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Undersøgelse af effektiviteten og sikkerheden af ugentlig lonapegsomatropin sammenlignet med daglig somatropin hos børn og unge med lav vækst eller vækstsvigt på grund af væksthormonsufficiente lidelser

29. september 2026 opdateret af: Ascendis Pharma A/S

En afgørende, parallel-arm, fase 3, åben-label, aktivt kontrolleret, global, multicenter, randomiseret kurvundersøgelse, der undersøger effektiviteten og sikkerheden af én gang om ugen lonapegsomatropin sammenlignet med daglig somatropin hos prepubertale børn og unge med væksthæmning eller lav højde på grund af væksthormon-suffisiente lidelser - Turner syndrom, SHOX-mangel, lille for gestationsalder og idiopatisk lav højde

This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates. The purpose of the study is to see how well treatment with once-weekly lonapegsomatropin works compared to treatment with daily somatropin. Approximately 186 participants will be distributed equally (1:1), to receive either lonapegsomatropin for 2 years or somatropin for 1 year followed by lonapegsomatropin for 1 year. This trial will be conducted in the United States, France, Germany, Italy, Romania, Spain and South Korea.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

186

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

  • Forenede Stater
    • California
      • Palo Alto, California, Forenede Stater, 94304
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Sacramento, California, Forenede Stater, 95821
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Colorado
      • Aurora, Colorado, Forenede Stater, 80045
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Centennial, Colorado, Forenede Stater, 80112
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Delaware
      • Wilmington, Delaware, Forenede Stater, 19803
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • District of Columbia
      • Washington D.C., District of Columbia, Forenede Stater, 20010
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Florida
      • Orlando, Florida, Forenede Stater, 32806
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • St. Petersburg, Florida, Forenede Stater, 33701
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Georgia
      • Atlanta, Georgia, Forenede Stater, 30329
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Idaho
      • Idaho Falls, Idaho, Forenede Stater, 83404
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Louisiana
      • New Orleans, Louisiana, Forenede Stater, 70118
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Minnesota
      • Minneapolis, Minnesota, Forenede Stater, 55454
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Saint Paul, Minnesota, Forenede Stater, 55102
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • New York
      • Lake Success, New York, Forenede Stater, 11042
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Ohio
      • Cincinnati, Ohio, Forenede Stater, 45229
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Oklahoma
      • Oklahoma City, Oklahoma, Forenede Stater, 73104
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Texas
      • Edinburg, Texas, Forenede Stater, 78539
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Fort Worth, Texas, Forenede Stater, 76104
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • San Antonio, Texas, Forenede Stater, 78229
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • San Antonio, Texas, Forenede Stater, 78232
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Utah
      • Salt Lake City, Utah, Forenede Stater, 84112
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Virginia
      • Charlottesville, Virginia, Forenede Stater, 22908
        • Rekruttering
        • Ascendis Pharma Investigational Site
    • Washington
      • Seattle, Washington, Forenede Stater, 98105
        • Rekruttering
        • Ascendis Pharma Investigational Site
  • Frankrig
      • Angers, Frankrig, 49933
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Le Kremlin-Bicêtre, Frankrig, 94270
        • Rekruttering
        • Ascendis PharmaInvestigational Site
      • Montpellier, Frankrig, 34090
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Paris, Frankrig, 75012
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Toulouse, Frankrig, 31300
        • Rekruttering
        • Ascendis Pharma Investigational Site
  • Italien
      • Florence, Italien, 50139
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Genova, Italien, 16147
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Naples, Italien, 80131
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Naples, Italien, 80138
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Trieste, Italien, 34123
        • Rekruttering
        • Ascendis Pharma Investigational Site
  • Rumænien
      • Iași, Rumænien, 700106
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Târgu Mureş, Rumænien, 540136
        • Rekruttering
        • Ascendis Pharma Investigational Site
  • Spanien
      • Barcelona, Spanien, 08035
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Barcelona, Spanien, 8950
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Madrid, Spanien, 28046
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Málaga, Spanien, 29011
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Zaragoza, Spanien, 50009
        • Rekruttering
        • Ascendis Pharma Investigational Site
  • Sydkorea
      • Sejong, Sydkorea, 30099
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Seongnam, Sydkorea, 13620
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Seoul, Sydkorea, 06273
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Seoul, Sydkorea, 03722
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Seoul, Sydkorea, 05505
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Seoul, Sydkorea, 05278
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Suwon, Sydkorea, 16499
        • Rekruttering
        • Ascendis Pharma Investigational Site
  • Tyskland
      • Cologne, Tyskland, 50931
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Düsseldorf, Tyskland, 40225
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Hanover, Tyskland, 30173
        • Rekruttering
        • Ascendis Pharma Investigational Site
      • Tübingen, Tyskland, 72076
        • Rekruttering
        • Ascendis Pharma Investigational Site

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

2 år til 17 år (Barn)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Inclusion Criteria:

  1. Chronological age between ≥2 and <18 years, at start of screening.

  2. Naïve to growth hormone and growth hormone promoting therapies.

  3. Prepubertal.

  4. Able to stand without assistance.

  5. Diagnosis of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according to the following disease-specific criteria:

    TS or SHOX-D (Léri-Weill dyschondrosteosis):

    1. Diagnosis confirmed by a genetic test. NOTE: Historical test results are acceptable for proof of diagnosis. For karyotypes, a minimum of 20 cells must be counted.
    2. Impaired growth or short stature defined as:

    (i.) AHV <25th percentile over a time span of 6-16 months prior to screening utilizing a historical height properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height <5th percentile for sex and age according to the Centers for Disease Control Growth Charts for the United States

    SGA without catch-up growth:

    c. Birth weight and/or birth length < -2.0 SDS for gestational age according to the 2006 World Health Organization Child Growth Standards. For infants born premature, the Fenton Preterm Infant Growth Chart (Fenton 2013) should be used.

    d. Impaired growth or short stature defined as: (i.) AHV <25th percentile over a time span of 6-16 months prior to screening properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height < -2.0 SDS for age and sex according to the 2000 Centers for Disease Control Growth Charts for the United States for children ≥ 3 years or height < -2.5 SDS for age and sex according to the for children ≥ 2 years and < 3 years

    ISS:

    e. Height < -2.25 SDS for sex and age according to the Centers for Disease Control Growth Charts for the United States with no identifiable cause for short stature.

    f. Documented normal GH-IGF-1 axis, defined as either: (i.)IGF-1 SDS >0 at screening based on central laboratory OR (ii.)Historical documentation of normal peak GH upon stimulation test (as defined by local institution) g. 46,XX chromosome as determined by karyotype or microarray if female. For karyotypes, a minimum of 30 cells must be counted.

  6. 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.

  7. Written, signed informed consent provided by parent(s) or legal guardian(s) of the participant. Assent should be signed by participant as required by IRB/HREC/IEC.

Exclusion Criteria:

  1. Advanced bone age X-ray by central reading defined as >20% above chronological age in months (Greulich 1959).

  2. Closed epiphyses as defined as bone age of ≥14.0 years in females or ≥16.0 years in males.

  3. Current clinical diagnosis of diabetic retinopathy

  4. Any diagnosis or presence at screening of the following:

    1. Untreated moderate or severe sleep apnea as determined by formal (local) read of an inpatient or at-home sleep study.
    2. Prader Willi syndrome with severe obesity, history of severe upper airway obstruction, or severe respiratory impairment.

  5. Signs/symptoms of intracranial hypertension, active proliferative retinopathy.

  6. Uncontrolled hypo- or hyperthyroidism.

  7. Uncontrolled diabetes mellitus (defined as: HbA1c >7.5% from central laboratory at screening).

  8. Known history or diagnosis of any gastrointestinal inflammatory condition, HIV, radiation exposure, other skeletal dysplasias, growth hormone deficiency, and/or cardio-thoracic surgery due to their independent effects on growth.

  9. Any significant hepatic or renal abnormality, such as abnormal renal function (defined as eGFR <60 mL/min/1.73m2).

  10. Undiagnosed or uncontrolled hypertension.

  11. Receiving treatment with any agent that might influence growth or interfere with GH secretion or action including any sex steroids and stimulants for attention-deficit/hyperactivity disorder (ADHD).

  12. High dose inhaled glucocorticoid for more than 28 consecutive days total over the course of 12 months.

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

  14. Participation in another interventional clinical trial involving an investigational compound within 90 days prior to screening or in parallel to this trial.

  15. Any disease or condition that, in the judgement of the investigator, may make the participant unlikely to comply with the requirements of the protocol or any condition that presents undue risk from the investigational product or trial procedures.

  16. Exclusion Criteria only applicable to TS:

    1. Presence of Y chromosome material on genetic testing without history of gonadectomy.
    2. Less than 10% of 45,X mosaicism.
    3. Any known, clinically significant, congenital or acquired cardiovascular dysfunction that might interfere with growth.

  17. Exclusion Criteria only applicable to SGA:

    a. Any known clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements: (i.)Chromosomal aneuploidy, significant gene mutations, or medical syndromes with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, abnormal SHOX-1 gene analysis or absence of GH receptors.

    (ii.)Congenital abnormalities (causing skeletal abnormalities), including but not limited to skeletal dysplasias.

  18. Exclusion Criteria only applicable to ISS:

    1. Known history of any condition that causes disproportionate short stature (i.e. skeletal dysplasias), chromosomal aneuploidy, significant gene mutations, or medical syndromes with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, abnormal SHOX-1 gene analysis or absence of gH receptors.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Lonapegsomatropin, once daily

Participants will receive Lonapegsomatropin by subcutaneous injection for 2 years (104 weeks)
Kombinationsprodukt: Lonapegsomatropin [SKYTROFA®]

Subcutaneous injection once weekly
Aktiv komparator: somatropin, once daily

Participants will receive somatropin by subcutaneous injection for 1 year (52 weeks) followed by lonapegsomatropin for 1 year (52 weeks)
Kombinationsprodukt: Somatropin Pen Injector

Subcutaneous injection once daily

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Annualized Height Velocity (AHV) (cm/year)
Tidsramme: 52 Weeks

To evaluate the efficacy of lonapegsomatropin as compared to somatropin in children and adolescents with TS, SHOX-D, SGA, or ISS
52 Weeks

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Annualized Height Velocity (AHV) (cm/year)
Tidsramme: 104 Weeks

To evaluate lonapegsomatropin as compared to somatropin on additional measures of efficacy in children and adolescents with TS, SHOX-D, SGA, or ISS
104 Weeks
Change from baseline in height standard deviation score (SDS)
Tidsramme: 52 Weeks and 104 Weeks

To evaluate lonapegsomatropin as compared to somatropin on additional measures of efficacy in children and adolescents with TS, SHOX-D, SGA, or ISS
52 Weeks and 104 Weeks
Change from baseline in Bone Age (years)
Tidsramme: 52 Weeks and 104 Weeks

To evaluate the safety and tolerability of lonapegsomatropin in children and adolescents with TS, SHOX-D, SGA, or ISS via Central Reader
52 Weeks and 104 Weeks
Change from baseline in ratio of Bone Age/chronological age
Tidsramme: 52 Weeks and 104 Weeks

To evaluate the safety and tolerability of lonapegsomatropin in children and adolescents with TS, SHOX-D, SGA, or ISS via Central Reader
52 Weeks and 104 Weeks
Number of participants with treatment-related adverse events (AEs)
Tidsramme: 52 Weeks and 104 Weeks

To evaluate the safety and tolerability of lonapegsomatropin in children and adolescents with TS, SHOX-D, SGA, or ISS
52 Weeks and 104 Weeks

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Ascendis Pharma A/S

Efterforskere

  • Studieleder: Medical Director, MD, Ascendis Pharma A/S

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

12. december 2025

Primær færdiggørelse (Anslået)

1. februar 2028

Studieafslutning (Anslået)

1. marts 2029

Datoer for studieregistrering

Først indsendt

14. oktober 2025

Først indsendt, der opfyldte QC-kriterier

24. oktober 2025

Først opslået (Faktiske)

28. oktober 2025

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

30. september 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

29. september 2026

Sidst verificeret

1. september 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • ASND0047

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ja

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Abonner