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DwarfismPhase 2NCT03255694

A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short Stature

A Phase 2 study testing PEG-somatropin in people with Dwarfism, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 12 sites in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

After the first stage (52 weeks) of Phase II clinical trial, Pegylated recombinant human growth hormone (PEG-rhGH) injection of appropriate dose in compliance with ISS clinical treatment strategy is used to treat children with ISS (Idiopathic Short Stature). Read more about this study

Who is it for?
Ages 4 to 9 years
Conditions the study is for
Dwarfism
What is being tested?
PEG-somatropin
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
360 participants
Where
12 sitesChina
Study dates
12 May 2017to Jun 2030
Phase
Phase 2
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 4 to 9 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • All subjects who have completed the first stage (52 weeks) of Phase II clinical trial (including negative controls) with completed follow-up records may be enrolled in the extension period study.
  • Before the extension period study, the investigator shall fully inform the subjects and their guardians of all the information about the extension period study, including detailed follow-up procedure, treatment plan, laboratory examination items during follow-ups and possible benefits and risks. The extension period study shall only be initiated after the subjects and their guardians are well informed, and agree to cooperate and complete the treatment, follow-ups and examinations during the study, and sign the written informed consent.

Who cannot join

  • Subjects who have taken the following medications within 2 months before entering the extension period study:
  • Aromatase inhibitors (which include but are not limited to Lelrozol and Anastrozole), with continuous medication ≥1 month;
  • Gonadotropin releasing hormone analogues (which include but are not limited to Triptorelin, Leuprorelin and Goserelin),, with continuous medication ≥1 month;
  • Sex steroids (which include but are not limited to any type of estrogen, progestin and androgen) , with continuous medication ≥1 month;
  • Protein anabolic drugs (which include but are not limited to Oxandrolone, Danazol and Strombafort), with continuous medication ≥1 month;
  • Glucocorticoids via oral/intravenous administration for more than 1 month..

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

China12 sites

  • Beijing Children's Hospital, Capital Medical University, National Center for Children's Health

    Beijing, Beijing Municipality

  • The First Hospital of Jilin University

    Changchun, Jilin

  • Hunan Children's Hospital

    Changsha, Hunan

  • The Children's Hospital of Zhejiang University School of Medicine

    Hangzhou, Zhejiang

  • Jiangxi Provincial Children's Hospital

    Nanchang, Jiangxi

  • Children's Hospital of Soochow University

    Naning, Jiangsu

  • The First Affiated Hospital of Nanjing Medical Universit

    Nanjing, Jiangsu

  • Shanghai Children's Hospital of Fudan University

    Shanghai

  • Shanghai Children's Hospital

    Shanghai

  • Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan

    Wuhan, Hubei

  • Affiliated Hospital of Jiangnan University

    Wuxi, Jiangsu

  • Wuxi Children's Hospital

    Wuxi, Jiangsu

Other trials in China

About this study

Official title: A Phase 2 Study Extension Period of Pegylated Somatropin (PEG-somatropin) in the Treatment of Children With Idiopathic Short Stature: An Open, Non-controlled Observational Study.

After the first stage (52 weeks) of Phase II clinical trial, Pegylated recombinant human growth hormone (PEG-rhGH) injection of appropriate dose in compliance with ISS clinical treatment strategy is used to treat children with ISS (Idiopathic Short Stature). The long-term efficacy and safety of the investigational product are evaluated, which can provide more scientific and reliable medication guidance information for clinical diagnosis and treatment.

Conditions
Dwarfism
Being tested
PEG-somatropin
Medical subject headings
Dwarfism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PEG-somatropinDrug

    Also called Polyethylene Glycol Recombinant Human Somatropin Injection

    After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.

Study groups in NCT03255694
Study groupRoleWhat they receive
PEG-somatropinExperimentalAfter the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for the high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.
Receives: Drug: PEG-somatropin

What is being measured

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

  1. Change of yearly height velocity (ΔHV)

    Change of yearly height velocity before and after treatment. Yearly Height Velocity=12×(Height Yx - Height at Baseline)/(Date of Yx - Date of Baseline)(Yx refers to the height value at particular timepoint x)

    Measured: Baseline，the end of 3-year addendum

Secondary outcomes (14)

  1. Standard deviation score of height at the actual age (ΔHT SDS)

    Standard deviation score of height at the actual age.

    Measured: Baseline，every 3 months，the end of 3-year addendum

  2. Change fo Bone maturation

    Change fo Bone maturation before and after treatement (bone age/chronological age)

    Measured: Baseline，every 3 months，the end of 3-year addendum

  3. Change of IGF-1 SDS (ΔIGF-1 SDS)

    Change of IGF-1 SDS before and after treatement

    Measured: Baseline，every 3 months，the end of 3-year addendum

  4. Changes of standard deviation scores of body mass index (ΔBMI SDS)

    Changes of standard deviation scores of body mass index

    Measured: Baseline，every 3 months，the end of 3-year addendum

  5. The yearly average dose of PEG-rhGH injection

    Measured: Baseline，every 3 months，the end of 3-year addendum

  6. Final height (FH)

    Final height

    Measured: Baseline，every 3 months，the end of 3-year addendum

  7. The improvement of FH compared with the baseline predicted adult height (PAH)

    Measured: Baseline，every 3 months，the end of 3-year addendum

  8. Improvement of NAH (near adult height)

    For subjects who reach NAH with treatment but fail to follow-ups before reaching FH, the improvement of NAH in comparison with the baseline PAH (predicted adult height) shall be evaluated

    Measured: Baseline，every 3 months，the end of 3-year addendum

  9. the improvement of PAH

    For subjects who fail to reach NAH with treatment and fail to follow-ups before reaching FH, the improvement of PAH in comparison with the baseline PAH shall be evaluated

    Measured: Baseline，every 3 months，the end of 3-year addendum

  10. The changes of the scores evaluated by the Quality of Life Scale

    Measured: Baseline，every 3 months，the end of 3-year addendum

  11. The changes of lean body mass (LBM) (optional)

    Measured: Baseline，every 3 months，the end of 3-year addendum

  12. The changes of fat mass (torso) (FM) (optional)

    Measured: Baseline，every 3 months，the end of 3-year addendum

  13. The changes of the percentage of body fat (optional)

    Measured: Baseline，every 3 months，the end of 3-year addendum

  14. The changes of bone mineral density (BMD) (optional)

    Measured: Baseline，every 3 months，the end of 3-year addendum

Timeline

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

  1. First posted21 Aug 2017
  2. Study start12 May 2017
  3. Primary completionJun 2030 (estimated)
  4. Study completionJun 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
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
360 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03255694
Sponsor's protocol ID
GenSci 033 CT-Extension Period
Status last verified
Aug 2025
First submitted
14 Jul 2017
Last updated
15 Aug 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PEG-somatropin in people with Dwarfism, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 12 sites in China.

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 2 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: under way, not looking for participants, as of the sponsor's last update on 15 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in China. 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.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change of yearly height velocity (ΔHV) (time frame: Baseline，the end of 3-year addendum). 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 NCT03255694.

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 15 Aug 2025.

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