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Trial record 1 of 2 for:    New Insights | Turner Syndrome | United States
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A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05690386
Recruitment Status : Recruiting
First Posted : January 19, 2023
Last Update Posted : April 5, 2024
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S ( Ascendis Pharma Endocrinology Division A/S )

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
Turner Syndrome Biological: Lonapegsomatropin Drug: Somatropin Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated 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)
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
Actual Study Start Date : February 15, 2023
Estimated Primary Completion Date : October 2025
Estimated Study Completion Date : October 2025


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




Primary Outcome Measures :
  1. Annualized Height Velocity (AHV) (cm/year) [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: 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 [ Time Frame: 104 weeks ]
    Calculated as a ratio


Other Outcome Measures:
  1. Insulin-like growth factor 1 (IGF-1) standard deviation score (SDS) [ Time Frame: 26 weeks, 52 weeks, and 104 weeks ]
    Via Central Lab analysis



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   1 Year to 10 Years   (Child)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05690386


Contacts
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Contact: Ascendis Pharma +1 650 352 8389 NewInsiGHTSTrial@Ascendispharma.com

Locations
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United States, California
Ascendis Pharma Investigational Site Recruiting
Los Angeles, California, United States, 90027
Ascendis Pharma Investigational Site Recruiting
Palo Alto, California, United States, 94304
Ascendis Pharma Investigational Site Recruiting
San Diego, California, United States, 92123
United States, Colorado
Ascendis Pharma Investigational Site Recruiting
Aurora, Colorado, United States, 80045
United States, Florida
Ascendis Pharma Investigational Site Recruiting
Orlando, Florida, United States, 32827
Ascendis Pharma Investigational Site Recruiting
Saint Petersburg, Florida, United States, 33701-4804
United States, Georgia
Ascendis Pharma Investigational Site Recruiting
Atlanta, Georgia, United States, 30322
United States, Idaho
Ascendis Pharma Investigational Site Recruiting
Idaho Falls, Idaho, United States, 83404
United States, Illinois
Ascendis Pharma Investigational Site Recruiting
Chicago, Illinois, United States, 60611
United States, Massachusetts
Ascendis Pharma Investigational Site Recruiting
Boston, Massachusetts, United States, 02114
United States, Minnesota
Ascendis Pharma Investigational Site Recruiting
Saint Paul, Minnesota, United States, 55102
United States, Nevada
Ascendis Pharma Investigational Site Recruiting
Las Vegas, Nevada, United States, 89113
United States, New York
Ascendis Pharma Investigational Site Recruiting
Lake Success, New York, United States, 11042
Ascendis Pharma Investigational Site Withdrawn
New York, New York, United States, 10017
Ascendis Pharma Investigational Site Withdrawn
New York, New York, United States, 10029
United States, North Carolina
Ascendis Pharma Investigational Site Recruiting
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Ascendis Pharma Investigational Site Recruiting
Cincinnati, Ohio, United States, 45229
United States, Oklahoma
Ascendis Pharma Investigational Site Recruiting
Oklahoma City, Oklahoma, United States, 73104
United States, Oregon
Ascendis Pharma Investigational Site Recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
Ascendis Pharma Investigational Site Withdrawn
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Ascendis Pharma Investigational Site Recruiting
El Paso, Texas, United States, 79902
Ascendis Pharma Investigational Site Recruiting
Fort Worth, Texas, United States, 76104
United States, Washington
Ascendis Pharma Investigational Site Recruiting
Seattle, Washington, United States, 98105
United States, Wisconsin
Ascendis Pharma Investigational Site Withdrawn
Milwaukee, Wisconsin, United States, 53211
Sponsors and Collaborators
Ascendis Pharma Endocrinology Division A/S
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Responsible Party: Ascendis Pharma Endocrinology Division A/S
ClinicalTrials.gov Identifier: NCT05690386    
Other Study ID Numbers: ASND0034
First Posted: January 19, 2023    Key Record Dates
Last Update Posted: April 5, 2024
Last Verified: April 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Turner Syndrome
Gonadal Dysgenesis
Syndrome
Disease
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
Sex Chromosome Disorders of Sex Development
Male Urogenital Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Sex Chromosome Disorders
Chromosome Disorders
Genetic Diseases, Inborn
Gonadal Disorders
Endocrine System Diseases