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

Turner Syndrome Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05690386
• Other study ID #: ASND0034
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: February 15, 2023
• Est. completion date: June 2026

Study information

• Verified date: April 2024
• Source: Ascendis Pharma A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A 104 week dose finding open label trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 48
• Est. completion date: June 2026
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 1 Year to 10 Years

Eligibility

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 <25?? 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) <10?? 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.

Related Conditions & MeSH terms

• Gonadal Dysgenesis
• Syndrome
• Turner Syndrome

Intervention

Biological:
• Lonapegsomatropin
Once-weekly subcutaneous injection of Lonapegsomatropin

Drug:
• Somatropin
Once-daily subcutaneous injection of Somatropin

Locations

Country	Name	City	State
United States	Ascendis Pharma Investigational Site	Atlanta	Georgia
United States	Ascendis Pharma Investigational Site	Aurora	Colorado
United States	Ascendis Pharma Investigational Site	Boston	Massachusetts
United States	Ascendis Pharma Investigational Site	Chapel Hill	North Carolina
United States	Ascendis Pharma Investigational Site	Chicago	Illinois
United States	Ascendis Pharma Investigational Site	Cincinnati	Ohio
United States	Ascendis Pharma Investigational Site	El Paso	Texas
United States	Ascendis Pharma Investigational Site	Fort Worth	Texas
United States	Ascendis Pharma Investigational Site	Idaho Falls	Idaho
United States	Ascendis Pharma Investigational Site	Lake Success	New York
United States	Ascendis Pharma Investigational Site	Las Vegas	Nevada
United States	Ascendis Pharma Investigational Site	Oklahoma City	Oklahoma
United States	Ascendis Pharma Investigational Site	Orlando	Florida
United States	Ascendis Pharma Investigational Site	Palo Alto	California
United States	Ascendis Pharma Investigational Site	Portland	Oregon
United States	Ascendis Pharma Investigational Site	Saint Paul	Minnesota
United States	Ascendis Pharma Investigational Site	Saint Petersburg	Florida
United States	Ascendis Pharma Investigational Site	San Diego	California
United States	Ascendis Pharma Investigational Site	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma Endocrinology Division A/S

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Insulin-like growth factor 1 (IGF-1) standard deviation score (SDS)	Via Central Lab analysis	26 weeks, 52 weeks, and 104 weeks
Primary	Annualized Height Velocity (AHV) (cm/year)	Calculated based on the difference between the AHV at 6 months and baseline	26 weeks
Secondary	Annualized Height Velocity (AHV) (cm/year)	Calculated based on the difference between the AHVs at 12 and 24 months and baseline	52 weeks and 104 weeks
Secondary	Change from baseline in height standard deviation score (SDS)	Calculated based on the difference between the heights SDS at 6, 12, and 24 months and baseline.	26 weeks, 52 weeks and 104 weeks
Secondary	Change from baseline in Bone age (calculated years)	Annual change in bone age measurements as per Gruelich-Pyle method	52 weeks and 104 weeks
Secondary	Change from baseline in ratio of bone age/chronological age	Calculated as a ratio	104 weeks