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

• Register: ClinicalTrials.gov
• Last refreshed on: 18 March 2013
• Main ID: NCT01428297
• Date of registration: 19/08/2011
• Primary sponsor: Novartis Pharmaceuticals
• Public title: A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton Syndrome
• Scientific 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
• Date of first enrolment: May 2011
• Target sample size: 85
• Recruitment status: Active, not recruiting
• URL: http://clinicaltrials.gov/show/NCT01428297
• Study type: Interventional
• Study design: Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Countries of recruitment

France, Germany, Netherlands, United States

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion criteria

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:

1. History of involvement of the skin creases

2. Personal history of asthma or hay fever

3. History of generally dry skin in the past year

4. Onset before age of 2 years

5. 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 Cohorts A, AA and AB (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 Cohorts A, AA and AB (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.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Atopic Dermatitis

Healthy Volunteers

Netherton Syndrome

Intervention(s)

Drug: BPR277 ointment

Drug: Placebo (Vehicle)

Primary Outcome(s)

Part 1: Change in Local Tolerability Score [Time Frame: Baseline, Week 2]

Part 1: Number of Participants with Adverse Events as a Measure of Safety [Time Frame: 2 weeks]

Part 2 and 3: (Patients with Atopic Dermatitis / Netherton syndrome) Change in Total Lesional Signs Score at the treated skin area [Time Frame: Baseline, Week 4]

Secondary Outcome(s)

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 [Time Frame: Baseline, Week 4]

Part 2 and 3: BPR277 Concentration in Plasma [Time Frame: 4 weeks]

Part 2 and 3: BPR277 Concentration in the Skin [Time Frame: 4 weeks]

Part 2 and 3: BPR277 Urinary Excretion [Time Frame: 4 weeks]

Part 2 and 3A: Change in Skin Capacitance [Time Frame: Baseline, Week 4]

Part 2 and 3A: Change in Transepidermal Water Loss [Time Frame: Baseline, Week 4]

Secondary ID(s)

2011-000917-38

CBPR277X2101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors