The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia
ConditionHypophosphatasia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorAstraZeneca
About this trial
The effect of monoallelic variants in the ALPL gene on the natural course of hypophosphatasia (HPP) in children and adults in Russia (ATLANTIS)
Eligibility criteria
Qualifiers
Age ≥4 to <18 years, or ≥18 years at the time of enrollment;
Signed ICF for patients ≥18 years, or legal representatives (parents) of patients aged ≥4 to <18 years;
Written informed assent (for patients aged ≥14 to <18 years only);
No history of HPP treatment with enzyme-replacement therapy;
Disqualifiers
Confirmed conditions presenting with clinical features overlapping with HPP, including but not limited to: cerebral palsy, Duchenne muscular dystrophy, limb-girdle muscular dystrophy (Erb-Roth dystrophy), acquired secondary myopathies of various etiologies;
Сurrent participation in any clinical study (patients participating in other non interventional studies may be included);
Homozygous or compound heterozygous mutation in the ALPL gene
In the opinion of the investigator, the patient is not able to return for follow-up visits or obtain required follow-up studies.
Trial design
Treatments tested in this trial
- Not listed
Trial groups
No trial groups listed
Sponsors and collaborators
Source ClinicalTrials.gov