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NCT Number: NCT05775523

A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin

The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.

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Key information

Conditions

Growth Hormone Deficiency Bone Diseases Bone Diseases, Developmental Bone Diseases, Endocrine Brain Diseases Central Nervous System Diseases Dwarfism Dwarfism, Pituitary Endocrine System Diseases Failure to Thrive Hypopituitarism Hypothalamic Diseases Musculoskeletal Diseases Nervous System Diseases Pathological Conditions, Signs and Symptoms Pituitary Diseases Signs and Symptoms

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Ascendis Investigational Site, Phoenix, Arizona, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Paediatric patients with GHD who are on treatment with lonapegsomatropin
  • Patients being clinically managed in Europe or the USA
  • Appropriate written informed consent/assent as applicable for the age of the patient
  • Patients willing to comply with follow-up requirements of the study

Exclusion criteria

  • Patients participating in any interventional clinical trial for short stature
  • Patients being treated with a GH or IGF-1 therapy, other than lonapegsomatropin, at enrollment
  • Patients for whom treatment with lonapegsomatropin is contraindicated
  • Patients with closed epiphyses
  • Patients with active malignant tumours
  • Patients under antitumour therapy within the past 12 months prior to instituting GH therapy
  • Hypersensitivity to somatropin or any of the excipients in lonapegsomatropin

Treatment and study plan

No intervention

Drug

No intervention

Primary outcomes

  1. Occurrence of neoplasms (benign, malignant and unspecified)

    Time frame: 5 years

  2. Occurrence of type 2 diabetes mellitus

    Time frame: 5 years

Secondary outcomes

  1. Occurrence of renal, hepatic, immunologic and neurologic adverse events

    Time frame: 5 years

  2. Occurrence of medication errors in patients treated with lonapegsomatropin

    Time frame: 5 years

    Frequency and type of medication errors as reported by treating physician

  3. Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy

    Time frame: 5 years

    IGF-1 level (ng/mL)

  4. Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy

    Time frame: 5 years

    IGF-1 Standard Deviation Score (SDS)

  5. Compare the occurrence of neoplasms (benign, malignant and unspecified) in patients treated with lonapegsomatropin with historical data from literature

    Time frame: 5 years

  6. Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin with historical data from literature

    Time frame: 5 years

Study contacts

Contact information is provided by the study sponsor or research team.

Deborah Bowlby

CONTACT

[email protected]

+1 844-442-7236

Sponsors and collaborators

Lead sponsor

Ascendis Pharma Endocrinology Division A/S

Industry

Registry information

Official study title

A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin

Acronym: SkyPASS

Important dates

Study start
2023
Primary completion
2033
Study completion
2033
First posted
Mar 20, 2023
Registry last updated
Feb 4, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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