Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Number of Participants Who Experienced an AE
研究概览
简要总结
The purpose of this study is to investigate the safety and tolerability of combination therapy with recombinant human growth hormone (rHGH) and testosterone in adult male patients with facioscapulohumeral muscular dystrophy (FSHD) over 24 weeks.
详细描述
This is a single-center, open-label study of daily human growth hormone (Genotropin®, 5.0 μg/kg via subcutaneous injection) and testosterone (testosterone enanthate, 140mg via intramuscular injection every two weeks) for 24 weeks in men with FSHD with a 12 week washout period. A total of 20 subjects will be enrolled at the University of Rochester Medical Center in Rochester, NY.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •A genetically confirmed diagnosis of FSHD (or clinical symptoms suggestive of FSHD with a first degree relative with genetically confirmed FSHD)
- •Hematocrit of ≤ 50%
- •Prostate-specific antigen ≤ 4.0 ng/ml (or ≤ 3.0 ng/ml if the participant has a first-degree relative with prostate cancer)
- •Fasting blood glucose <126 mg/dl
- •Able to walk continuously for six minutes (cane, walker, orthoses allowed)
- •Able to independently administer intramuscular and subcutaneous injections (or have a family member who is capable and willing to administer these injections)
排除标准
- •Obesity (BMI>35 kg/m2)
- •Cardiovascular disease (heart failure, coronary artery disease, uncontrolled hypertension, untreated hypercholesterolemia)
- •Untreated thyroid disease
- •Deep vein thrombosis
- •Untreated severe sleep apnea
- •Past pituitary disease
- •Significant musculoskeletal injury and/or pain that affects walking
- •A systolic blood pressure over 160 or a diastolic pressure over 100
- •Plans to dramatically change exercise habits
- •Liver disease
- •Renal disease
- •Cancer (other than basal cell skin cancer)
- •Plans to conceive
- •Heavy alcohol use (greater than 50g/day)
- •Current testosterone or HGH use
- •Current use of medications that interfere with the growth hormone or gonadal endocrine axis.
研究组 & 干预措施
Combination therapy
Testosterone Enanthate and Somatropin
干预措施: Testosterone Enanthate (Drug)
Combination therapy
Testosterone Enanthate and Somatropin
干预措施: Somatropin (Drug)
结局指标
主要结局
Number of Participants Who Experienced an AE
时间窗: 36 weeks
AEs were collected through patient report, interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations.
次要结局
- Mean Change in Level of Free Testosterone in the Blood(Baseline to 24 weeks)
- Mean Change in Level of Total Testosterone in the Blood(Baseline to 24 weeks)
- Mean Change in Level of IGF-1 in the Blood(Baseline to 24 weeks)
- Mean Change in Level of TSH in the Blood(Baseline to 24 weeks)
- Mean Change in Level of Luteinizing Hormone in the Blood(Baseline to 24 weeks)
- Mean Change in Level of FSH in the Blood(Baseline to 24 weeks)
- Mean Change in Total Lean Body Mass(Baseline to 24 weeks)
研究者
Chad Heatwole
Professor of Neurology
University of Rochester