None listed
Conditions
Brief summary
KER-050 is a muscle anabolic agent proposed to increase muscle mass and strength and is in development for the treatment of Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Duchenne muscular dystrophy is the most common form of muscular dystrophy and is estimated to affect 1 in every 3500 males globally. FSHD is one of the most prevalent adult forms of muscular dystrophy and can also affect children. The study hypothesis is that Ker-050 is safe to administer to participants and provides an indication that has an effect on the growth of adipose tissue and bone.
Interventions
Investigational product: KER-050 will be administered subcutaneously. Intervention adherence will be assessed by study monitors, this will be done by audit of nurses notes and pharmacy logs.
The study contains two parts.
1. Single ascending dose study (SAD), 4 cohorts of 10 participants randomised 4 (study drug): 1 (placebo).
Cohort 1: 0.05mg/kg single subcutaneous on day 1
Cohort 2: 0.5 mg/kg single subcutaneous on day 1
Cohort 3: 1.5mg/kg single subcutaneous on day 1
Cohort 4: 4.5 mg/kg sin
Investigational product: KER-050 will be administered subcutaneously. Intervention adherence will be assessed by study monitors, this will be done by audit of nurses notes and pharmacy logs. The study contains two parts. 1. Single ascending dose study (SAD), 4 cohorts of 10 participants randomised 4 (study drug): 1 (placebo). Cohort 1: 0.05mg/kg single subcutaneous on day 1 Cohort 2: 0.5 mg/kg single subcutaneous on day 1 Cohort 3: 1.5mg/kg single subcutaneous on day 1 Cohort 4: 4.5 mg/kg single subcutaneous on day 1 2. Multiple ascending dose study (MAD) 4 cohorts of 10 participants randomised 4 (study drug): 1 (placebo). Cohort 1: TBD* mg/kg subcutaneous on day 1, 15* and 29 Cohort 2: TBD* mg/kg subcutaneous on day 1, 15* and 29 Cohort 3: TBD* mg/kg subcutaneous on day 1, 15* and 29 Cohort 4: TBD* mg/kg subcutaneous on day 1, 15* and 29 (* specific dose levels and frequency to be confirmed following review of SAD data)
Sponsors
Keros Therapeutics Australia Pty Ltd
Study design
Allocation
Randomised controlled trial
Intervention model
Other
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
45 Years to 75 Years
Healthy volunteers
Yes
Inclusion criteria
1. Postmenopausal* female, aged 45 to 75 years (inclusive) at screening. *NOTE: Postmenopausal is defined as: • 12 months of spontaneous amenorrhea, OR 6 months of spontaneous amenorrhea with serum FSH levels greater than 40 IU/L; OR • 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy; 2. Non-smoker or social smoker who agrees to smoke 8 or less cigarettes per week or is willing to abstain from smoking/nicotine products during the study; 3. Weight range within 50 kg to 110 kg (inclusive); 4. In good health as determined by review of medical history, physical examination, vital signs, oxygen saturation, clinical laboratory tests, 12-lead ECG, and any abnormal findings that are assessed as not clinically significant by the Investigator.
Exclusion criteria
1. Clinically significant (as determined by the Investigator) cardiac, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease; 2. History of or current malignancy (excluding basal cell carcinoma that has been resected with no evidence of metastatic disease for 3 years); 3. Chronic stable diseases including frequent migraines, type 2 diabetes, hypertension, hyperthyroid disorder, hypothyroid disorder, gastroesophageal reflux disease, or mild depression/anxiety; 4. Current opportunistic infection (eg, invasive candidiasis or pneumocystis pneumonia); 5. Serious local infection (eg, cellulitis, abscess) or systemic infection (eg, septicemia) within the 3 months prior to screening; 6. History of severe allergic or anaphylactic reactions; 7. Surgery within 3 months prior to screening (other than minor cosmetic surgery or minor dental procedures); 8. Fever (body temperature greater than 38°C) or symptomatic viral or bacterial infection within 2 weeks prior to screening that has not resolved prior to dosing; 9. Clinically relevant/significant laboratory findings (up to 2 repeats permitted) at screening including, but not limited to: • Alanine transaminase and aspartate transaminase greater than or equal to 1.2 times the upper limit of normal (ULN), isolated and mainly unconjugated hyperbilirubinemia consistent with Gilbert's should not be excluded; • Creatinine outside normal laboratory range; • Serum creatine kinase greater than 1.5 times the ULN; 10. Donated blood (1 unit or more) within 1 month prior to dosing or plans to donate blood during the study; 11. Recent hormone replacement therapy, within 3 months prior to dosing or plans to begin hormone replacement therapy at any time during the study. Estrogen replacement is permitted; 12. Received systemic glucocorticoid therapy for more than 1 month within 6 months before screening; 13. Changes in medications that may affect muscle function (e.g. statins, beta-blockers, etc.) within 3 months prior to dosing; 14. Positive screen for alcohol and/or potential drugs of abuse (cannabis and metabolites, cocaine and metabolites, amphetamines, barbiturates, benzodiazepines and/or opioids) by urine drug screen at screening and Day -1. Up to 1 repeat permitted; 15. Treatment with another investigational drug, investigational device, or approved therapy for investigational use within 1 month or 7 half-lives prior to dosing;
Outcome results
None listed