Evaluation of Testosterone Nasal Gel in Hypogonadal Boys

• ClinicalTrials.gov Identifier: NCT03345797
• Recruitment Status: Unknown
• First Posted: November 17, 2017
• Last Update Posted: July 5, 2019
• Sponsor: Acerus Biopharma Inc.
• Information provided by (Responsible Party): Acerus Pharmaceuticals Corporation ( Acerus Biopharma Inc. )

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Hypogonadism	Drug: Testosterone Nasal Gel [Natesto]	Phase 1

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 Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 20 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Intervention Model Description: dual arm: naïve vs TRT experienced
• Masking: None (Open Label)
• Primary Purpose: Other
• 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
• Actual Study Start Date: March 1, 2018
• Estimated Primary Completion Date: November 30, 2020
• Estimated Study Completion Date: November 30, 2020

Arms and Interventions

Arm	Intervention/treatment
Experimental: Naive patients - ARM 1; 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	Drug: Testosterone Nasal Gel [Natesto]; nasal gel containing 4.5% w/w testosterone
Experimental: Non-naive patients - ARM 2; 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	Drug: Testosterone Nasal Gel [Natesto]; nasal gel containing 4.5% w/w testosterone

Outcome Measures

Primary Outcome Measures :

1. Maximum plasma concentration (Cmax) [ Time Frame: 48 hours (approx) ]
   Cmax for serum testosterone, serum DHT and serum estradiol
2. Area under the curve (AUC) [ Time Frame: 48 hours (approx) ]
   AUC for serum testosterone, serum DHT and serum estradiol
3. Minimum serum concentration (Cmin) [ Time Frame: 48 hours (approx) ]
   Cmin for serum testosterone, serum DHT and serum estradiol
4. Time to reach maximum plasma concentration (tmax) [ Time Frame: 48 hours (approx) ]
   tmax for serum testosterone, serum DHT and serum estradiol

Secondary Outcome Measures :

1. Incidence of Treatment-Emergent Adverse Events (Safety) [ Time Frame: 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.
2. Incidence of Treatment-Emergent Adverse Events (Tolerability) [ Time Frame: 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

Eligibility Criteria

• Ages Eligible for Study: 12 Years to 17 Years (Child)
• Sexes Eligible for Study: Male
• Gender Based Eligibility: Yes
• Gender Eligibility Description: hypogonadal boys
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Contacts

Contact: Matthew Gronski, Ph.D.; 289-334-1461; mgronski@aceruspharma.com

Contact: Nathan Bryson, Ph.D.; 416-679-0776; nbryson@aceruspharma.com

Locations

United Kingdom

University of Glasgow, Royal Hospital for Children; Recruiting

Glasgow, Scotland, United Kingdom, G51 4TF

Contact: Faisal S Ahmed, MD +44 141 451 5841 Faisal.Ahmed@glasgow.ac.uk

Principal Investigator: Faisal S Ahmed, MD

Cambridge University Hospital's NHS Foundation Trust; Recruiting

Cambridge, United Kingdom, CB2 0QQ

Contact: Carlo Acerini, MD +44 1223 336865 cla22@cam.ac.uk

Principal Investigator: Carlo L Acerini, MD

Alder Hey Children's Hospital; Not yet recruiting

Liverpool, United Kingdom, L14 5AB

Principal Investigator: Dan Hawcutt

Sponsors and Collaborators

Acerus Biopharma Inc.

Investigators

• Principal Investigator: Syed Faisal Ahmed, MD; Royal Hospital for Children, U. of Glasgow

More Information

• Responsible Party: Acerus Biopharma Inc.
• ClinicalTrials.gov Identifier: NCT03345797
• Other Study ID Numbers: NAT-2017-01
• First Posted: November 17, 2017
• Last Update Posted: July 5, 2019
• Last Verified: July 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Acerus Pharmaceuticals Corporation ( Acerus Biopharma Inc. ):

Paediatric

Additional relevant MeSH terms:

Hypogonadism; Gonadal Disorders; Endocrine System Diseases; Testosterone; Androgens; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs