Status:

COMPLETED

A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics

Lead Sponsor:

PTC Therapeutics

Conditions:

Renal Impairment

Eligibility:

All Genders

18-80 years

Phase:

PHASE1

Brief Summary

This is a non-randomized, open-label, single-dose study to compare the PK of 21 desacetyl-DFZ and, if data permits, deflazacort in 8 subjects with ESRD to that of 8 healthy matched control subjects (a...

Detailed Description

This is a non-randomized, open-label, single-dose study to compare the PK of 21 desacetyl-DFZ and, if data permits, deflazacort in 8 subjects with ESRD to that of 8 healthy matched control subjects (a...

Eligibility Criteria

Inclusion

  • Continuous non-smokers or moderate smokers
  • For a female of non-childbearing potential: must have undergone a sterilization procedures or be postmenopausal with amenorrhea for at least 1 year prior to the first dose and FSH serum levels consistent with postmenopausal status
  • A non vasectomized, male subject must agree to use a condom with spermicide or abstain from sexual intercourse during the study until 90 days
  • If male, must agree not to donate sperm from dosing until 90 days Subject with ESRD on Hemodialysis
  • Adult male or female, 18 80 years of age
  • BMI ≥ 18.5 and ≤ 40.0 kg/m2 - Subject is maintained on a stable regimen of HD at least 3 months Healthy Subject
  • Healthy adult male and female subjects will be matched 1:1 to a specific subject in the ESRD cohort based upon age, BMI, and gender \[1:1\]. The following criteria should be fulfilled:
  • 18 to 80 years of age. Age must be within ± 15 years of the matched subject's age in the ESRD cohort
  • BMI ≥ 18.5 and ≤ 40.0 kg/m2. BMI must be within ± 15% of the matched subject's BMI in the ESRD cohort
  • Has a CLcr ≥ 90 mL/min

Exclusion

  • Subject is mentally or legally incapacitated or has significant emotional problems at the time of the screening visit or expected during the conduct of the study
  • History or presence of hypersensitivity or idiosyncratic reaction to the study drugs or related compounds (e.g., steroids or their formulations including lactose and corn starh)
  • History (within the last year prior to dosing) or presence of peptic ulcers
  • History or presence of:
  • Gastritis or esophagitis, diverticulitis, ulcerative colitis (if there is probability of impending perforation), abscess or pyogenic infections, or fresh intestinal anastomosis
  • Previous corticoids-induced myopathy
  • Ocular herpes simplex
  • Symptomatic cardiomyopathy at screening
  • Immunosuppression or other contraindications for corticosteroid treatment
  • History of chronic systemic fungal or viral infections
  • Galactose intolerance, Lapp lactose deficiency, or glucose-galactose malabsorption
  • Osteoporosis
  • Myasthenia gravis
  • Epilepsy
  • Idiopathic hypocalcuria
  • Seated blood pressure is less than 90/40 mmHg or greater than 170/100 mmHg
  • Seated heart rate is lower than 40 bpm or higher than 99 bpm
  • QTcF interval is \> 500 msec
  • Has received any live or live-attenuated vaccine within 30 days
  • Has received any immunosuppressive agents, coal tar, and/or radiation therapies within 30 days
  • Has received injectable corticoids in the 12 weeks prior to dosing or any oral form of corticoids in 30 days
  • Unable to refrain from or anticipates the use of:
  • Any drug known to be moderate or strong inhibitors or inducers of cytochrome P450 (CYP) 3A or P-glycoprotein (P-gp) for 14 days or 28 days, respectively
  • Any medication or substance which cannot be discontinued at least 14 days
  • Female subjects of childbearing potential
  • Female subjects who are pregnant or lactating
  • Positive results at screening for HIV, HBsAg or HCV
  • Has been on a diet incompatible with the on study diet within 28 days
  • Donation of blood or significant blood loss within 56 days
  • Plasma donation within 7 days
  • Participation in another clinical trial within 28 days Subject with ESRD
  • Is a regular user of any medication that would significantly alter glomerular filtration rate, e.g., cimetidine
  • Has presence of a renal carcinoma or acute renal disease caused by infection or drug toxicity
  • History of drug abuse within the past 2 years
  • Has a positive urine/breath alcohol or urine/serum/saliva drug testing Normal Renal Function
  • History or presence of alcoholism or drug abuse within the past 2 years
  • Positive urine drug or urine/breath alcohol results

Key Trial Info

Start Date :

December 1 2014

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

February 1 2015

Estimated Enrollment :

16 Patients enrolled

Trial Details

Trial ID

NCT02286622

Start Date

December 1 2014

End Date

February 1 2015

Last Update

August 18 2017

Active Locations (2)

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Page 1 of 1 (2 locations)

1

University of Miami Division of Clinical Pharmacology

Miami, Florida, United States, 33136

2

Orlando Clinical Research Center

Orlando, Florida, United States, 32809