Testing XYOSTED for testosterone replacement in adolescent males with hypogonadism

Open-Label, Multiple-Dose, 52-Week Study + 24-Month Long-Term Safety Extension to Evaluate the Safety, PK, & Efficacy of XYOSTED® for Testosterone Replacement in Male Adolescents With Deficiency or Absence of Endogenous Testosterone Due to Primary or Secondary Hypogonadism

This study is testing if a new testosterone treatment called XYOSTED can help teenage boys with low testosterone levels go through puberty safely and effectively.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 12 Years to 17 Years
• Sex: Male
• Sponsor: Halozyme Therapeutics (Industry-sponsored)
• Locations: 20 sites (Los Angeles, California and 19 other locations)
• Trial ID: NCT06689085 on ClinicalTrials.gov

What this trial studies

This is a 52-week open-label study investigating the safety, pharmacokinetics, and efficacy of XYOSTED, a testosterone replacement therapy, in male adolescents aged 12 to under 18 with primary or secondary hypogonadism. The study will assess the effectiveness of XYOSTED by monitoring the continuation or induction of puberty, dosage adjustments based on testosterone levels, and overall safety. Participants will undergo a thorough screening process, including clinical examinations and testosterone measurements, before starting treatment. The study aims to achieve targeted testosterone levels through dose adjustments made every three months.

Who should consider this trial

Good fit: Ideal candidates are male adolescents aged 12 to under 18 diagnosed with primary or secondary hypogonadism requiring testosterone therapy.

Not a fit: Patients who do not have a deficiency or absence of endogenous testosterone or those with conditions unrelated to hypogonadism may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide an effective testosterone replacement therapy option for adolescent males with hypogonadism, aiding in their pubertal development.

How similar studies have performed: Other studies have shown success with testosterone replacement therapies in similar populations, indicating a potential for positive outcomes in this approach.

Eligibility criteria

Inclusion Criteria:

1. Diagnosed with a deficiency or absence of endogenous testosterone due to primary or secondary hypogonadism of a known etiology. Children with combined hormone deficiencies are permitted to enroll (but the child must already be receiving treatment for concomitant hormonal deficiencies)
2. Participants receiving prior testosterone treatment must be receiving a stable dose for at least 12 weeks prior to Screening. Treatment naive participants are permitted to enroll.
3. Have parent(s) or a legal guardian who will voluntarily provide written informed consent for the child to participate in the study
4. Willing to provide assent for participation in the study
5. Be a male 12 to \< 18 years of age at the time of consent/assent
6. Have Legally Authorized Representative who is able to understand and comply with all study procedures and agrees to have the child participate in the study program as outlined in the protocol
7. Requires chronic pharmacologic support for the initiation and/or continuation of pubertal maturation
8. Have a body mass index (BMI)-for-age greater than the 5th percentile and weigh ≥ 30 kg.
9. If sexually active with a female partner of child-bearing potential, agrees to:

   1. Practice true abstinence including 30 days after the last IP administration, or,
   2. Use 2 adequate forms of highly effective contraception, one of which should be a physical barrier, during the study and for 30 days after the last IP administration.

Exclusion Criteria:

1. Has abnormal thyroid function tests at Screening. May supplement per usual clinical practice and rescreen up to two times.
2. Has suspected or known constitutional growth delay in growth and puberty (CDGP)
3. Has evidence of possible nutritional or gastrointestinal disorder that may impact growth (e.g., abrupt weight loss within the 3 months prior to Screening, unmanaged celiac disease, inflammatory bowel disease)
4. Has a known allergy or hypersensitivity to XYOSTED, or to any of its ingredients (testosterone enanthate and sesame oil)
5. Participants receiving prior treatment with testosterone who are not on a stable dose for at least 12 weeks prior to Screening.
6. Has an allergy to foods or products containing sesame seeds or sesame oil
7. Has Stage 1 hypertension, defined as the average of 2 or more seated right arm BP measurements exceeding the 95th percentile for age, sex, and height, or SBP ≥ 130 mm Hg and/or DBP ≥ 80 mm Hg at Screening or Day 1.
8. Has a clinically significant abnormal clinical laboratory test value at Screening, as determined by the Investigator including hematocrit \> 48%, or \>50% for patients living at high altitude if not receiving testosterone treatment, or hematocrit \> 52% if already receiving testosterone treatment.
9. Has a history of deep venous thrombosis or pulmonary embolism
10. Has evidence of a clinically significant 12-lead electrocardiogram (ECG) abnormality at Screening, as determined by the Investigator
11. Has a current suspected or diagnosed (and unresected) tumor of the pituitary gland with the exception of Rathke's cleft cyst or a stable non-functioning pituitary microadenoma (ie, lesion size \< 10 mm that has not increased in size over a period of 1 year on repeat imaging), as determined by the Investigator
12. Has an active malignancy or has received treatment for a malignancy within the 12 months before Screening
13. Is currently receiving antipsychotic medication for any reason or is currently receiving selective serotonin reuptake inhibitor (SSRI) medication for depression
14. Is receiving any other medication or has a condition that would preclude safe participation in the study or confound the evaluation of safety, as determined by the Investigator
15. Has a history of suicidal behavior (i.e., actions intended to harm oneself), suicidal ideation (i.e., thoughts and plans about suicide), or suicide attempts
16. Has any affirmative responses on the Columbia Suicide Severity Rating Scale (C-SSRS) questionnaire to questions #3, #4, or #5 or any affirmative response to questions #1 or #2 within the past 12 months on the suicide ideation questions (first section) OR any affirmative response on the suicidal behavior questions (the second section).
17. Is currently taking supraphysiologic doses of systemic glucocorticoids for more than 3 weeks, except for intermittent short courses of exogenous glucocorticoids as needed for the treatment of asthma
18. Has received any other investigational compound within 1 month prior to screening or 5 half-lives of the investigational product (whichever is longer)
19. Has received gonadotropin-releasing hormone (GnRH) agonists, aromatase inhibitors, androgens (eg, dehydroepiandrosterone \[DHEA\]), anabolic steroids such as oxandrolone, or other sex steroids within 12 months before the Screening visit, or would require these treatments at any time during the study.
20. Receiving cytochrome P450 (CYP) 3A4 or P glycoprotein (P-gp) inhibitors/inducers or medications that are metabolized by CYP3A4 or P-gp within 30 days of enrolment.
21. Has a history of alcohol or drug abuse
22. Has a history or clinical manifestations of significant renal, hepatic, cardiovascular, metabolic, neurologic, psychiatric, or other conditions that would preclude participation in the study, as determined by the Investigator
23. Has chronic urticaria or dermatographism
24. Has 25-hydroxy-vitamin D blood level \< 20 ng/mL. Participants with initial vitamin D blood measurement \< 20 ng/mL may enroll while they receive supplementation per clinical practice

Where this trial is running

Los Angeles, California and 19 other locations

• Children's Hospital Los Angeles — Los Angeles, California, United States (Recruiting)
• Rady Children's Hospital - San Diego — San Diego, California, United States (Recruiting)
• University of California San Francisco — San Francisco, California, United States (Recruiting)
• Children's Hospital Colorado — Aurora, Colorado, United States (Recruiting)
• Nemours Children's Specialty Care - Jacksonville — Jacksonville, Florida, United States (Recruiting)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (Recruiting)
• Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
• University of Massachusetts Memorial Medical Center — Worcester, Massachusetts, United States (Recruiting)
• M Health Fairview U Minnesota — Minneapolis, Minnesota, United States (Recruiting)
• Washington University School of Medicine in St. Louis — St Louis, Missouri, United States (Recruiting)
• The DOCS — Las Vegas, Nevada, United States (Recruiting)
• Children's Hospital at Montefiore — The Bronx, New York, United States (Recruiting)
• OUHSC Pediatric Diabetes & Endocrinology — Oklahoma City, Oklahoma, United States (Recruiting)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
• Prisma Health Children's Hospital - Midlands — Columbia, South Carolina, United States (Recruiting)
• MedResearch — El Paso, Texas, United States (Recruiting)
• Texas Children's Hospital — Houston, Texas, United States (Recruiting)
• Primary Children's Hospital — Salt Lake City, Utah, United States (Recruiting)
• Seattle Children's Hospital — Seattle, Washington, United States (Recruiting)
• MultiCare Institute for Research & Innovation — Tacoma, Washington, United States (Recruiting)

Study contacts

• Study coordinator: Director Clinical Operations, MPH, CPM

Conditions Hypogonadism, Male; primary hypogonadism; secondary hypogonadism