Evaluation of Testosterone Nasal Gel in Hypogonadal Boys

Phase 1

Terminated

• Conditions: Hypogonadism
• Interventions: Drug: Testosterone Nasal Gel [Natesto]

• Registration Number: NCT03345797
• Lead Sponsor: Acerus Biopharma Inc.

Brief Summary

PK study to evaluate serum levels of testosterone post nasal delivery in two cohorts of hypogonadal boys.

Detailed Description

ARM 1 - 10 prepubertal, 12-17 years old boys with no prior exposure to TRT will receive single dose of 5.5 mg on day one and single dose of 11 mg on day 2 with repeat blood draws to assess serum levels of testosterone and metabolites.

ARM 2 - 10 Tanner Stage 3, 12-17 years old boys on TRT with bone age \>= 13 years will receive single dose of 11 mg on day one, and single dose of 11 mg in the morning and a second 11 mg dose in the afternoon on day 2 with repeat blood draws to assess serum levels of testosterone and metabolites.

Study Timeline

• Study First Submitted: 2017-10-10
• Study First Submitted QC: 2017-11-14
• Study First Posted: 2017-11-17
• Study Start: 2018-03-01
• Primary Completion: 2020-04-11
• Study Completion: 2022-04-11
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-24
• Last Update Posted: 2023-10-26

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 8

Inclusion Criteria

ARM 1 (naïve patients): Participants who meet all of the following inclusion criteria will be eligible for participation in the study:

1. Hypogonadal boys;
2. Chronological age 12 to <18 years;
3. No prior exposure to TRT;
4. Prepubertal
5. Parent/guardian and patient able to understand and provide signed informed consent;

ARM 2 (non-naïve patients): Participants who meet all of the following inclusion criteria will be eligible for participation in the study:

1. Hypogonadal boys with a bone age of ≥13 years (a historical value within the last 12 months will be acceptable);
2. Chronological age 12 to <18 years;
3. Taking an existing TRT treatment dose;
4. Tanner Stage ≥3
5. Parent/guardian and patient able to understand and provide signed informed consent;

Exclusion Criteria

ARM 1 (naïve patients) AND ARM 2 (non-naïve patients): Participants who meet any of the following criteria will be excluded from participation in the study:

1. Any active allergic condition or presentation of symptoms including allergic rhinitis;
2. An upper respiratory tract infection;
3. Use of any form of intranasal medication delivery other than periodic short-term (less than 3 days) use of sympathomimetic decongestants, within the last 3 months;
4. In the opinion of the Investigator, significant intercurrent disease of any type, in particular liver, kidney, heart disease, stroke, or psychiatric illness;
5. History of pituitary or hypothalamic tumors or history of any malignancy excluding basal cell or squamous cell carcinoma of the skin curatively treated by surgery;
6. History of nasal disorders, nasal or sinus surgery, nasal fracture within the previous 6 months or nasal fracture that caused a deviated anterior nasal septum surgery, mucosal inflammatory disorders, specifically Sjogren's syndrome;
7. History of severe adverse drug reactions to testosterone therapies;
8. Current treatment with other androgens (e.g., dehydroepiandrosterone [DHEA]), anabolic steroids, or other sex hormones;
9. Treatment with estrogens, gonadotropin-releasing hormone (GnRH) agonists, or growth hormone within the previous 12 months;
10. Treatment with drugs that interfere with the metabolism of testosterone, such as anastrozole, clomiphene, dutasteride, finasteride, flutamide, ketoconazole, spironolactone, or testolactone;
11. Diabetes mellitus;
12. Participation in any other research study during the conduct of this study or 30 days prior to the initiation of this study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Naive patients - ARM 1	Testosterone Nasal Gel [Natesto]	TRT naïve subjects, Tanner Stage of 0, receiving Testosterone Nasal Gel \[Natesto\] - Single dose of 5.5 mg Natesto on Day 1 and a single dose of 11 mg Natesto on Day 2
Non-naive patients - ARM 2	Testosterone Nasal Gel [Natesto]	TRT non-naïve subjects (have had prior testosterone treatment), Tanner Stage \>/= 3, receiving Testosterone Nasal Gel \[Natesto\] - Single dose of 11 mg Natesto on Day 1. On Day 2, 11 mg dose of Natesto in the morning and 11 mg dose of Natesto 12 hours later

Primary Outcome Measures

Name	Time	Method
Maximum plasma concentration (Cmax)	48 hours (approx)	Cmax for serum testosterone, serum DHT and serum estradiol
Area under the curve (AUC)	48 hours (approx)	AUC for serum testosterone, serum DHT and serum estradiol
Minimum serum concentration (Cmin)	48 hours (approx)	Cmin for serum testosterone, serum DHT and serum estradiol
Time to reach maximum plasma concentration (tmax)	48 hours (approx)	tmax for serum testosterone, serum DHT and serum estradiol

Secondary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events (Safety)	5 days	Safety assessments will include adverse events, clinical laboratory measurements of serum testosterone, DHT and estradiol levels, and vital sign measurements. A phone call will be made 3 days after the patient leaves the clinic to collect any SAEs or AEs that may occur.
Incidence of Treatment-Emergent Adverse Events (Tolerability)	48 hours (approx)	To include a adverse events that relate to tolerability and a patient and healthcare provider questionnaire on ease of use dispenser and gel administration

Trial Locations

Locations (3)

University of Glasgow, Royal Hospital for Children; 🇬🇧 Glasgow, Scotland, United Kingdom

Alder Hey Children's Hospital; 🇬🇧 Liverpool, United Kingdom

Cambridge University Hospital's NHS Foundation Trust; 🇬🇧 Cambridge, United Kingdom