A Trial Evaluating Safety, Tolerability and Pharmacokinetics of Subcutaneous Single Doses of ACP-015 in Healthy Adult Male Subjects.
- Conditions
- Skeletal Dysplasia in ChildrenAchondroplasiaThanatophoric DysplasiaHypochondroplasiaCampomelic DysplasiaOsteogenesis ImperfectaAchondrogenesisMetabolic and Endocrine - Other endocrine disordersMusculoskeletal - Other muscular and skeletal disordersHuman Genetics and Inherited Disorders - Other human genetics and inherited disorders
- Registration Number
- ACTRN12618001157268
- Lead Sponsor
- Ascendis Pharma Growth Disorders A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Male
- Target Recruitment
- 50
Able to give Informed consent
- Able to comply with study protocol per investigator judgement
- Healthy adult males aged 25-60 years at time of Screening
- Attainment of adult height (defined as subject’s confirmation of no change in standing height within 12 months of screening)
- BMI 18 – 27 kg/m2
- Body weight <100 kg
- In good general health as determined by medical history, physical exams and laboratory evaluation at time of screening
- Screening chemistry and hematology laboratory results in the normal range of the reference laboratory (or considered not clinically significant by the investigator)
- Subject must agree to not father a baby while on this study and for 90 days following the end of the study.. Males with female partners of child bearing potential must agree to use a barrier method of contraception (ie, condom) for the entire study period and for 90 days following the end of the study. Female partners must use a highly effective form of contraception (including oral, injectable or implantable contraception, or IUD) for the entire study period and for 90 days post end of the study.
- Agreement to refrain from donating sperm during clinical trial participation and for 90 days following the end of the study
1. Participation in any investigational medication or device trials within 90 days or 5 half-lives (whichever is longer) prior to screening (and during enrollment in current trial)
2. Prior participation in investigation trial (at any time) involving use of vosoritide
3. History or presence of malignant disease, other than basal cell epithelioma/carcinoma or completely resected squamous skin cancer with no recurrence for 12 months
4. Any history of significant bone disorders including (but not limited to) osteoporosis, hypo- or hyperparathyroidism, or inherited skeletal dysplasias
5. Baseline systolic blood pressure less than 90 mm Hg
6. Past history of, or presence of orthostatic hypotension defined as a decrease of more than or equal to 20 mm Hg systolic blood pressure or more than 20 bpm increase in heart rate at 3 minutes of standing), or with symptoms of lightheadedness, dizziness, or fainting upon standing at screening. the more than 20bpm increase in heart rate upon standing must result in a heart rate of more than 120 bpm or be considered clinically significant by the investigator.
7. Subject has electrographically significant abnormalities that might interfere with ECG analysis including evidence of a previous MI, LVH, flat T waves (particularly in the inferior leads) or more than minor non- specific ST-T wave changes or:
- QRS more than 110 msec
- QT interval corrected using Fridericia’s formula (QTcF) more than 440 msec (men and women)
- PR interval more than 220 msec
- Heart rate less than 50 BPM or more than 90 BPM
- Complete right bundle branch block or left bundle branch block.
8. Subject has a history of, disease or dysfunction of the pulmonary, cardiovascular, endocrine, hematologic, neurological, immune, gastrointestinal, genitourinary, or other body system, that is clinically significant in the opinion of the Investigator, including
- Anemia
- Pathologic factures
- Diabetes mellitus, Type I or II
- History of drug abuse (including alcoholism) or positive drug screen during screening
- History of cardiac or valvular disease (including hypertension or hypotension)
9. Acute illness that can affect hydration or volume status [such as that associated with decreased nutritional intake or increased volume loss (such as diarrhea)]
10. Known hypersensitivity to the components of the trial medication (trehalose, tris(hydroxymethyl)aminomethane, succinate and PEG)
11. Poor peripheral venous access
12. Planned procedure or surgery during the course of the study
13. Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule (including non-compliance)
14. Subject uses any prescription drug or over-the-counter medication, including herbals or routine vitamins or minerals, within 7 days prior to check-in (or 14 days if the drug is a potential inducer or inhibitor of cytochrome P450 [CYP] 3A4 or P-glycoprotein [P gp] [eg, St. John’s Wort, rifampin, cyclosporine, or ritonavir]) or 5 half-lives (whichever is longer) or subject requires continued use of a prescription drug or over-the-counter medication during study participation, with the exception of over-the-counter analgesics (eg, occasional [less than 2 times/week] acetaminophen, ibuprofen, naproxen]
15. Subject is unwilling to refrain from strenuous exercise from 7 days prior to check-in through completion of the study
16. Subject is unwilling to abstain from ingestion of
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method