Turner SyndromePhase 2NCT05690386
A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome
A Phase 2 study testing Lonapegsomatropin and Somatropin in people with Turner Syndrome, run by Ascendis Pharma Endocrinology Division A/S at 19 sites in United States.
Trial summary
A 104 week dose finding open label trial followed by an optional 78 week open label extension of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus daily somatropin product in prepubertal individuals with Turner syndrome. Read more about this study
- Who is it for?
- Ages 1 year to 10 years, women only
- Conditions the study is for
- Turner Syndrome
- What is being tested?
- Lonapegsomatropin and Somatropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 19 sitesUnited States
- Study dates
- 15 Feb 2023to Dec 2027
- 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 1 year to 10 years
- Women only
- No healthy volunteers
- Child
Who may be able to join
- Age between 1 and 10 years, inclusive.
- TS diagnosis via genetic test.
- Prepubertal status.
- Naïve to growth hormone therapy or growth hormone secretagogue.
- Exhibit impaired growth defined by at least one of the following:
- AHV< 6 cm/year or <25ᵗʰ percentile over a time span of 6-18 months for children of 2 years and older.
Show all 12Show fewer
- Height (or length for individuals < 2 years old) <10ᵗʰ percentile for sex and age according to the 2000 CDC Growth Charts for the United States.
- Bone age within normal limits for chronological age, defined as no more than 20% above or below chronological age in months or delayed for chronological age (greater than 20% below chronological age), at screening.
- Biochemically euthyroid (including when on thyroid hormone supplementation).
- If on hormone replacement therapies for any hormone deficiencies other than growth hormone (e.g. adrenal, thyroid), must be on adequate and stable doses for ≥4 weeks prior to and throughout Screening.
- Fundoscopy at Screening without signs/symptoms of intracranial hypertension or proliferative retinopathy or evidence of any other retinal disease for which growth hormone therapy is contraindicated.
- Capable of giving signed informed consent. Participants and/or parents or legal guardians of participants must sign an informed consent statement. Assent should be obtained from all participants competent to understand the protocol, per IRB requirements.
Who cannot join
- Turner Syndrome with presence of Y-chromosomal material on genetic testing and without a history of gonadectomy.
- Diagnosis of diabetes mellitus.
- Known history of clinically relevant conditions that may have an effect on growth, e.g. but not limited to celiac disease, malnutrition, treatment with potential growth-influencing medications for Attention-deficit/ hyperactivity disorder (ADHD), etc.
- Any known, clinically significant, congenital or acquired cardiac/cardiovascular dysfunction that might interfere with growth as determined by transthoracic echocardiogram.
- Known history or presence of malignancy.
- Individuals with history of intracranial tumor or cysts, with evidence of growth within the last 12 months prior to Screening.
Show all 18Show fewer
- Note - Individuals with a history of intracranial tumor may be eligible if there is no evidence of residual tumor as determined by MRI/CT scan(s) performed within 6 to 12 months prior to screening.
- Hepatic transaminases (i.e., AST or ALT) above 3 times the upper limit of normal according to the central laboratory at screening.
- Major medical conditions and/or presence of contraindication to hGH treatment.
- Abnormal renal function.
- Clinically relevant systemic illness, acute critical illness, and complications following open heart surgery, abdominal surgery, multiple accidental traumas, acute respiratory failure, or similar conditions within 6 months prior to Screening.
- Poorly controlled hypertension.
- Receiving prior or concurrent treatment with any agent that might influence growth or interfere with GH secretion or action such as, but not limited to, non steroidal anabolic agents, sex steroids, etc.
- Oral/intravenous/intramuscular corticosteroids within 90 days prior to or throughout Screening.
- Known or suspected hypersensitivity to study intervention(s) or related products.
- Participation in any other trial involving an investigational compound within 90 days prior to Screening or in parallel to this trial.
- Any disease or condition that, in the judgement of the investigator, may make the individual unlikely to comply with the protocol or presents undue risk.
- Female who is pregnant, plans to be pregnant, or is breastfeeding.
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
Ascendis Pharma Investigational Site
Atlanta, Georgia, 30322
Ascendis Pharma Investigational Site
Aurora, Colorado, 80045
Ascendis Pharma Investigational Site
Boston, Massachusetts, 02114
Ascendis Pharma Investigational Site
Chapel Hill, North Carolina, 27599
Ascendis Pharma Investigational Site
Chicago, Illinois, 60611
Ascendis Pharma Investigational Site
Cincinnati, Ohio, 45229
Ascendis Pharma Investigational Site
El Paso, Texas, 79902
Ascendis Pharma Investigational Site
Fort Worth, Texas, 76104
Ascendis Pharma Investigational Site
Idaho Falls, Idaho, 83404
Ascendis Pharma Investigational Site
Lake Success, New York, 11042
Ascendis Pharma Investigational Site
Las Vegas, Nevada, 89113
Ascendis Pharma Investigational Site
Oklahoma City, Oklahoma, 73104
Ascendis Pharma Investigational Site
Orlando, Florida, 32827
Ascendis Pharma Investigational Site
Palo Alto, California, 94304
Ascendis Pharma Investigational Site
Portland, Oregon, 97239
Ascendis Pharma Investigational Site
Saint Paul, Minnesota, 55102
Ascendis Pharma Investigational Site
San Diego, California, 92123
Ascendis Pharma Investigational Site
Seattle, Washington, 98105
Ascendis Pharma Investigational Site
St. Petersburg, Florida, 33701-4804
About this study
Official title: New InsiGHTS: A Multicenter, Phase 2, Randomized, Open-label, Active-controlled, Parallel Group Clinical Trial to Investigate the Safety, Tolerability, and Efficacy of Different Dose Levels of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Individuals With Turner Syndrome
A 104 week dose finding open label trial followed by an optional 78 week open label extension of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus daily somatropin product in prepubertal individuals with Turner syndrome. Approximately 48 individuals (12 individuals per arm) will be randomized to receive one of three doses of lonapegsomatropin or a daily injection of somatropin. This is a trial that will be conducted in the United States.
- Conditions
- Turner Syndrome
- Being tested
- Lonapegsomatropin, Somatropin
- Medical subject headings
- Turner Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LonapegsomatropinBiological
Once-weekly subcutaneous injection of Lonapegsomatropin
SomatropinDrug
Once-daily subcutaneous injection of Somatropin
| Study group | Role | What they receive |
|---|---|---|
| Lonapegsomatropin at 0.24 mg hGH/kg/week | Experimental | Lonapegsomatropin at 0.24 mg hGH/kg/week administered once-weekly by subcutaneous injection Receives: Biological: Lonapegsomatropin |
| Lonapegsomatropin at 0.30 mg hGH/kg/week | Experimental | Lonapegsomatropin at 0.30 mg hGH/kg/week administered once-weekly by subcutaneous injection Receives: Biological: Lonapegsomatropin |
| Lonapegsomatropin at 0.36 mg hGH/kg/week | Experimental | Lonapegsomatropin at 0.36 mg hGH/kg/week administered once-weekly by subcutaneous injection Receives: Biological: Lonapegsomatropin |
| Somatropin at 0.05 mg/kg/day | Active comparator | Somatropin at 0.05 mg/kg/day administered once-daily by subcutaneous injection Receives: Drug: Somatropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Height Velocity (AHV) (cm/year)
Calculated based on the difference between the AHV at 6 months and baseline
Measured: 26 weeks
Secondary outcomes (4)
Annualized Height Velocity (AHV) (cm/year)
Calculated based on the difference between the AHVs at 12, 24, 36 and 42 months and baseline
Measured: 52 weeks, 104 weeks, 156 weeks and 182 weeks
Change from baseline in height standard deviation score (SDS)
Calculated based on the difference between the heights SDS at 6, 12, 24, 36 and 42 months and baseline
Measured: 26 weeks, 52 weeks, 104 weeks, 156 weeks and 182 weeks
Change from baseline in Bone age (calculated years)
Annual change in bone age measurements as per Gruelich-Pyle method
Measured: 52 weeks, 104 weeks and 156 weeks
Change from baseline in ratio of bone age/chronological age
Calculated as a ratio
Measured: 52 weeks, 104 weeks and 156 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2023
- Study start15 Feb 2023
- Primary completion18 Oct 2024
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Turner SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Randomised
- Intervention model
- Parallel groups
- Model description
- An open label, parallel group with subjects randomized into 1 of 4 treatment groups (1:1:1:1)
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05690386
- Sponsor's protocol ID
- ASND0034
- Status last verified
- May 2026
- First submitted
- 10 Jan 2023
- Last updated
- 13 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Davis SM, Abuzzahab MJ, Tryggestad JB, Fechner PY, Gutmark-Little I, Woods K, Fadoju D, Stafford D, Nwosu BU, Ikle JM, Abdelrahman SG, Zhen X, Mao M, Komirenko AS, Shu AD, Klein KO; New InsiGHTS Investigator Group; the. Efficacy and Safety of Lonapegsomatropin in Children With Turner Syndrome: 52-Week Results From the New InsiGHTS Trial. J Clin Endocrinol Metab. 2026 Oct 26:dgag436. doi: 10.1210/clinem/dgag436. Online ahead of print.
PubMed 42929032
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- New InsiGHTS: A Multicenter, Phase 2, Randomized, Open-label, Active-controlled, Parallel Group Clinical Trial to Investigate the Safety, Tolerability, and Efficacy of Different Dose Levels of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Individuals With Turner Syndrome
- ClinicalTrials.gov ID
- NCT05690386
- Acronym / sponsor ID
- ASND0034
- Conditions
- Turner Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/SIndustry (pharma, biotech, devices)
- Study start
- 15 Feb 2023 (actual)
- Primary completion
- 18 Oct 2024 (actual)
- Study completion
- Dec 2027 (estimated)
- First posted
- 19 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A 104 week dose finding open label trial followed by an optional 78 week open label extension of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus daily somatropin product in prepubertal individuals with Turner syndrome. Approximately 48 individuals (12 individuals per arm) will be randomized to receive one of three doses of lonapegsomatropin or a daily injection of somatropin. This is a trial that will be conducted in the United States.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- An open label, parallel group with subjects randomized into 1 of 4 treatment groups (1:1:1:1)
| Arm | Type | Receives |
|---|---|---|
| Lonapegsomatropin at 0.24 mg hGH/kg/week | Experimental | Biological: LonapegsomatropinLonapegsomatropin at 0.24 mg hGH/kg/week administered once-weekly by subcutaneous injection |
| Lonapegsomatropin at 0.30 mg hGH/kg/week | Experimental | Biological: LonapegsomatropinLonapegsomatropin at 0.30 mg hGH/kg/week administered once-weekly by subcutaneous injection |
| Lonapegsomatropin at 0.36 mg hGH/kg/week | Experimental | Biological: LonapegsomatropinLonapegsomatropin at 0.36 mg hGH/kg/week administered once-weekly by subcutaneous injection |
| Somatropin at 0.05 mg/kg/day | Active comparator | Drug: SomatropinSomatropin at 0.05 mg/kg/day administered once-daily by subcutaneous injection |
Who may qualify
- Ages
- 1 year to 10 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- Age between 1 and 10 years, inclusive.
- TS diagnosis via genetic test.
- Prepubertal status.
- Naïve to growth hormone therapy or growth hormone secretagogue.
- Exhibit impaired growth defined by at least one of the following:
Key exclusion criteria (18 listed)
- Turner Syndrome with presence of Y-chromosomal material on genetic testing and without a history of gonadectomy.
- Diagnosis of diabetes mellitus.
- Known history of clinically relevant conditions that may have an effect on growth, e.g. but not limited to celiac disease, malnutrition, treatment with potential growth-influencing medications for Attention-deficit/ hyperactivity disorder (ADHD), etc.
- Any known, clinically significant, congenital or acquired cardiac/cardiovascular dysfunction that might interfere with growth as determined by transthoracic echocardiogram.
- Known history or presence of malignancy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LonapegsomatropinOnce-weekly subcutaneous injection of Lonapegsomatropin | Biological | Lonapegsomatropin at 0.24 mg hGH/kg/week, Lonapegsomatropin at 0.30 mg hGH/kg/week, Lonapegsomatropin at 0.36 mg hGH/kg/week |
| SomatropinOnce-daily subcutaneous injection of Somatropin | Drug | Somatropin at 0.05 mg/kg/day |
Primary outcome
Annualized Height Velocity (AHV) (cm/year)
Time frame: 26 weeks
Calculated based on the difference between the AHV at 6 months and baseline
4 secondary outcomes
Annualized Height Velocity (AHV) (cm/year)
Time frame: 52 weeks, 104 weeks, 156 weeks and 182 weeks
Change from baseline in height standard deviation score (SDS)
Time frame: 26 weeks, 52 weeks, 104 weeks, 156 weeks and 182 weeks
Change from baseline in Bone age (calculated years)
Time frame: 52 weeks, 104 weeks and 156 weeks
Change from baseline in ratio of bone age/chronological age
Time frame: 52 weeks, 104 weeks and 156 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2026.
- Derived from this study: PubMed 42929032Davis SM, Abuzzahab MJ, Tryggestad JB, Fechner PY, Gutmark-Little I, Woods K, Fadoju D, Stafford D, Nwosu BU, Ikle JM, Abdelrahman SG, Zhen X, Mao M, Komirenko AS, Shu AD, Klein KO; New InsiGHTS Investigator Group; the. Efficacy and Safety of Lonapegsomatropin in Children With Turner Syndrome: 52-Week Results From the New InsiGHTS Trial. J Clin Endocrinol Metab. 2026 Oct 26:dgag436. doi: 10.1210/clinem/dgag436. Online ahead of print.
Related trials for Turner Syndrome
RecruitingPhase 3
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Turner Syndrome, Short Stature Homeobox Gene Mutation
RecruitingObservational study
Gonadal Tissue Freezing for Fertility Preservation in Individuals at Risk for Ovarian Dysfunction, Premature Ovarian Insufficiency and Clinically Indicated Gonadectomy
Turner Syndrome, Post-menarcheal Adolescents
Not yet recruitingObservational study
Hematological Profile in Children With Numerical Chromosomal Disorders
Down Syndrome, Turner Syndrome
RecruitingObservational study
Natural History of Noncirrhotic Portal Hypertension
Cystic Fibrosis, Immunologic Deficiency Syndrome
Active, not recruitingPhase 2
Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity
Hypothalamic Obesity, Hypothalamic Tumor
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lonapegsomatropin and Somatropin in people with Turner Syndrome, run by Ascendis Pharma Endocrinology Division A/S at 19 sites in United States.
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 12 inclusion and 18 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 13 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States. 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?
Annualized Height Velocity (AHV) (cm/year) (time frame: 26 weeks). 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 NCT05690386, which also lists 1 linked publication.
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 13 May 2026.