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HypophosphatasiaATLANTISNCT07390240

The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia

An observational study of Hypophosphatasia, run by AstraZeneca at 4 sites in Russia.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

The effect of monoallelic variants in the ALPL gene on the natural course of hypophosphatasia (HPP) in children and adults in Russia (ATLANTIS) Read more about this study

Who is it for?
Any age
Conditions the study is for
Hypophosphatasia
How many people
55 participants
Where
4 sitesRussia
Study dates
29 Dec 2025to 30 Jun 2030
Phase
Observational
Are there results?
Not yet: the study is under way

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 Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: This multicenter observational study will include 70 paediatric and adult patients newly diagnosed with HPP who carry monoallelic variants in the ALPL gene and are monitored by physicians of various specialties at 20 clinical centres in different regions of Russia. For inclusion to this study, patients should have a documented history of at least 2 reduced alkaline phosphatase (ALP) values relative to age- and sex-specific reference range measured on separate occasions and an HPP diagnosis, but no history of enzyme-replacement therapy. The specific study inclusion and exclusion criteria are described in the sections below.

Who may be able to join

  • 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;
  • Diagnosis of symptomatic HPP confirmed by the presence of ALL of the following criteria:
  • reduced alkaline phosphatase (ALP) activity relative to age- and sex-specific reference ranges, confirmed by at least two separate measurements;
Show all 11Show fewer
  • the identification of a monoallelic pathogenic, likely pathogenic, or variant of uncertain significance in the ALPL gene on genetic testing;
  • presence of clinically relevant manifestations*, consistent with HPP, confirmed by at least one of the following: i. Musculoskeletal pain requiring medical intervention or limiting activities of daily living, present at screening or documented within the previous 12 months (e.g., based on medical records, functional test results, treatment prescriptions and/or rehabilitation interventions); chronic or recurrent pain lasting ≥3 months may be considered based on medical history beyond the 12-month period prior to screening, provided appropriate documentation is available; ii. Muscle weakness and/or reduced physical function (e.g., impaired mobility, increased fatigue), present at screening or documented within the previous 12 months (e.g., based on medical records, functional test results, treatment prescriptions and/or rehabilitation interventions); iii. History of fractures, pseudofractures, or delayed fracture healing, confirmed by medical documentation and/or imaging studies performed within 24 months prior to screening (provided no subsequent interventions have occurred that may have altered the findings; if such interventions have occurred, repeat imaging may be performed at the investigator's discretion, if applicable within routine clinical practice); iv. Skeletal abnormalities (e.g., radiographic signs of rickets/osteomalacia), confirmed by medical documentation and/or imaging studies performed within 24 months prior to screening (provided no subsequent interventions have occurred that may have altered the findings; if such interventions have occurred, repeat imaging may be performed at the investigator's discretion, if applicable within routine clinical practice); v. Dental manifestations (e.g., premature loss of deciduous teeth with intact roots before the age of 5 years without significant trauma), confirmed by medical and/or dental records; vi. Limitation in mobility, endurance, or activities of daily living, present at screening or documented within the previous 12 months based on routine clinical assessment using questionnaires, scales, functional tests and/or other relevant clinical tools used in routine practice; such tools may include, for example, the 6-minute walk test (6MWT), Timed Up-and-Go test, Chair-Rise test, Borg scale, IPAQ, PedsQL, EQ 5D-3L, bioimpedance measurements, and other methods used in routine clinical practice; if symptoms are present at screening, confirmation may be based on clinical assessments performed within 3 months prior to screening (provided there are no clinically significant changes in the patient's condition) or at the screening visit; vii. Need for analgesics, physiotherapy, and/or other supportive care for clinical manifestations of HPP, present at screening or during the period prior to screening, confirmed by at least one of the following criteria (based on medical records):
  • regular use of analgesics (≥3 times per week for at least 4 consecutive weeks) within the previous 6 months, or escalation of analgesic therapy within the previous 6 months;
  • ≥1 structured course of physiotherapy and/or rehabilitation interventions prescribed for musculoskeletal manifestations of HPP within the previous 12 months, or ongoing sessions according to specialist recommendations;
  • use of non-pharmacological and/or medical measures to reduce symptoms and support function, including orthoses, insoles, assistive devices, local injections, or other symptomatic interventions; viii. Other clinical manifestations associated with HPP, in the investigator's opinion, present at screening or documented within the previous 12 months (based on clinical assessment and/or medical records).

Who cannot join

  • 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;
  • Current participation in any clinical study (patients participating in other non interventional studies may be included);
  • Homozygous or compound heterozygous mutation in the ALPL gene;
  • Monoallelic pathogenic, likely pathogenic, or variant of uncertain significance in the ALPL gene in the absence of clinically relevant manifestations of HPP at the time of screening and/or in medical history consistent with the inclusion criterion #5;
  • In the opinion of the investigator, the patient is not able to return for follow-up visits or obtain required follow-up studies;
  • Pregnant and breastfeeding women.

Trial locations and contacts

4 sites in 1 country. Contact the study team or a site near you to ask about taking part.

AstraZeneca Clinical Study Information Center contact

1-877-240-9479

information.center@astrazeneca.com

Russia4 sites, 3 recruiting

  • Research site

    Moscow

    Completed

  • Research Site

    Moscow

    Recruiting

  • Research Site

    Rostov-on-Don

    Recruiting

  • Research site

    Saint Petersburg

    Recruiting

Other trials in Russia

About this study

Official title: The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia (HPP) in Children and Adults in Russia

The effect of monoallelic variants in the ALPL gene on the natural course of hypophosphatasia (HPP) in children and adults in Russia (ATLANTIS)

Read the detailed description

Non-interventional, multi-center, cohort study for evaluation of clinical and patient reported outcomes in routine care settings

Medical subject headings
Hypophosphatasia

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. (1) Mean age (in full years) at the HPP diagnosis;

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  2. (2) Mean age at the onset of initial HPP symptoms (including separately any, skeletal, and non-skeletal symptoms);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  3. (3) Proportions of male and female patients

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  4. (4) Proportions of adults and children at baseline (childhood- and adult-onset HPP);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  5. (5) Proportion of patients with a family history of HPP in a first-degree relative

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  6. (6) Proportions of patients with specific skeletal and non-skeletal manifestation locations/sites affected at baseline:

    1. Dental;
    2. Skeletal;
    3. Muscular;
    4. Rheumatologic;
    5. Renal;
    6. Respiratory;
    7. Neurologic;
    8. Growth / Development;
    9. Other;

    Measured: Day 0 (Visit 1)

  7. (7) Proportions of patients with history and/or presence of specific skeletal manifestations at baseline:

    1. Rickets, including all cases, and radiologically confirmed cases;
    2. Skeletal deformities, including all deformities and specific deformities - genu varum, genu valgum;
    3. Osteomalacia;
    4. Osteopenia;
    5. Osteoporosis (for patients aged ≥18 years);
    6. Fractures, including separately: all fractures, recurrent metatarsal fractures, all pseudofractures (Looser's zones), atypical femoral fractures, vertebral fractures, wrist fractures, rib fractures, stress fractures of the tibia, and poorly healing fractures (non union); fractures by site: feet, femur (hip), wrist, vertebrae, other bones;
    7. Craniosynostosis;
    8. Bone pain;
    9. Other;

    Measured: Day 0 (Visit 1)

  8. (8) Proportions of patients with history and/or presence of specific dental manifestations at baseline:

    1. Early nontraumatic loss of primary (deciduous) teeth;
    2. Early atraumatic loss of secondary (adult) teeth;
    3. Periodontal disease;
    4. Other dental abnormalities (separately - abnormal tooth shape and color, enamel thinning/hypoplasia, loss of alveolar bone, enlarged pulp chambers, etc.);

    Measured: Day 0 (Visit 1)

  9. (9) Proportions of patients with history and/or presence of specific muscular manifestations at baseline:

    1. Muscle weakness;
    2. Enthesopathy;
    3. Low muscle tone;

    Measured: Day 0 (Visit 1)

  10. (10) Proportions of patients with history and/or presence of specific rheumatologic manifestations at baseline:

    1. Calcium pyrophosphate deposition disease, including separately: all cases, pseudogout, and chondrocalcinosis;
    2. Joint pain;
    3. Calcific arthritis and osteoarthritis;
    4. Joint stiffness.
    5. Painful periarticular calcifications of tendons (total and separately - shoulders, elbows, wrists, hips and Achilles' tendons);

    Measured: Day 0 (Visit 1)

  11. (11) Proportions of patients with history and/or presence of specific renal manifestations at baseline:

    1. Nephrocalcinosis;
    2. Nephrolithiasis (Kidney stones);
    3. Renal impairment / failure (chronic kidney disease);
    4. Other.

    Measured: Day 0 (Visit 1)

  12. (12) Proportions of patients with history and/or presence of specific respiratory manifestations at baseline:

    1. Frequent respiratory infections;
    2. Chest deformity (e.g., pectus excavatum, kyphosis, scoliosis, flail chest);
    3. Other.

    Measured: Day 0 (Visit 1)

  13. (13) Proportions of patients with history and/or presence of specific neurologic / psychiatric manifestations at baseline:

    1. Vitamin B6-responsive seizures;
    2. Intracranial hypertension;
    3. Delayed motor milestones;
    4. Cognitive developmental delays;
    5. Waddling gait / difficulty walking;
    6. Depression;
    7. Anxiety;
    8. Attention Deficit/Hyperactivity Disorder;
    9. Insomnia / Poor Sleep Quality;
    10. Other.

    Measured: Day 0 (Visit 1)

  14. (14) Proportions of patients with history and/or presence of other specific manifestations at baseline:

    1. Fever;
    2. Emesis;
    3. Anorexia;
    4. Polyuria;
    5. Dehydration;
    6. Constipation;
    7. Chronic fatigue;
    8. Other.

    Measured: Day 0 (Visit 1)

  15. (15) Median Standard Deviation Score (SDS) for height at baseline (for patients aged <18 years at the respective timepoints only);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  16. (16) Proportion of patients with short stature at baseline (SDS for height <-2.0);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  17. (17) Median SDS for body weight at baseline (for patients aged <10 years at the respective timepoints only);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  18. (18) Proportion of patients with low body weight at baseline (SDS for body weight < 2.0);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  19. (19) Median SDS for body mass index at baseline (for patients aged <18 years at the respective timepoints only);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  20. (20) Proportion of patients with low body weight at baseline (SDS for body mass index < 2.0);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  21. (21) Proportions of patients with other non-specific comorbidities at baseline;

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  22. (22) Proportions of patients with a history and/or presence of specific radiologic signs (X-ray of both hands, posteroanterior view on a single film including the distal forearms) at baseline:

    1. Metaphyseal rarefaction of bone tissue involving the proximal and middle phalanges;
    2. Epimetaphyseal osteopenia (rarefaction of bone structure) of the forearm bones;
    3. Widened, laminated/striated appearance of the physes of the phalanges and metacarpals, and of the distal epimetaphyseal regions of all visualized bones;
    4. Transverse metaphyseal lines consistent with pathological remodeling in the distal radius and ulna.

    Measured: Day 0 (Visit 1)

  23. (23) Proportions of patients with history and/or presence of specific radiologic signs (radiography of both lower limbs, posteroanterior view on a single full-length film, including the distal lower legs) at baseline:

    1. Epimetaphyseal osteopenia (rarefaction of bone structure) of the femur, tibia, and fibula;
    2. Transverse striations;
    3. Altered metaphyseal shape: widening;
    4. Epiphyseal hypoplasia;
    5. Flame-shaped metaphyseal changes extending from the epiphyses toward the metaphyses, consistent with hypophosphatasia-related demineralization;
    6. Widening and deformity of the growth plates with a laminated/striated appearance;
    7. Bowing of the long bones of the lower extremities.

    Measured: Day 0 (Visit 1)

  24. (24) Proportions of patients with history and/or presence of specific radiologic signs on standing spine radiographs in anteroposterior and lateral views (preferably full-length) at baseline

    1. Altered spinal alignment (any, scoliosis, kyphosis);
    2. Altered vertebral shape (any, flattening (platyspondyly), concavity of the upper/lower endplates, wedging);
    3. Altered vertebral structure-osteopenia (of a single vertebra or systemic).

    Children unable to maintain an upright position during radiography may undergo the examination in the supine position in anteroposterior and lateral views).

    Measured: Day 0 (Visit 1)

  25. (25) Proportions of patients with history and/or presence of specific radiologic signs on skull radiographs in anteroposterior and lateral views at baseline:

    1. Altered contour;
    2. Craniosynostosis;
    3. Altered shape;
    4. Presence of pathological fractures.

    Measured: Day 0 (Visit 1)

  26. (26) Proportions of patients with history and/or presence of specific radiologic signs on foot radiographs in anteroposterior and lateral views (in the presence of clinical signs of a pathological "march" fracture) at baseline:

    1. Flattening of the transverse arch;
    2. Flattening of the longitudinal arch;
    3. Altered bone structure of the feet, including meta-epiphyseal regions;
    4. Presence of a pathological (march) fracture.

    Measured: Day 0 (Visit 1)

  27. (27) Median Rickets Severity Scale (RSS) at baseline (only for children ≤14 years at the respective timepoints);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  28. (28) Proportions of patients with history and/or presence of specific laboratory findings at baseline:

    1. Decreased ALP compared to age- and sex-specific reference values;
    2. Elevated PLP in serum (plasma);
    3. Increased PEA in urine;
    4. Decreased GFR (< 60 ml/min/1.73m2);
    5. Hypocalcemia;
    6. Hypercalciuria;
    7. Decreased PTH;
    8. Increased PTH;
    9. Decreased 25-hydroxyvitamin D;

    Measured: Day 0 (Visit 1)

  29. (29) a Mean and median values of specific laboratory findings at baseline:

    1. ALP (U/L)
    2. Serum/plasma PLP (ng/ml);
    3. Urine PEA (m mol / mol creatinine);
    4. GFR (ml/min/1.73m2);
    5. Serum calcium (mmol/L);
    6. Urine calcium (mmol/24h);
    7. Serum PTH (pmol/L);
    8. Serum 25-hydroxyvitamin D (ng/ml);

    Measured: Day 0 (Visit 1)

  30. (30) Proportions of patients with specific ALPL gene mutations (for each gene variant);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  31. (31) Mean and median number of fractures at baseline;

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  32. (32) Mean and median number of dental losses at baseline;

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  33. (33) Mean and median 6-Minute Walk Test (6MWT) distance (m) at baseline (only for patients ≥5 years);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  34. (34) Proportion of patients with decreased 6MWT distance (m) at baseline (<80% of the predicted norm, only for patients ≥5 years);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  35. (35) Mean and median Chair-Rise Test time (s) at baseline (only for patients ≥18 years);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  36. (36) Mean and median Timed Up and Go (TUG) Test time (s) at baseline (only for adults ≥18 years);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  37. (37) a Mean and median values of specific bioimpedance parameters at baseline

    1. Total Skeletal Muscle Mass (SMM) (kg);
    2. Lean Body Mass (LBM) (kg);
    3. Phase angle (degrees);

    Measured: Day 0 (Visit 1)

  38. (38) Mean and median Rating of Perceived Exertion (RPE) score, based on Borg Scale, during the following specific tests at baseline (when the respective tests have been performed):

    1. 6MWT;
    2. Chair-Rise Test;
    3. TUG Test;

    Measured: Day 0 (Visit 1)

  39. (39) Mean and median weekly physical activity duration, measured using the International Physical Activity Questionnaire (IPAQ) Diary, at baseline

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  40. (40) Mean and median Pediatric Quality of Life Inventory (PedsQL) score at baseline (only for children <18 years);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  41. (41) Proportion of patients with different levels of European Quality of Life 5 Dimensions 3-Level (EQ-5D-3L) questionnaire for each of the following dimensions at baseline

    (1 / 2 / 3): mobility, self-care, usual activities, pain/discomfort, and anxiety/depression (only for adults aged ≥ 18 years);

    Measured: Day 0 (Visit 1)

  42. (42) Mean and median EQ-5D-3L Visual Analogue Scale (VAS) score at baseline (only for adults aged ≥ 18 years);

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  43. (43) Proportion of patients with different Modified Ashworth Scale (MAS) scores (0, 1, 1+, 2, 3, or 4) for each specific muscle / muscle group at baseline:

    1. Left biceps;
    2. Right biceps;
    3. Paravertebral muscles;
    4. Left femoral quadriceps;
    5. Right femoral quadriceps;
    6. Left calf muscles;
    7. Right calf muscles;

    Measured: Day 0 (Visit 1)

  44. (44) Proportion of patients with clinically significant spasticity (MAS score ≥2 in at least one muscle / muscle group) at baseline;

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  45. (45) Mean and median dynamic component (D) measure based on the modified Tardieu Scale (MTS) on the for each specific muscle / muscle group at baseline (degrees):

    1. Left biceps;
    2. Right biceps;
    3. Paravertebral muscles;
    4. Left femoral quadriceps;
    5. Right femoral quadriceps;
    6. Left calf muscles;
    7. Right calf muscles;

    Measured: Day 0 (Visit 1)

  46. (46) Proportion of patients with different MTS Resistance scores (0, 1, 2, 3, 4, or 5) for each specific muscle / muscle group at baseline:

    1. Left biceps;
    2. Right biceps;
    3. Paravertebral muscles;
    4. Left femoral quadriceps;
    5. Right femoral quadriceps;
    6. Left calf muscles;
    7. Right calf muscles;

    Measured: Day 0 (Visit 1)

  47. (47) Proportion of patients with clonus (MTS Resistance score ≥3 in at least one muscle / muscle group) at baseline;

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  48. (48) Proportion of patients requiring physiotherapy and/or rehabilitation at baseline;

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  49. (49) Proportion of patients with previous hospitalizations due to HPP manifestations at baseline;

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  50. (50) Median number of previous hospitalizations due to HPP manifestations at baseline

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  51. (51) Annualized rate of hospitalizations due to HPP manifestations prior to baseline

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  52. (52) Mean and median duration of previous hospitalizations due to HPP manifestations at baseline

    In order to achieve the primary objectives, the following variables will be estimated

    Measured: Day 0 (Visit 1)

  53. (53) Proportions of patients with different degrees of disability according to national criteria at baseline

    1. Group I;
    2. Group II;
    3. Group III;
    4. No disability;

    Measured: Day 0 (Visit 1)

  54. (54) Proportions of patients who had specific previous medical interventions & surgeries at baseline:

    1. Respiratory support;
    2. Craniotomy;
    3. Other neurosurgical operations;
    4. Dental prostheses;
    5. Fracture fixation/stabilization;
    6. Joint replacement surgeries;
    7. Other orthopedic surgeries;
    8. Orthotics;
    9. Dental implants;
    10. Physical therapy;

    Measured: Day 0 (Visit 1)

  55. (55) Proportions of patients who previously received specific treatments at baseline:

    1. Pyridoxine;
    2. Antiepileptic medications;
    3. Loop diuretics;
    4. Vitamin D;
    5. Analgesics, including separately: Non-steroidal anti-inflammatory drugs (NSAIDs), opioid analgesics, and all analgesics;
    6. Anti-reflux medications;
    7. Teriparatide;
    8. Anti-sclerostin monoclonal antibodies;
    9. Antiresoprtive medications;
    10. Corticosteroids;
    11. Phosphate binders.

    Measured: Day 0 (Visit 1)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Feb 2026
  2. Study start29 Dec 2025
  3. Primary completion30 Jun 2030 (estimated)
  4. Study completion30 Jun 2030 (estimated)

Resources

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
Cohort
Time perspective
Other
Enrolment
55 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07390240
Sponsor's protocol ID
D8400R00003
Acronym
ATLANTIS
Status last verified
Sep 2026
First submitted
15 Dec 2025
Last updated
28 Sep 2026
Results posted
No
Individual data sharing
Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

Questions about this trial

What is this clinical trial studying?

An observational study of Hypophosphatasia, run by AstraZeneca at 4 sites in Russia.

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 11 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 28 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Russia. 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?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact AstraZeneca Clinical Study Information Center (1-877-240-9479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

(1) Mean age (in full years) at the HPP diagnosis; (time frame: Day 0 (Visit 1)). The study lists 55 primary outcomes in all. 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 NCT07390240.

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 Sep 2026.

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