An observational study in Hypophosphatasia, sponsored by Dr. Lothar Seefried. Completed at 1 site in Germany. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-07-12.
Sponsored by Dr. Lothar Seefried · Observational
Among 13 studies in Hypophosphatasia with a site in Germany — 4 open to participants now, 5 under way, 1 of them phase 3. Hypophosphatasia · Germany.
Among the 13 studies in Hypophosphatasia with a site in Germany, this one is the only one that started in 2018, is one of 7 observational ones, and was the 7th registered.
46 studies on the registry are indexed under Hypophosphatasia; 11 are open to participants now.
Browse Hypophosphatasia studies →4 of the 13 studies in Hypophosphatasia with a site in Germany are open now. Recruiting first, the newest first:
Alesta Therapeutics · Sites in Germany, New Zealand, United Kingdom
Ages 18–50 · Healthy volunteers
University of Bonn · Sites in Germany
Ages 18+
University of Bonn · Sites in Germany
Ages 18+
Alexion Pharmaceuticals, Inc. · Sites in Germany, United States, Australia and 10 more countries
A study can be open while its sites in Germany have not opened yet or have stopped. Only those recruiting at a site in Germany →
Of the 9 Hypophosphatasia studies recruiting now, 8 set no upper age limit and 1 stops at 75 or younger; 5 take children under 18; 1 takes healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
13 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 2 · Phase 2 5 · Phase 3 2
First in 2010; this study’s year is marked. 1 started in 2018.
This study's enrollment of 23 is below the median of 58 across 24 observational studies indexed under Hypophosphatasia.
Median enrollment 48 across the 6 interventional studies, 60 across the 7 observational. Of the 8 that completed or were terminated, 4 (50%) have results posted.
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9 of 13 have an industry lead sponsor. The 8 most frequent:
This is the only study on the registry with Dr. Lothar Seefried as lead sponsor.
Dr. Lothar Seefried has 1 study filed under Hypophosphatasia — fewer than ten, so there is no comparison to draw.
2 of the studies in Hypophosphatasia with a site in Germany that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07179640Alesta TherapeuticsA 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 | 2027-01 |
| NCT06574282University of BonnCharacteristics of Hypophosphatasia in Adult Patients in Rheumatology and Their Value in Developing an Algorithm to HPP-diagnosis - the COHIR Multi-center Study | 2027-09 |
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Among the 13 studies in Hypophosphatasia with a site in Germany, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
Hypophosphatasia is a rare inherited metabolic disorder due to inactivating mutations of the ALPL-Gene. Particularly among adult patients, clinical manifestation exhibits a broad range of signs and symptoms, most commonly associated with musculoskeletal disabilities and compromised quality of life. Enzyme replacement therapy with Asfotase alfa (AA) is available and approved for patients with pediatric onset of the disease.
This single-center observational cohort study aims at collecting clinical routine data regarding the course treatment, quality of life and physical performance in patients treated with Asfotase alfa in line with the label for pediatric-onset hypophosphatasia.
Study population consists of male and female HPP patients with pediatric-onset HPP currently being managed for the disease at the Orthopedic Institute of the Julius-Maximilians-University Würzburg, Germany and receiving commercial AA treatment as per the standard of care.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Physical Performance
6 Minute Walk Test \[meters\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Physical Performance
Short Physical Performance Battery \[points\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Physical Performance
Timed Up and Go Test \[seconds\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Physical Performance
Handheld dynamometry \[kilogram\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Physical Performance
Jumping mechanography \[W/kg bodyweight\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Physical Performance
Use of assistive devices descriptive measure
Time frame: 1 year of treatment compared to pre-treatment baseline
Body composition
Bioelectrical Impedance Analysis \[proportional mass of muscle, water and fat in kg\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Health related quality of life
Questionnaire
Time frame: 1 year of treatment compared to pre-treatment baseline
Bone Mineral Density
Dual x-ray absorptiometry (DXA)
Time frame: 1 year of treatment compared to pre-treatment baseline
Skeletal pathology
Descriptive assessment of available imaging (x-Ray, CT, MRI, histological sections)
Time frame: 1 year of treatment compared to pre-treatment baseline
Laboratory evaluation
Parathyroid hormone \[PTH, pg/ml\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Laboratory evaluation
Serum-Calcium \[mmol/l\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Laboratory evaluation
Serum-Phosphorus \[mmol/l\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Laboratory evaluation
Serum-Creatinine \[µmol/l\], incl. calculated eGFR
Time frame: 1 year of treatment compared to pre-treatment baseline
Laboratory evaluation
Fibroblast Growth Factor 23 / FGF-23 \[RU/ml\]
Time frame: 1 year of treatment compared to pre-treatment baseline
Safety assessment
Injection site reactions (descriptive)
Time frame: 1 year of treatment compared to pre-treatment baseline
Safety assessment
Injection associated reactions (descriptive)
Time frame: 1 year of treatment compared to pre-treatment baseline
Safety assessment
Ectopic calcifications (descriptive)
Time frame: 1 year of treatment compared to pre-treatment baseline
Safety assessment
Adverse events (descriptive)
Time frame: 1 year of treatment compared to pre-treatment baseline
Plan to share: No
No publications or documents are linked to this record.
Ran from Sep 2018 to primary completion in May 2021, 32 months.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jul 2022. You cannot join it, but the record below documents what was studied.
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