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

NCT ID: NCT05690386

Last Updated: 2026-01-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

48 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-15

Study Completion Date

2027-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Turner Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

An open label, parallel group with subjects randomized into 1 of 4 treatment groups (1:1:1:1)
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lonapegsomatropin at 0.24 mg hGH/kg/week

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

Group Type EXPERIMENTAL

Lonapegsomatropin

Intervention Type BIOLOGICAL

Once-weekly subcutaneous injection of Lonapegsomatropin

Lonapegsomatropin at 0.30 mg hGH/kg/week

Lonapegsomatropin at 0.30 mg hGH/kg/week administered once-weekly by subcutaneous injection

Group Type EXPERIMENTAL

Lonapegsomatropin

Intervention Type BIOLOGICAL

Once-weekly subcutaneous injection of Lonapegsomatropin

Lonapegsomatropin at 0.36 mg hGH/kg/week

Lonapegsomatropin at 0.36 mg hGH/kg/week administered once-weekly by subcutaneous injection

Group Type EXPERIMENTAL

Lonapegsomatropin

Intervention Type BIOLOGICAL

Once-weekly subcutaneous injection of Lonapegsomatropin

Somatropin at 0.05 mg/kg/day

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

Group Type ACTIVE_COMPARATOR

Somatropin

Intervention Type DRUG

Once-daily subcutaneous injection of Somatropin

Interventions

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Lonapegsomatropin

Once-weekly subcutaneous injection of Lonapegsomatropin

Intervention Type BIOLOGICAL

Somatropin

Once-daily subcutaneous injection of Somatropin

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Age between 1 and 10 years, inclusive.
2. TS diagnosis via genetic test.
3. Prepubertal status.
4. Naïve to growth hormone therapy or growth hormone secretagogue.
5. Exhibit impaired growth defined by at least one of the following:

1. AHV\< 6 cm/year or \<25ᵗʰ percentile over a time span of 6-18 months for children of 2 years and older.
2. 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.
6. 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.
7. Biochemically euthyroid (including when on thyroid hormone supplementation).
8. 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.
9. 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.
10. 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.

Exclusion Criteria

1. Turner Syndrome with presence of Y-chromosomal material on genetic testing and without a history of gonadectomy.
2. Diagnosis of diabetes mellitus.
3. 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.
4. Any known, clinically significant, congenital or acquired cardiac/cardiovascular dysfunction that might interfere with growth as determined by transthoracic echocardiogram.
5. Known history or presence of malignancy.
6. 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.
7. Hepatic transaminases (i.e., AST or ALT) above 3 times the upper limit of normal according to the central laboratory at screening.
8. Major medical conditions and/or presence of contraindication to hGH treatment.
9. Abnormal renal function.
10. 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.
11. Poorly controlled hypertension.
12. 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.
13. Oral/intravenous/intramuscular corticosteroids within 90 days prior to or throughout Screening.
14. Known or suspected hypersensitivity to study intervention(s) or related products.
15. Participation in any other trial involving an investigational compound within 90 days prior to Screening or in parallel to this trial.
16. 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.
17. Female who is pregnant, plans to be pregnant, or is breastfeeding.
Minimum Eligible Age

1 Year

Maximum Eligible Age

10 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Ascendis Pharma Endocrinology Division A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Ascendis Pharma Investigational Site

Boston, Massachusetts, United States

Site Status

Ascendis Pharma Investigational Site

Saint Paul, Minnesota, United States

Site Status

Ascendis Pharma Investigational Site

Las Vegas, Nevada, United States

Site Status

Ascendis Pharma Investigational Site

Lake Success, New York, United States

Site Status

Ascendis Pharma Investigational Site

Palo Alto, California, United States

Site Status

Ascendis Pharma Investigational Site

San Diego, California, United States

Site Status

Ascendis Pharma Investigational Site

Aurora, Colorado, United States

Site Status

Ascendis Pharma Investigational Site

Orlando, Florida, United States

Site Status

Ascendis Pharma Investigational Site

St. Petersburg, Florida, United States

Site Status

Ascendis Pharma Investigational Site

Atlanta, Georgia, United States

Site Status

Ascendis Pharma Investigational Site

Idaho Falls, Idaho, United States

Site Status

Ascendis Pharma Investigational Site

Chicago, Illinois, United States

Site Status

Ascendis Pharma Investigational Site

Chapel Hill, North Carolina, United States

Site Status

Ascendis Pharma Investigational Site

Cincinnati, Ohio, United States

Site Status

Ascendis Pharma Investigational Site

Oklahoma City, Oklahoma, United States

Site Status

Ascendis Pharma Investigational Site

Portland, Oregon, United States

Site Status

Ascendis Pharma Investigational Site

El Paso, Texas, United States

Site Status

Ascendis Pharma Investigational Site

Fort Worth, Texas, United States

Site Status

Ascendis Pharma Investigational Site

Seattle, Washington, United States

Site Status

Countries

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United States

Other Identifiers

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ASND0034

Identifier Type: -

Identifier Source: org_study_id

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