Pseudoxanthoma ElasticumPhase 2NCT04441671
Oral Pyrophosphate Absorption in PXE Disease
A Phase 2 study testing Disodium Pyrophosphate in people with Pseudoxanthoma Elasticum, run by Tampere University Hospital.
Trial summary
Pyrophosphate is an endogenous, non-toxic metabolite inhibiting soft tissue calcification. The aim of our study is to find optimal dosing and safety of oral disodium-PPi (Na2H2PPi). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Pseudoxanthoma Elasticum
- What is being tested?
- Disodium Pyrophosphate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 8 Dec 2020to 1 Apr 2021
- Phase
- Phase 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults (>18 yrs)
- clinically and genetically proven PXE
- Body mass index (BMI) 18,5-34,9 kg/m2
Who cannot join
- Special groups according to researchers' decision.
- Pregnancy
- No effective contraception in females in child-bearing age.
About this study
Official title: Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)
Pyrophosphate is an endogenous, non-toxic metabolite inhibiting soft tissue calcification. The aim of our study is to find optimal dosing and safety of oral disodium-PPi (Na2H2PPi). Absorption curves (pharmacokinetics), AUC0-t, Cmax and Tmax for PPi and phosphate will be provided for healthy controls and PXE-patients both fasting and with standard meal intake.
Read the detailed description
Phase II oral capsulized disodium-PPi (Na2H2PPi) powder absorption study in subjects with PXE (n=8-12) will be done in the ward of Internal Medicine of Tampere University Hospital and University Medical Center Utrecht.
After a 10-hour fast at 8.00 a.m. 30 mg/kg (first day) and 50 mg/kg (second day) capsulized dose of PPi will be given with 2 dl water. At 12.00 a.m. another capsulized 30 mg/kg (first day) or 50 mg/kg (second day) single dose of PPi with a standard mixed meal (lunch) will be given with 2 dl water at the time when a subject starts eating.
Plasma and urine sampling include plasma and spot urine electrolytes, creatinine, and pyrophosphate. Plasma sampling will be done at 0, 15, 30, 60, 120 and 240 min after ingestion of PPi. Urine spot sample will be taken at 0 and 240 min after ingestion of PPi.
Physical activity is restricted. Side-effects will be recorded.
- Conditions
- Pseudoxanthoma Elasticum
- Being tested
- Disodium Pyrophosphate
- Medical subject headings
- Pseudoxanthoma Elasticum
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Disodium PyrophosphateDrug
Also called Na2H2PPi
Absorption trial
| Study group | Role | What they receive |
|---|---|---|
| Open label | Experimental | Disodiumpyrophosphate, capsuled powder, First day: 30 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Second day: 50 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Receives: Drug: Disodium Pyrophosphate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Concentration area under the curve 0-t of pyrophosphate
Measured: two days
Maximal concentration of pyrophosphate
Cmax
Measured: two days
Timepoint of maximal pyrophosphate concentration
Tmax
Measured: two days
Secondary outcomes (3)
Concentration area under the curve 0-t of phosphate
AUC0-t
Measured: two days
Maximal concentration of phosphate
Cmax
Measured: two days
Timepoint of maximal phosphate concentration
Tmax
Measured: two days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2020
- Study start8 Dec 2020
- Primary completion1 Apr 2021
- Study completion1 Apr 2021
Why it stopped: Withdrawn
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pseudoxanthoma ElasticumStudies of the same condition, open and under way.
- More trials run by Tampere University HospitalThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tampere University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- UMC Utrecht, Hungarian Academy of Sciences
- Responsible party
- Sponsor
- Investigators
- Pasi I Nevalainen, MD, PhD, Principal investigator, Tampere University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04441671
- Sponsor's protocol ID
- R19017M
- Other IDs
- 2019-002109-24, KLnro 38/2019, 2019
- Status last verified
- Apr 2021
- First submitted
- 20 Jan 2020
- Last updated
- 22 Apr 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Only anonymized data can be shared
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)
- ClinicalTrials.gov ID
- NCT04441671
- Acronym / sponsor ID
- R19017M
- Conditions
- Pseudoxanthoma Elasticum
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Tampere University HospitalUniversity, hospital or other
- Collaborators
- UMC Utrecht, Hungarian Academy of Sciences
- Study start
- 8 Dec 2020 (actual)
- Primary completion
- 1 Apr 2021 (actual)
- Study completion
- 1 Apr 2021 (actual)
- First posted
- 22 Jun 2020 (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.
Pyrophosphate is an endogenous, non-toxic metabolite inhibiting soft tissue calcification. The aim of our study is to find optimal dosing and safety of oral disodium-PPi (Na2H2PPi). Absorption curves (pharmacokinetics), AUC0-t, Cmax and Tmax for PPi and phosphate will be provided for healthy controls and PXE-patients both fasting and with standard meal intake.
Detailed description
Phase II oral capsulized disodium-PPi (Na2H2PPi) powder absorption study in subjects with PXE (n=8-12) will be done in the ward of Internal Medicine of Tampere University Hospital and University Medical Center Utrecht.
After a 10-hour fast at 8.00 a.m. 30 mg/kg (first day) and 50 mg/kg (second day) capsulized dose of PPi will be given with 2 dl water. At 12.00 a.m. another capsulized 30 mg/kg (first day) or 50 mg/kg (second day) single dose of PPi with a standard mixed meal (lunch) will be given with 2 dl water at the time when a subject starts eating.
Plasma and urine sampling include plasma and spot urine electrolytes, creatinine, and pyrophosphate. Plasma sampling will be done at 0, 15, 30, 60, 120 and 240 min after ingestion of PPi. Urine spot sample will be taken at 0 and 240 min after ingestion of PPi.
Physical activity is restricted. Side-effects will be recorded.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open label | Experimental | Drug: Disodium PyrophosphateDisodiumpyrophosphate, capsuled powder, First day: 30 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Second day: 50 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Adults (>18 yrs)
- clinically and genetically proven PXE
- Body mass index (BMI) 18,5-34,9 kg/m2
Key exclusion criteria (3 listed)
- Special groups according to researchers' decision.
- Pregnancy
- No effective contraception in females in child-bearing age.
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 |
|---|---|---|
| Disodium PyrophosphateAlso: Na2H2PPiAbsorption trial | Drug | Open label |
Primary outcome
Concentration area under the curve 0-t of pyrophosphate
Time frame: two days
Maximal concentration of pyrophosphate
Time frame: two days
Cmax
Timepoint of maximal pyrophosphate concentration
Time frame: two days
Tmax
3 secondary outcomes
Concentration area under the curve 0-t of phosphate
Time frame: two days
Maximal concentration of phosphate
Time frame: two days
Timepoint of maximal phosphate concentration
Time frame: two days
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 22 Apr 2021.
- Individual participant dataSharing planned: No. Only anonymized data can be shared
Related trials for Pseudoxanthoma Elasticum
Recruiting
Purinergic Compounds in Pseudoxanthoma Elasticum
Pseudoxanthoma Elasticum
RecruitingObservational study
Impact of Optimized Recruitment and Follow-up of Patients With Pseudoxanthoma Elasticum (PXE)
Pseudoxanthoma Elasticum
Recruiting
CHOPXE - Analysis of Choriocapillaris Flow Deficits in Patients With Pseudoxanthoma Elasticum
Pseudoxanthoma Elasticum, Tomography, Optical Coherence
RecruitingObservational study
Progression Assessment of PXE-associated Alterations
Pseudoxanthoma Elasticum, Angioid Streaks
Active, not recruitingObservational study
Progression Rate of Pseudoxanthoma Elasticum-associated Choroidal and Retinal Degeneration
Pseudoxanthoma Elasticum
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.
Pasi I Nevalainen, MD, PhD principal investigator
Tampere University Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Disodium Pyrophosphate in people with Pseudoxanthoma Elasticum, run by Tampere University 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 3 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 22 Apr 2021. 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 phase mean?
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 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?
Concentration area under the curve 0-t of pyrophosphate (time frame: two days). The study lists 3 primary outcomes in all. 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 22 Apr 2021.