Comparison of Weekly Somatrogon to Daily Genotropin in Children Born Small for Gestational Age or With Idiopathic Short Stature. (MISSION)

April 29, 2026 updated by: Rabin Medical Center

Multicenter Interventional Study: Somatrogon Impact on Outcomes in Naive Small for Gestational Age or Idiopathic Short Stature Pediatric Patients Compared With Daily Growth Hormone

This study is a randomized, open-label, active controlled, parallel group study comparing the efficacy and safety of once weekly Somatrogon to daily Growth Hormone (Genotropin) in pre-pubertal children with short stature either born Small for Gestational Age (SGA) or with Idiopathic Short Stature (ISS). The planned study duration is 12 months with a screening period of up to 30 days. The study will consist of two groups: 140 children with SGA who are naïve to GH treatment will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months. A second group will include 114 children with ISS who are naïve to GH treatment who will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

254

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • France
      • Angers, France
        • Not yet recruiting
        • Centre Hospitalier Universitaire D'Angers
        • Contact:
      • Le Kremlin-Bicêtre, France
      • Lyon, France
      • Marseille, France
      • Paris, France
        • Not yet recruiting
        • Hopital Necker - Enfants Malades
        • Contact:
      • Paris, France
  • India
      • Maheshra, India
      • Navi Mumbai, India
        • Not yet recruiting
        • Apollo Hospitals Enterprise Limited
        • Contact:
      • New Delhi, India
      • New Delhi, India
        • Not yet recruiting
        • All India Institute of Medical Sciences (AIIMS) - New Delhi
        • Contact:
  • Israel
      • Bear Sheva, Israel
        • Not yet recruiting
        • Soroka Hospital
        • Contact:
      • Ramat Gan, Israel
      • Rishon LeZiyyon, Israel
        • Not yet recruiting
        • Assaf Harofe Medical Center
        • Contact:
      • Tel Aviv, Israel
        • Not yet recruiting
        • Dana-Duek children's hospital
        • Contact:
    • Israe
      • Jerusalem, Israe, Israel, 9103102
        • Not yet recruiting
        • Shaare Zedek Medical Center
        • Contact:
    • Israel
      • Petah Tikva, Israel, Israel, 4920235
        • Recruiting
        • Schneider Children Medical Center- the institute of Endocrinology and Diabetes
        • Contact:
  • Japan
      • Izumi-Shi, Japan
        • Not yet recruiting
        • Osaka Women's and Children's Hospital
        • Contact:
      • Nara, Japan
        • Not yet recruiting
        • Nara Prefecture General Medical Center
        • Contact:
      • Osaka, Japan
      • Setagaya-Ku, Japan
        • Not yet recruiting
        • National Center for Child Health and Development
        • Contact:
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Not yet recruiting
        • University of California Los Angeles (UCLA) - Mattel Children's Hospital
        • Contact:
    • Colorado
      • Centennial, Colorado, United States, 3433325
        • Not yet recruiting
        • Rocky Mountain Pediatric Endocrinology
        • Contact:
    • Florida
      • Jacksonville, Florida, United States, 19803
        • Not yet recruiting
        • Nemours Children's Clinic - Jacksonville
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Not yet recruiting
        • Children's Minnesota - Minneapolis
        • Contact:
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
      • Morristown, New Jersey, United States, 07901
      • New Brunswick, New Jersey, United States, 08901
        • Not yet recruiting
        • Rutgers Robert Wood Johnson Medical School - The Child Heath Institute of New Jersey
        • Contact:
    • New York
      • Buffalo, New York, United States, 13210
        • Not yet recruiting
        • The State University of New York (SUNY) School of Medicine and Biomedical Sciences
        • Contact:
      • New York, New York, United States, 10016
        • Not yet recruiting
        • Hassenfeld Children's Hospital at NYU Langone
        • Contact:
          • Emily Briedbart
    • Texas
      • Austin, Texas, United States, 78723
      • Fort Worth, Texas, United States, 76104
    • Utah
      • Salt Lake City, Utah, United States, 84112

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Diagnosis of SGA or ISS. SGA, defined as born with a birth weight and/or length <-2 SDS below the mean for gestational age. ISS, defined as height < -2 SDS for age and gender without evidence of GHD
  2. Females aged ≥3 years and <9 years. Males aged ≥3 years and <11 years
  3. Pre-pubertal- Tanner stage 1 for breasts and testes.
  4. A bone age of not more than chronological age recorded in previous 8 weeks.
  5. Current height < -2 SDS for age and gender.
  6. Participants using hormonal replacement therapy(s) must be on an optimized and stable treatment regimen (hormone levels within normal ranges on screening) for at least three months prior to screening
  7. Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.

Exclusion Criteria:

  1. History of cancer, radiation therapy or chemotherapy.
  2. History of GHD.
  3. Children who are small due to malnutrition, defined as a Z score of weight for height and/or BMI below -2 for age, according to national standards.
  4. History of HIV-positive, acquired immune deficiency syndrome (AIDS), hepatitis B, hepatitis C, or tuberculosis.
  5. Microcephaly (Head Circumference < -2 SDS)
  6. Any chronic disease or diagnosis, likely to affect growth, including but not limited to gastrointestinal disorder, celiac disease, untreated thyroid disease, diabetes mellitus and metabolic disorders.
  7. Known or suspected skeletal dysplasias
  8. Known or suspected chromosomal abnormalities
  9. IGF-1 >2 SDS
  10. Any disorder or condition which, in the opinion of the investigator, might jeopardize participant's safety or compliance with the protocol
  11. Prior exposure to growth promoting therapy
  12. Current use of any prohibited concomitant medication(s): Any rhGH or growth-promoting therapy, Any therapy that affects appetite or weight, Psychiatric medications associated with weight changes and/or diabetes, excluding medications used to treat ADHD, Any androgen or estrogen therapy including over the counter supplements, Systemic corticosteroids (inhaled or oral) exceeding the doses: Inhaled: > 400 μg/day of inhaled budesonide or equivalent. Oral: > 8 mg/m2/day of oral hydrocortisone or equivalent.
  13. Previous administration with an investigational drug within 90 days.
  14. Fasting blood glucose >126 mg/dL
  15. Renal impairment
  16. Hepatic dysfunction.
  17. Pregnancy
  18. Known hypersensitivity to the components of the study intervention

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Somatrogon
Somatrogon will be administered subcutaneously (s.c) once weekly using a multi dose disposable prefilled pen for single patient use intended for SC self injection.
Drug: Somatrogon
Once weekly Growth Hormone
Other Names:
  • NGENLA 60mg SSOL 1x1.2mL PFP US
Active Comparator: Genotropin
Genotropin will be administrated subcutaneously (s.c) daily using Genotropin Pen growth hormone delivery devices or Genotropin two chamber cartridges (will be supplied in their primary commercial packaging)
Drug: Genotropin
Daily Growth Hormone
Other Names:
  • Genotropin PEN 12 1x U2 US

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annual Height Velocity
Time Frame: • Annualized HV after 12 months of treatment
Annual Height Velocity in cm. Annual Height Velocity at 12 months is based on the difference between the heights at 12 months and baseline.
• Annualized HV after 12 months of treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS)
Time Frame: Baseline and at 12 months
Via central lab analysis
Baseline and at 12 months
height SDS
Time Frame: 3, 6, 9 and 12 months
Changes in height Standard Deviation Score (SDS)
3, 6, 9 and 12 months
Bone maturation
Time Frame: at screening and after 12 months
Annual change in bone age measurements as per Gruelich-Pyle method
at screening and after 12 months
Insulin-like Growth Factor-1 (IGF-1)
Time Frame: Screening and after 3, 6, 9 and 12 months
Via central lab analysis
Screening and after 3, 6, 9 and 12 months
change in health-related quality of life
Time Frame: baseline and after 12 months
Assessed by QoLISSY questionnaire
baseline and after 12 months
Height Velocity over time
Time Frame: HV over 3, 6 and 9 months
HV over 3, 6 and 9 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
IGFBP-3
Time Frame: At baseline and after 12 months
Via central lab
At baseline and after 12 months
Frequency of abnormal lab results in the Somatrogon arm
Time Frame: At screening, baseline and after 1, 3, 6 ,9 and 12 months
At screening, baseline and after 1, 3, 6 ,9 and 12 months
SAE's in the Somatrogon arm
Time Frame: At screening, baseline and after 1, 3, 6 ,9 and 12 months
At screening, baseline and after 1, 3, 6 ,9 and 12 months
AE's in the Somatrogon arm
Time Frame: At screening, baseline and after 1, 3, 6 ,9 and 12 months
At screening, baseline and after 1, 3, 6 ,9 and 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Rabin Medical Center

Collaborators

Pfizer

Investigators

  • Study Director: Moshe Phillip, Professor, Schneider Children's Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2026

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

January 1, 2028

Study Registration Dates

First Submitted

November 6, 2025

First Submitted That Met QC Criteria

November 6, 2025

First Posted (Actual)

November 10, 2025

Study Record Updates

Last Update Posted (Actual)

April 30, 2026

Last Update Submitted That Met QC Criteria

April 29, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 0081-24-RMC

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

We would only share patient data with regulatory agencies that require the information.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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