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Enzyme Replacement Therapy

Participants with Pediatric-onset HPP for Hypophosphatasia

N/A
Waitlist Available
Research Sponsored by Alexion Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSource™.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2016 Phase 2 & 3 trial • 69 Patients • NCT01176266
68%
Pyrexia
59%
Tooth loss
48%
Injection site erythema
43%
Vomiting
29%
Diarrhoea
26%
Nasopharyngitis
25%
Upper respiratory tract infection
25%
Cough
23%
Gastroenteritis
23%
Respiratory tract infection
23%
Constipation
19%
Ear infection
19%
Craniosynostosis
17%
Injection site discolouration
16%
Rash
16%
Injection site induration
16%
Pain in extremity
14%
Rhinitis
14%
Contusion
14%
Headache
14%
Dermatitis diaper
14%
Injection site haematoma
13%
Bronchitis
13%
Gastrooesophageal reflux disease
13%
Anaemia
12%
Urinary tract infection
12%
Injection site pain
12%
Injection site reaction
12%
Dyspnoea
12%
Conjunctivitis
12%
Dry skin
12%
Pneumonia
10%
Influenza
10%
Injection site swelling
10%
Abdominal pain
10%
Dental caries
10%
Fall
10%
Use of accessory respiratory muscles
10%
Eczema
9%
Injection site pruritus
9%
Atelectasis
9%
Hypocalcaemia
9%
Tachypnoea
9%
Alanine aminotransferase increased
9%
Respiratory distress
9%
Erythema
9%
Tachycardia
9%
Hypoglycaemia
9%
Hypokalaemia
9%
Kyphosis
7%
Device related infection
7%
Hand-foot-and-mouth disease
7%
Otitis media
7%
Tonsillitis
7%
Catheter site erythema
7%
Toothache
7%
Arthropod bite
7%
Procedural pain
7%
Rib fracture
7%
Oxygen saturation decreased
7%
Papilloedema
7%
Congenital bowing of long bones
7%
Cerumen impaction
7%
Wheezing
7%
Lymphadenopathy
7%
Excessive granulation tissue
7%
Nephrocalcinosis
7%
Injection site papule
7%
Injection site rash
7%
Teething
7%
Aspartate aminotransferase increased
7%
Knee deformity
6%
Food intolerance
6%
Gastroenteritis viral
6%
Gastrointestinal infection
6%
Viral upper respiratory tract infection
6%
Injection site atrophy
6%
Tooth fracture
6%
Vitamin D decreased
6%
Bone disorder
6%
Cerebral ventricle dilatation
6%
Hepatomegaly
6%
Granuloma skin
6%
Heat rash
6%
Bradycardia
6%
Respiratory disorder
6%
Neutropenia
6%
Agitation
6%
Hypertension
6%
Catheter site inflammation
6%
Device occlusion
6%
Irritability
6%
Oropharyngeal pain
6%
Rhinorrhoea
6%
Tracheomalacia
4%
Hydrocephalus
4%
Intracranial pressure increased
4%
Syringomyelia
4%
Cardiac arrest
4%
Apnoea
4%
Respiratory failure
4%
Pneumonia viral
4%
Arnold-Chiari malformation
4%
Femur fracture
3%
Cyanosis
3%
Gastroenteritis rotavirus
3%
Respiratory syncytial virus infection
3%
Rhinovirus infection
3%
Sepsis
3%
Viral infection
3%
Acute respiratory failure
3%
Pneumonia aspiration
3%
Convulsion
3%
Cardio-respiratory arrest
3%
Drug hypersensitivity
3%
Failure to thrive
3%
Feeding disorder
3%
Hyponatraemia
3%
Osteopenia
3%
Feeding tube complication
1%
Meningitis staphylococcal
1%
Adenoviral upper respiratory infection
1%
Bacteraemia
1%
Beta haemolytic streptococcal infection
1%
Bronchiolitis
1%
Clostridium difficile colitis
1%
Corona virus infection
1%
Enterobacter infection
1%
Lower respiratory tract infection
1%
Parainfluenzae virus infection
1%
Postoperative wound infection
1%
Pseudomonas infection
1%
Respiratory tract infection viral
1%
Serratia infection
1%
Adenoidal hypertrophy
1%
Asthma
1%
Bronchospasm
1%
Collapse of lung
1%
Hypoxia
1%
Brain oedema
1%
Encephalitis
1%
Febrile convulsion
1%
Haemorrhagic stroke
1%
Intracranial hypotension
1%
Motor dysfunction
1%
CSF pressure
1%
Enterovirus test positive
1%
Incorrect dose administered
1%
Arrhythmia
1%
Pancreatitis acute
1%
Pneumatosis intestinalis
1%
Tracheostomy
1%
Anaphylactoid reaction
1%
Optic neuropathy
1%
Coagulopathy
1%
Hyperparathyroidism tertiary
1%
Breath holding
1%
Staring
1%
Renal failure
1%
Deep vein thrombosis
1%
Dehydration
1%
Tracheal operation
1%
Wean from ventilator
1%
Staphylococcal infection
1%
Staphylococcal sepsis
1%
Stenotrophomonas infection
1%
Obstructive airways disorder
1%
Pneumonitis
1%
Pulmonary hypertension
1%
Pulmonary oedema
1%
Respiratory arrest
1%
Motor developmental delay
1%
Neurological symptom
1%
Petit mal epilepsy
1%
Aspiration tracheal
1%
Dystrophic calcification
1%
Fluid overload
1%
Hyperkalaemia
1%
Hypernatraemia
1%
Hyperphosphataemia
1%
Hypoalbuminaemia
1%
Hypophagia
1%
Hypervolaemia
1%
Cardiopulmonary failure
1%
Wolff-Parkinson-White syndrome
1%
Inguinal hernia
1%
Rectal prolapse
1%
Arthralgia
1%
Joint contracture
1%
Pathological fracture
1%
Cranial operation
1%
Medical device removal
1%
Serratia sepsis
1%
Upper respiratory tract inflammation
1%
Hypophosphatasia
1%
Hepatic enzyme increased
1%
Human rhinovirus test positive
1%
Neurological examination abnormal
1%
Nuclear magnetic resonance imaging
1%
Respiratory rate increased
1%
Respiratory syncytial virus test positive
1%
Weight decreased
1%
Chills
1%
Device dislocation
1%
Oedema
1%
Abdominal wound dehiscence
1%
Brain herniation
1%
Endotracheal intubation complication
1%
Eye injury
1%
Humerus fracture
100%
80%
60%
40%
20%
0%
Study treatment Arm
Asfotase Alfa

Trial Design

1Treatment groups
Experimental Treatment
Group I: Participants with Pediatric-onset HPPExperimental Treatment1 Intervention
Adult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Asfotase alfa
FDA approved

Find a Location

Who is running the clinical trial?

Alexion Pharmaceuticals, Inc.Lead Sponsor
259 Previous Clinical Trials
141,028 Total Patients Enrolled
25 Trials studying Hypophosphatasia
1,502 Patients Enrolled for Hypophosphatasia
AlexionLead Sponsor
246 Previous Clinical Trials
39,221 Total Patients Enrolled
25 Trials studying Hypophosphatasia
1,502 Patients Enrolled for Hypophosphatasia
Xcenda, LLCUNKNOWN
1 Previous Clinical Trials
500 Total Patients Enrolled
~9 spots leftby Sep 2025
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