3: A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton Syndrome

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT01428297

Collaborator

(none)

Enrollment

Locations

Arms

33.1

Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The study is divided in 3 parts, starting with the safety assessment of BPR277 ointment in Healthy volunteers (Part 1). If found to be well tolerated in Part 1, BPR277 ointment will be assessed in two different patients groups to evaluate safety and efficacy in atopic dermatitis (Part 2) and in Netherton syndrome (Part 3).

Condition or Disease	Intervention/Treatment	Phase
Healthy Volunteers Atopic Dermatitis Netherton Syndrome	Drug: BPR277 ointment (controlled application) Drug: Placebo (Vehicle) Drug: BPR277 ointment Drug: Placebo (Vehicle) Drug: BPR277 Drug: Placebo (Vehicle)	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

12 participants

Allocation:

Randomized

Intervention Model:

Single Group Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

Official Title:

A First-In-human Study to Evaluate Safety and Tolerability of Topical BPR277 in Healthy Volunteers, and Proof of Concept (PoC) Studies to Evaluate the Safety, Tolerability, and Efficacy of Topical BPR277 in Patients With Atopic Dermatitis and Netherton Syndrome

Study Start Date :

May 1, 2011

Actual Primary Completion Date :

Feb 1, 2014

Actual Study Completion Date :

Feb 1, 2014

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort A and B, BPR277 and Placebo (vehicle)	Drug: BPR277 ointment (controlled application) Drug: Placebo (Vehicle)
Experimental: Part 2 BPR277	Drug: BPR277 ointment
Placebo Comparator: Part 2 Placebo (vehicle)	Drug: Placebo (Vehicle)
Experimental: Part 3 BPR277 and Placebo (vehicle)	Drug: BPR277 Drug: Placebo (Vehicle)

Outcome Measures

Primary Outcome Measures

Systemic and local tolerability of BPR277 ointment, as measured by change in local tolerability score, number of adverse events and clinically significant changes in standard hematology, blood chemistry. [2-4 weeks]
Part 2 and 3: Patients with Atopic Dermatitis / Netherton syndrome: Change in Total Lesional Signs Score at the treated skin area [4 weeks]

Secondary Outcome Measures

Part 2 and 3: Patients with Atopic Dermatitis / Netherton syndrome: Change for each sign comprising the total lesional signs score at the treated skin area [4 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

Inclusion Criteria:

Part 1 Healthy volunteers

Healthy male and female subjects of non childbearing potential, 18 to 65 years of age inclusive and in good health

Part 2 Patients with atopic dermatitis:

Male and female subjects, 18 to 65 years of age inclusive and having passed screening examinations
Presence of atopic dermatitis confirmed by Itchy skin condition in the past 12 months (must have) Plus three or more of the following:

History of involvement of the skin creases
Personal history of asthma or hay fever
History of generally dry skin in the past year
Onset before age of 2 years
Visible flexural dermatitis

Diagnosis of at least moderate atopic dermatitis by the IGA and a minimum target area (right or left) situated on the forearm including the antecubital fossa with a corresponding baseline total lesional sign score (TLSS)

Part 3 Patients with Netherton Syndrome:

Patients with Netherton syndrome, male and female subjects, 18 to 65 years of age inclusive and having passed screening examinations
Confirmed diagnosis of Netherton syndrome (SPINK5 mutation or LEKTI deficiency in the skin).
Minimum total lesional sign score NS (TLSS-NS) of 5-9 for two selected target areas at baseline. The TLSS-NS values need to be similar between the two areas at baseline.

Exclusion Criteria:

Part 1 Healthy volunteers :

History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or history of serious allergic reaction.
Use of any prescription drugs, herbal supplements, within four (4) weeks prior to initial dosing, and/or over-the-counter (OTC) medication, dietary supplements (vitamins included) within two (2) weeks prior to initial dosing.

Part 2 Patients with atopic dermatitis:

History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or history of serious allergic reaction.
History of abnormal skin reactivity to UV light. Unusual exposure to UV light in the previous 3 weeks to study start (screening), including tanning and sun beds.
Pregnant or nursing (lactating) women.
Women of child-bearing potential must use highly effective contraception (as further defined in study protocol)
Use of topical prescription treatment for eczema within 1 week prior to initial dosing of topical corticosteroids (TCS).
Recent previous treatment with systemic treatment including phototherapy. A washout period will be required for such patients to be eligible to participate in the trial.

Part 3 Patients with Netherton Syndrome:

History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or history of serious allergic reaction.
History of abnormal skin reactivity to UV light. Unusual exposure to UV light in the previous 3 weeks to study start (screening), including tanning and sun beds.
Pregnant or nursing (lactating) women.
Women of child-bearing potential must use highly effective contraception (as further defined in study protocol)
Use of topical prescription treatment within 2 week prior to initial dosing of study drug.
Recent previous treatment with systemic treatment. A washout period will be required for such patients to be eligible to participate in the trial.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigative Site	New Haven	Connecticut	United States	06519
2	Novartis Investigative Site	Saint Louis	Missouri	United States	63104
3	Novartis Investigative Site	Fargo	North Dakota	United States	58104
4	Novartis Investigative Site	Utrecht	The Netherlands	Netherlands	3508 GA