Clinical trial NCT03345797: Evaluation of Testosterone Nasal Gel in Hypogonadal Boys
Official title: A Multicenter, Open Label, Variable Dose, Two Arm Pilot Paediatric Phase 1 PK Study to Evaluate Testosterone Nasal Gel (4.5% w/w) in Hypogonadal Boys
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Hypogonadism
Keywords: Paediatric
Lead sponsor: Acerus Pharmaceuticals Corporation
Sponsor class: INDUSTRY
First posted: 2017-11-17
Enrollment: 8
Sex: MALE
Minimum age (years): 12
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: dual arm: naïve vs TRT experienced
Primary purpose: OTHER

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Maximum plasma concentration (Cmax)
timeFrame: 48 hours (approx)
measure: Area under the curve (AUC)
timeFrame: 48 hours (approx)
measure: Minimum serum concentration (Cmin)
timeFrame: 48 hours (approx)
measure: Time to reach maximum plasma concentration (tmax)
timeFrame: 48 hours (approx)
Secondary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events (Safety)
timeFrame: 5 days
measure: Incidence of Treatment-Emergent Adverse Events (Tolerability)
timeFrame: 48 hours (approx)
Other outcomes:
Locations:
city: Glasgow
country: United Kingdom
facility: University of Glasgow, Royal Hospital for Children
state: Scotland
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospital's NHS Foundation Trust
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's Hospital
Study officials:
affiliation: Royal Hospital for Children, U. of Glasgow
name: Syed Faisal Ahmed, MD
role: PRINCIPAL_INVESTIGATOR
References: