > 12 weeks, former or current anti-osteoporosis treatment at any time (regardless drug holiday), all kind of sex steroids (excluding oral contraception), anti-convulsants)
- Current malignant disorders
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 |
|---|---|---|
| MicroindentationMicroindentation is a new technology directly measuring bone strength by a minimal invasive technique. | Other | Control-Group, HPP-Group |
| High resolution peripheral quantitative computed tomography (HRpQCT)HRpQCT scan can assess the cross-sectional geometry of the bone and is an appropriate investigation to evaluate bone quality. | Other | Control-Group, HPP-Group |
| Biochemical analysis of different bone markers.Measurement of different bone markers by biochemical analysis of blood samples. | Biological | Control-Group, HPP-Group |
Primary outcome
Differences in Bone Mineral Strength Index (BMSi) between the two groups, assessed by microindentation (OsteoProbe®).
Time frame: 1. October 2019 - 31.July 2020
Differences in BMSi between the HPP- and Control-Group will be evaluated by microindentation (OsteoProbe®). Microindentation is a technology directly measuring bone strength by a minimal invasive technique. By applying a standardized pressure with a probe, which at the same time measures the indentation depth in the tibia bone, a measure of bone strength is obtained and calculated as Bone Mineral Strength Index (BMSi) [1].
3 secondary outcomes
Correlation between BMSi and fracture prevalence in the HPP-Group and the Control-Group.
Time frame: 1. October 2019 - 31.July 2020
Evaluation of differences in bone microarchitecture between the HPP- and Control-Group by high resolution peripheral quantitative computed tomography (HRpQCT).
Time frame: 1. October 2019 - 31.July 2020
Evaluation of differences in bone homeostasis between the two groups by biochemical analysis of different bone markers (P1NP, CTx, BALP, Trab-5, Sclerostin, Osteocalcin and FGF23)
Time frame: 1. October 2019 - 31.July 2020
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2021.
- Background: PubMed 27840302Herrera S, Diez-Perez A. Clinical experience with microindentation in vivo in humans. Bone. 2017 Feb;95:175-182. doi: 10.1016/j.bone.2016.11.003. Epub 2016 Nov 11.
- Individual participant dataSharing planned: No
Related trials for Hypophosphatasia (HPP)
Enrolling by invitationObservational study
Registry of Patients With Hypophosphatasia
Hypophosphatasia (HPP)
Enrolling by invitationObservational study
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
Osteoporosis
RecruitingObservational study
Clarify HPP: Disease Burden of HPP Patients in China
Hypophosphatasia (HPP)
RecruitingObservational study
The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia
Hypophosphatasia
RecruitingPhase 1/2
A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1
Hypophosphatasia (HPP)
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?
An observational study of Hypophosphatasia (HPP), run by Hvidovre University Hospital at 1 site in Denmark.
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 9 inclusion and 17 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: status not confirmed, as of the sponsor's last update on 1 Sep 2021. 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 Denmark. 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?
Results are not available in the source record.
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?
Differences in Bone Mineral Strength Index (BMSi) between the two groups, assessed by microindentation (OsteoProbe®). (time frame: 1. October 2019 - 31.July 2020). 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 NCT04181164, 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 1 Sep 2021.