Trial Outcomes & Findings for Study of Testosterone and rHGH in FSHD (NCT NCT03123913)

NCT ID: NCT03123913

Last Updated: 2023-05-16

Results Overview

AEs were collected through patient report, interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

20 participants

Primary outcome timeframe

36 weeks

Results posted on

2023-05-16

Participant Flow

Participant milestones

Participant milestones
Measure
Combination Therapy
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Overall Study
STARTED
20
Overall Study
COMPLETED
19
Overall Study
NOT COMPLETED
1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Testosterone and rHGH in FSHD

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Combination Therapy
n=20 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Age, Continuous
41 years
STANDARD_DEVIATION 10 • n=5 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
Sex: Female, Male
Male
20 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
20 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
19 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
United States
20 participants
n=5 Participants

PRIMARY outcome

Timeframe: 36 weeks

AEs were collected through patient report, interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations.

Outcome measures

Outcome measures
Measure
Combination Therapy
n=20 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Number of Participants Who Experienced an AE
14 Participants

SECONDARY outcome

Timeframe: Baseline to 24 weeks

Outcome measures

Outcome measures
Measure
Combination Therapy
n=19 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Mean Change in Level of Free Testosterone in the Blood
17.42 pg/mL
Standard Deviation 77.94

SECONDARY outcome

Timeframe: Baseline to 24 weeks

Outcome measures

Outcome measures
Measure
Combination Therapy
n=19 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Mean Change in Level of Total Testosterone in the Blood
8.26 ng/dL
Standard Deviation 277.94

SECONDARY outcome

Timeframe: Baseline to 24 weeks

Outcome measures

Outcome measures
Measure
Combination Therapy
n=19 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Mean Change in Level of IGF-1 in the Blood
89.79 ng/mL
Standard Deviation 71.57

SECONDARY outcome

Timeframe: Baseline to 24 weeks

Outcome measures

Outcome measures
Measure
Combination Therapy
n=19 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Mean Change in Level of TSH in the Blood
0.37 uIU/mL
Standard Deviation 0.66

SECONDARY outcome

Timeframe: Baseline to 24 weeks

Outcome measures

Outcome measures
Measure
Combination Therapy
n=19 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Mean Change in Level of Luteinizing Hormone in the Blood
-3.48 mIU/mL
Standard Deviation 2.64

SECONDARY outcome

Timeframe: Baseline to 24 weeks

Outcome measures

Outcome measures
Measure
Combination Therapy
n=19 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Mean Change in Level of FSH in the Blood
-3.29 mIU/mL
Standard Deviation 4.47

SECONDARY outcome

Timeframe: Baseline to 24 weeks

Outcome measures

Outcome measures
Measure
Combination Therapy
n=19 Participants
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Mean Change in Total Lean Body Mass
2.21 Kg
Standard Deviation 1.79

Adverse Events

Combination Therapy

Serious events: 0 serious events
Other events: 20 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Combination Therapy
n=20 participants at risk
Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
General disorders
Decreased energy
5.0%
1/20 • 36 weeks
General disorders
Exercise fatigue
5.0%
1/20 • 36 weeks
General disorders
Soreness
10.0%
2/20 • 36 weeks
General disorders
Increased appetite
10.0%
2/20 • 36 weeks
General disorders
Itchiness
5.0%
1/20 • 36 weeks
Skin and subcutaneous tissue disorders
Poison ivy
5.0%
1/20 • 36 weeks
Psychiatric disorders
Vivid dreams
5.0%
1/20 • 36 weeks
Blood and lymphatic system disorders
Elevated IGF-1
15.0%
3/20 • 36 weeks
Blood and lymphatic system disorders
Elevated testosterone
5.0%
1/20 • 36 weeks
Blood and lymphatic system disorders
Elevated HbA1c
5.0%
1/20 • 36 weeks
Blood and lymphatic system disorders
Elevated insulin
5.0%
1/20 • 36 weeks
Blood and lymphatic system disorders
Elevated HCT
5.0%
1/20 • 36 weeks
Blood and lymphatic system disorders
Elevated glucose
5.0%
1/20 • 36 weeks
Renal and urinary disorders
Glycosuria
5.0%
1/20 • 36 weeks
Skin and subcutaneous tissue disorders
Rash
5.0%
1/20 • 36 weeks
Nervous system disorders
Parathesia
5.0%
1/20 • 36 weeks
Gastrointestinal disorders
Nausea
5.0%
1/20 • 36 weeks
Respiratory, thoracic and mediastinal disorders
Cough
10.0%
2/20 • 36 weeks
General disorders
Edema limbs
10.0%
2/20 • 36 weeks
Injury, poisoning and procedural complications
Fall
5.0%
1/20 • 36 weeks
General disorders
Fatigue
5.0%
1/20 • 36 weeks
General disorders
Flu-like symptoms
10.0%
2/20 • 36 weeks
Nervous system disorders
Headache
5.0%
1/20 • 36 weeks
General disorders
Injection site reaction
35.0%
7/20 • 36 weeks
Psychiatric disorders
Irritability
5.0%
1/20 • 36 weeks
Musculoskeletal and connective tissue disorders
Muscle cramp
10.0%
2/20 • 36 weeks
Respiratory, thoracic and mediastinal disorders
Nasal congestion
5.0%
1/20 • 36 weeks
General disorders
Pain
10.0%
2/20 • 36 weeks
Cardiac disorders
Palpitations
5.0%
1/20 • 36 weeks
Respiratory, thoracic and mediastinal disorders
Sore throat
5.0%
1/20 • 36 weeks
Renal and urinary disorders
Urine discoloration
5.0%
1/20 • 36 weeks
Investigations
Weight gain
5.0%
1/20 • 36 weeks
General disorders
White patches on tongue
5.0%
1/20 • 36 weeks

Additional Information

Chad Heatwole, MD, MS-CI

University of Rochester Medical Center

Phone: 585-275-7867

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place