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

Sponsor
Ascendis Pharma Endocrinology Division A/S (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05690386
Collaborator
(none)
48
Enrollment
18
Locations
4
Arms
33
Anticipated Duration (Months)
2.7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
48 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
An open label, parallel group with subjects randomized into 1 of 4 treatment groups (1:1:1:1)An open label, parallel group with subjects randomized into 1 of 4 treatment groups (1:1:1:1)
Masking:
None (Open Label)
Primary Purpose:
Treatment
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
Anticipated Study Start Date :
Jan 1, 2023
Anticipated Primary Completion Date :
Oct 1, 2025
Anticipated Study Completion Date :
Oct 1, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: Lonapegsomatropin at 0.24 mg hGH/kg/week

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

Biological: Lonapegsomatropin
Once-weekly subcutaneous injection of Lonapegsomatropin
Experimental: Lonapegsomatropin at 0.30 mg hGH/kg/week

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

Biological: Lonapegsomatropin
Once-weekly subcutaneous injection of Lonapegsomatropin
Experimental: Lonapegsomatropin at 0.36 mg hGH/kg/week

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

Biological: Lonapegsomatropin
Once-weekly subcutaneous injection of Lonapegsomatropin
Active Comparator: Somatropin at 0.05 mg/kg/day

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

Drug: Somatropin
Once-daily subcutaneous injection of Somatropin

Outcome Measures

Primary Outcome Measures

  1. Annualized Height Velocity (AHV) (cm/year) [26 weeks]

    Calculated based on the difference between the AHV at 6 months and baseline

Secondary Outcome Measures

  1. Annualized Height Velocity (AHV) (cm/year) [52 weeks and 104 weeks]

    Calculated based on the difference between the AHVs at 12 and 24 months and baseline

  2. Change from baseline in height standard deviation score (SDS) [26 weeks, 52 weeks and 104 weeks]

    Calculated based on the difference between the heights SDS at 6, 12, and 24 months and baseline.

  3. Change from baseline in Bone age (calculated years) [52 weeks and 104 weeks]

    Annual change in bone age measurements as per Gruelich-Pyle method

  4. Change from baseline in ratio of bone age/chronological age [104 weeks]

    Calculated as a ratio

Other Outcome Measures

  1. Insulin-like growth factor 1 (IGF-1) standard deviation score (SDS) [26 weeks, 52 weeks, and 104 weeks]

    Via Central Lab analysis

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Year to 10 Years
Sexes Eligible for Study:
Female
Accepts Healthy Volunteers:
No
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:

  6. AHV< 6 cm/year or <25ᵗʰ percentile over a time span of 6-18 months for children of 2 years and older.

  7. 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.

  8. Bone age at or below chronological age on left wrist radiograph at Screening.

  9. Biochemically euthyroid (including when on thyroid hormone supplementation).

  10. 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.

  11. 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.

  12. 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.

  1. Hepatic transaminases (i.e., AST or ALT) above 3 times the upper limit of normal according to the central laboratory at screening.

  2. Major medical conditions and/or presence of contraindication to hGH treatment.

  3. Abnormal renal function.

  4. 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.

  5. Poorly controlled hypertension.

  6. 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.

  7. Oral/intravenous/intramuscular corticosteroids within 90 days prior to or throughout Screening.

  8. Known or suspected hypersensitivity to study intervention(s) or related products.

  9. Participation in any other trial involving an investigational compound within 90 days prior to Screening or in parallel to this trial.

  10. 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.

  11. Female who is pregnant, plans to be pregnant, or is breastfeeding.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Ascendis Pharma Investigational Site Los Angeles California United States 90027
2 Ascendis Pharma Investigational Site Palo Alto California United States 94304
3 Ascendis Pharma Investigational Site San Diego California United States 92123
4 Ascendis Pharma Investigational Site Aurora Colorado United States 80045
5 Ascendis Pharma Investigational Site Orlando Florida United States 32827
6 Ascendis Pharma Investigational Site Chicago Illinois United States 60611
7 Ascendis Pharma Investigational Site Boston Massachusetts United States 02114
8 Ascendis Pharma Investigational Site Saint Paul Minnesota United States 55102
9 Ascendis Pharma Investigational Site New York New York United States 10017
10 Ascendis Pharma Investigational Site New York New York United States 10029
11 Ascendis Pharma Investigational Site Chapel Hill North Carolina United States 27599
12 Ascendis Pharma Investigational Site Cincinnati Ohio United States 45229
13 Ascendis Pharma Investigational Site Oklahoma City Oklahoma United States 73104
14 Ascendis Pharma Investigational Site Portland Oregon United States 97239
15 Ascendis Pharma Investigational Site Philadelphia Pennsylvania United States 19104
16 Ascendis Pharma Investigational Site Fort Worth Texas United States 76104
17 Ascendis Pharma Investigational Site Seattle Washington United States 98105
18 Ascendis Pharma Investigational Site Milwaukee Wisconsin United States 53211

Sponsors and Collaborators

  • Ascendis Pharma Endocrinology Division A/S

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Ascendis Pharma Endocrinology Division A/S
ClinicalTrials.gov Identifier:
NCT05690386
Other Study ID Numbers:
  • ASND0034
First Posted:
Jan 19, 2023
Last Update Posted:
Jan 25, 2023
Last Verified:
Jan 1, 2023
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Turner Syndrome
Gonadal Dysgenesis
Syndrome

Study Results

No Results Posted as of Jan 25, 2023