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Trial Outcomes & Findings for Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome (NCT NCT00208026)

NCT ID: NCT00208026

Last Updated: 2019-08-14

Results Overview

At each scheduled visit, blood concentration of pimecrolimus were obtained. This value reflects the amount of pimecrolimus in the blood. This is measured directly from the blood and provides an estimate of the degree of absorption of the treatment medication through the skin into the blood.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Each visit up to 18 months: Study Days 1, 7, 14, 28, 56, 84, 175, 360, and 520

Results posted on

2019-08-14

Participant Flow

This was an open-label,single-arm study to investigate the safety profile of topical pimecrolimus cream. Patients meeting the criteria for diagnosis of Netherton Syndrome were enrolled over a period of 3 years, from September 2005 through March 2008. The study was conducted in my medical office within The Children's Hospital of Philadelphia.

The eligibility was for male and female patients aged 2 to 18 years.They were required to have normal laboratory values within 3 months prior to enrollment. A 4 week washout period was required for systemic steroid, tacrolimus, immunosuppressives, phototherapy, and inhibitors of Cytochrome. 2 week washout for Isoenzyme,tacrolimus and pimecrolimus.

Participant milestones

Participant milestones
Measure
Elidel (Pimecrolimus) 1% Cream
Treatment with drug/Elidel.Single arm-open-label treatment arm. A Pilot Study of the Efficacy and Safety of Pimecrolimus Cream 1% for the Treatment of Netherton Syndrome:
Overall Study
STARTED
3
Overall Study
COMPLETED
3
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Elidel (Pimecrolimus) 1% Cream
n=3 Participants
Treatment with drug/Elidel.Single arm-open-label treatment arm. A Pilot Study of the Efficacy and Safety of Pimecrolimus Cream 1% for the Treatment of Netherton Syndrome:
Age, Categorical
<=18 years
3 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Sex: Female, Male
Female
3 Participants
n=5 Participants
Sex: Female, Male
Male
0 Participants
n=5 Participants
Region of Enrollment
United States
3 participants
n=5 Participants

PRIMARY outcome

Timeframe: Each visit up to 18 months: Study Days 1, 7, 14, 28, 56, 84, 175, 360, and 520

At each scheduled visit, blood concentration of pimecrolimus were obtained. This value reflects the amount of pimecrolimus in the blood. This is measured directly from the blood and provides an estimate of the degree of absorption of the treatment medication through the skin into the blood.

Outcome measures

Outcome measures
Measure
Patient 1
n=1 Participants
Treatment with drug/Elidel. Single arm-open-label treatment arm. A Pilot Study of the Efficacy and Safety of Pimecrolimus Cream 1% for the Treatment of Netherton Syndrome: Pimecrolimus 1% Cream: Open label single arm
Patient 2
n=1 Participants
Treatment with drug/Elidel. Single arm-open-label treatment arm. A Pilot Study of the Efficacy and Safety of Pimecrolimus Cream 1% for the Treatment of Netherton Syndrome: Pimecrolimus 1% Cream: Open label single arm
Patient 3
n=1 Participants
Treatment with drug/Elidel. Single arm-open-label treatment arm. A Pilot Study of the Efficacy and Safety of Pimecrolimus Cream 1% for the Treatment of Netherton Syndrome: Pimecrolimus 1% Cream: Open label single arm
Blood Pimecrolimus Levels
Day 1
0 ng/mL
0 ng/mL
0.312 ng/mL
Blood Pimecrolimus Levels
Day 7
1.260 ng/mL
0.625 ng/mL
3.630 ng/mL
Blood Pimecrolimus Levels
Day 14
0.821 ng/mL
0.421 ng/mL
3.930 ng/mL
Blood Pimecrolimus Levels
Day 28
0.592 ng/mL
0.311 ng/mL
7.080 ng/mL
Blood Pimecrolimus Levels
Day 56
0.743 ng/mL
0.182 ng/mL
3.920 ng/mL
Blood Pimecrolimus Levels
Day 84
0.390 ng/mL
0.368 ng/mL
5.010 ng/mL
Blood Pimecrolimus Levels
Day 175
0.470 ng/mL
0.484 ng/mL
2.060 ng/mL
Blood Pimecrolimus Levels
Day 360
0.388 ng/mL
0.247 ng/mL
4.280 ng/mL
Blood Pimecrolimus Levels
Day 520
0.224 ng/mL
0.170 ng/mL
6.140 ng/mL

Adverse Events

Elidel (Pimecrolimus) 1% Cream

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

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Albert C. Yan, M.D.

The Children's Hospital of Philadelphia

Phone: (215) 590-2158

Results disclosure agreements

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