NCT05690386|Active Not RecruitingPhase 2FDA Drug

A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome

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

Ascendis Pharma Endocrinology Division A/S ClinicalTrials.gov

Compare

1 other identifier

ASND0034

Study Type

interventional

Target

Locations

1 country

Sites

Timeline

RegisteredJan 2023

StartedFeb 2023

CompletionDec 2027

Brief 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. 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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P25-P50 for phase_2

Timeline

19mo left

Started Feb 2023

Longer than P75 for phase_2

Geographic Reach

1 country

19 active sites

Status

active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

4.8 years study duration

Study Progress67%

Feb 2023Dec 2027

First Submitted

Initial submission to the registry

January 10, 2023

Completed

9 days until next milestone

First Posted

Study publicly available on registry

January 19, 2023

Completed

27 days until next milestone

Study Start

First participant enrolled

February 15, 2023

Completed

1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 18, 2024

Completed

3.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2027

Expected

Last Updated

January 12, 2026

Status Verified

December 1, 2025

Enrollment Period

1.7 years

First QC Date

January 10, 2023

Last Update Submit

December 19, 2025

Conditions

Turner Syndrome

Outcome Measures

Primary Outcomes (1)

Annualized Height Velocity (AHV) (cm/year)
Calculated based on the difference between the AHV at 6 months and baseline
26 weeks

Secondary Outcomes (4)

Annualized Height Velocity (AHV) (cm/year)
52 weeks, 104 weeks, 156 weeks and 182 weeks
Change from baseline in height standard deviation score (SDS)
26 weeks, 52 weeks, 104 weeks, 156 weeks and 182 weeks
Change from baseline in Bone age (calculated years)
52 weeks, 104 weeks and 156 weeks
Change from baseline in ratio of bone age/chronological age
52 weeks, 104 weeks and 156 weeks

Other Outcomes (1)

Insulin-like growth factor 1 (IGF-1) standard deviation score (SDS)
26 weeks, 52 weeks, 104 weeks, 156 weeks and 182 weeks

Study Arms (4)

Lonapegsomatropin at 0.24 mg hGH/kg/week

EXPERIMENTAL

Lonapegsomatropin at 0.24 mg hGH/kg/week administered once-weekly by subcutaneous injection

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

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

Biological: Lonapegsomatropin

Somatropin at 0.05 mg/kg/day

ACTIVE COMPARATOR

Somatropin at 0.05 mg/kg/day administered once-daily by subcutaneous injection

Drug: Somatropin

Interventions

LonapegsomatropinBIOLOGICAL

Once-weekly subcutaneous injection of Lonapegsomatropin

Lonapegsomatropin at 0.24 mg hGH/kg/weekLonapegsomatropin at 0.30 mg hGH/kg/weekLonapegsomatropin at 0.36 mg hGH/kg/week

SomatropinDRUG

Once-daily subcutaneous injection of Somatropin

Somatropin at 0.05 mg/kg/day

Eligibility Criteria

Age1 Year - 10 Years

Sexfemale(Gender-based eligibility)

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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.
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.

You may not qualify if:

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.
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.
+3 more criteria

Contact the study team to confirm eligibility.