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

Phase 1

Completed

• Conditions: Healthy Volunteers; Atopic Dermatitis; Netherton Syndrome
• Interventions: Drug: BPR277 ointment; Drug: Placebo (Vehicle); Drug: BPR277 ointment (controlled application); Drug: BPR277

• Registration Number: NCT01428297
• Lead Sponsor: Novartis Pharmaceuticals

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2011-05
• Study First Submitted: 2011-08-19
• Study First Submitted QC: 2011-09-01
• Study First Posted: 2011-09-02
• Primary Completion: 2014-02
• Study Completion: 2014-02
• Status Verified: 2015-02
• Last Update Submitted: 2020-12-06
• Last Update Posted: 2020-12-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Part 2 BPR277	BPR277 ointment	-
Part 2 Placebo (vehicle)	Placebo (Vehicle)	-
Cohort A and B, BPR277 and Placebo (vehicle)	BPR277 ointment (controlled application)	-
Cohort A and B, BPR277 and Placebo (vehicle)	Placebo (Vehicle)	-
Part 3 BPR277 and Placebo (vehicle)	BPR277	-
Part 3 BPR277 and Placebo (vehicle)	Placebo (Vehicle)	-

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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

Trial Locations

Locations (1)

Novartis Investigative Site; 🇳🇱 Utrecht, The Netherlands, Netherlands