A Study of B244 Delivered as a Topical Spray to Assess Safety in Pediatric Subjects With Atopic Dermatitis

An Open-label, Multicenter, Phase Ib Study of B244 Delivered as a Topical Spray to Assess Safety in Pediatric Subjects Aged 2 to 17 Years With Atopic Dermatitis

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03775434

Enrollment

Registered

2018-12-14

Start date

2018-12-07

Completion date

2019-06-10

Last updated

2024-08-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Atopic Dermatitis, Eczema

Brief summary

An open-label, multicenter, Phase Ib study of B244 delivered as a topical spray to assess safety in pediatric subjects aged 2 to 17 years with atopic dermatitis Condition or disease Intervention/treatment Phase Atopic Dermatitis (Eczema) Biological: B244 Phase 1b

Detailed description

This is a Phase 1b, open-label, single arm, multiple site study assessing twice daily B244 application for 28 days in pediatric subjects with mild to moderate atopic dermatitis. Number of Subjects: The study will enroll 36 subjects in 3 cohorts of 12 subjects: * Cohort 1: subjects aged 2 to 5 years. * Cohort 2: subjects aged 6 to 11 years. * Cohort 3, subjects aged 12 to 17 years. At Screening and Baseline, all subjects must have confirmed diagnosis of atopic dermatitis, as defined by the Hanifin and Rajka criteria, which involves a minimum of 10% but no more than 60% body surface area and a Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD) scale of 2 or 3. The duration of the study will be approximately 7 weeks. Subjects will attend for a Screening visit between Days -21 and -14. If all eligibility criteria and none of the exclusion criteria are met, subjects will be enrolled into the study and will be required to undergo a 14 day washout period (Days-14 to -1). Subjects will attend the study center on Day 1 and the Baseline assessments will be performed before application of the first dose. On confirmation of continued eligibility the subject and parent or guardian of the subject will be coached on how to apply medication, depending on the affected areas. They will be instructed to apply B244 twice daily (approximately 12 hours apart) for 28 days. The first dose will be applied in the clinic under the supervision of clinical staff. Details of dose administration will be recorded in the study diary provided. The subjects with their parent or guardian will return to the study center on Days 7, 14, and 21 for completion of study assessments. There will be a final study visit on Day 28, this will be defined as the end of the study for the subjects. A time window of ±2 day will be permitted for these 4 visits. There will not be a period of confinement in the study center all visits will be outpatient visits. Safety monitoring will include review of TEAEs, vital signs and physical examination. Efficacy will be assessed using EASI, vIGA-AD scale, POEM and ItchMan scores.

Interventions

DRUGExperimental: B244

B244 suspension in 30ml/bottle

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Masking description

This is a single arm open label study. Subjects will be assigned to the study treatment arm. Each participant will be scheduled to receive investigational product (IP).

Intervention model description

All subjects will receive active product

Eligibility

Sex/Gender

ALL

Age

2 Years to 17 Years

Healthy volunteers

Inclusion criteria

* Subjects are eligible to be included in the study only if all of the following criteria apply: 1. Male and female subjects 2 to 17 years of age, inclusive. 2. Confirmed diagnosis of atopic dermatitis according to the Hanifin and Rajka criteria. 3. A minimum of 10% but no more than 60% of the subjects' body surface area (see Appendix 6 for guidance) is affected by atopic dermatitis (affected is defined by physical examination findings: erythema, edema, scaling, lichenification, and excoriation; with the excoriation serving as the physical examination correlate of pruritus). 4. A vIGA-AD scale of 2 or 3 at Screening and Baseline. 5. Subject, or the parent or guardian, to provide written informed consent and authorization for protected health information disclosure. 6. Subjects must be generally in good health based on Investigator's assessment (other than atopic dermatitis). 7. Normal vital signs, or with no clinically significant vital signs that in the opinion of the Investigator, would place the subject at increased risk or would confound the objectives of the study. 8. Females must not be pregnant, as confirmed by negative urine pregnancy testing. Female subjects aged ≥11 years old, or female patients \<11 years old who have started menstruating, will have urinary pregnancy test performed at Screening and prior to the first dose with negative results in order to participate in the study. Females must either practice abstinence from heterosexual contact or use one of the highly effective contraceptive options described in the Appendix 5. 9. Male subjects of reproductive potential, must be willing to practice effective contraception during the study while receiving study treatment from Day 1 and for 7 days after the last study visit (Day 28). 10. Ability to comprehend and comply with study procedures. 11. Agree to commit to participate in the current protocol. 12. Provide written informed consent prior to any study procedure being performed.

Exclusion criteria

Subjects are excluded from the study if any of the following criteria apply:  1. Clinically significant physical or mental disorder which, in the opinion of the Investigator, would place the subject at increased risk or would confound the objectives of the study. 2. Subjects with atopic dermatitis on the face only. 3. Active cutaneous bacterial, viral or fungal infection in any treatment area at Baseline (eg, clinically infected atopic dermatitis). 4. History or presence of immunological deficiencies or diseases, organ transplant, human immunodeficiency virus (HIV), diabetes, malignancy, malignant or pre-malignant skin conditions, serious active or recurrent infection, systemic immunosuppressive regimens, clinically significant renal disease severe hepatic disorders, or other severe uncontrolled conditions (eg, drug or alcohol abuse), that are significant and/or that may pose a health risk to the subject in the study or may have an impact on the study assessments. 5. Unstable atopic dermatitis or a consistent requirement for high-potency corticosteroids (class I-III steroids). 6. Active systemic or localized infection (including infected AD). 7. Subjects unable to comply with the excluded medication/therapy restriction 8. Known hypersensitivity to the study treatment. 9. Known to have hepatitis B, hepatitis C or HIV I or II tests. Details will be recorded in medical history, a blood sample will not be collected for confirmation. 10. Female subject who is pregnant, breastfeeding, or considering pregnancy during the study. 11. Any skin condition which in the Investigator's opinion may interfere with the evaluation of atopic dermatitis. 12. Use of any investigational drugs within the previous 30 days prior to dosing or within a period of less than 5 times the drug's half-life, whichever is longer. 13. Use of any biologic within a period of 5 times its half-life. 14. Children or relatives of the Sponsor, clinical research organization, or the Study Site personnel are excluded from participating in the study.

Design outcomes

Primary

Measure	Time frame	Description
Mean Change in Body Temperature From Baseline at Day 28	Baseline to Day 28	Body temperature (°C) will be obtained. Clinical significance of body temperature will be determined at the investigator's discretion. Change from Day 28 to Baseline.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)as Assessed by CTCAE v4.0	Baseline to Day 28	Safety and tolerability endpoints will consist of all adverse events reporting from Baseline to Day 28.
Number of Participants With Clinically Significant Changes From Baseline in Physical Exam.	Baseline to Day 28	A physical exam will be conducted by a physician assessing systems (General appearance, Dermatological, Musculoskeletal, Thyroid, HEENT \[Head, eyes, ears, nose, throat\], Lymphatic, Respiratory, Gastrointestinal, Cardiovascular, Neurological, Extremities, and other). Clinical significance of the physical exam will be determined at investigator's discretion. Results for each system were assessed as Normal, Abnormal CS (clinically significant), or Abnormal NCS (not clinically significant) and shift was analyzed from Baseline to Day 28.
Mean Change in Blood Pressure From Baseline at Day 28	Baseline to Day 28	Blood pressure will be obtained (mmHg). Clinical significance of blood pressure will be determined at the investigator's discretion. Change from Day 28 to Baseline.
Mean Change in Pulse Rate From Baseline at Day 28	Baseline to Day 28	Pulse rate (beats per minute \[bpm\]) will be obtained. Clinical significance of pulse rate will be determined at the investigator's discretion. Change from Day 28 to Baseline.

Other

Measure	Time frame	Description
Changes in Area Severity Index (EASI) Score.	Baseline, Days 7, 14, 21, and 28	EASI is a validated tool used to measure the severity and extent of atopic dermatitis where clinical investigators assess the presence and severity of erythema, edema/papulation, excoriation, and lichenification (score 0-3: none=0, mild=1, moderate=2, severe=3, half-points allowed) and area of involvement (score 0-6: 0=0% involvement, 1=1-9% involvement, 2=10-29% involvement, 3=30-49% involvement, 4=50-69% involvement, 5=70-89% involvement, 6=90-100% involvement) across head and neck, trunk (including the genital area), upper extremities, and lower extremities (including the buttocks). The EASI score can range from 0.0 to 72.0 with increments of 0.1 and higher scores representing a greater severity of atopic dermatitis.
Changes in Patient Oriented Eczema Measure (POEM Total Score).	Baseline, Days 7, 14, 21, and 28	POEM is a survey that consists of 7 questions that assesses the quality of life of patient's with eczema to determine their disease severity. The 7 questions are scored out of 4 points based on frequency of occurrence during the prior week (0 = no days, 1 = 1 to 2 days, 2 = 3 to 4 days, 3 = 5 to 6 days, and 4 = all days). A higher total score indicates a higher severity of disease (0 \[clear\] to 28 \[very severe\]).
Changes in Patient Reported Outcome (Self-reported ItchMan Scale).	Baseline, Days 7, 14, 21, and 28	The Burn Man Itch Scale is used as a self report tool for subjects to indicate how itchy the area of atopic dermatitis feels at the timepoints during the study. It's reported on a scale between 0-comfortable, no itch and 4-Itches most terribly; impossible to sit still; concentrate.
Changes in the Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD).	Baseline, Days 7, 14, 21, and 28	The vIGA-AD is a physician assessment ranking of Atopic Dermatitis symptoms from 0-clear, to 4- severe.

Countries

United States

Participant flow

Participants by arm

Arm	Count
B244 B244 suspension in 30ml/bottle. B244: B244 suspension (4x10E9 cells/ml) in 30ml/bottle	28
Total	28

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Adverse Event	2
Overall Study	Withdrawal by Subject	1

Baseline characteristics

Characteristic	B244
Age, Customized	7.4 years STANDARD_DEVIATION 4.8
Baseline vIGA-AD Score 2=Mild	11 Participants
Baseline vIGA-AD Score 3=Moderate	17 Participants
BMI	20.5 kg/m^2 STANDARD_DEVIATION 7.73
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	22 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants
Height	124.0 cm STANDARD_DEVIATION 29.95
Percentage of Total Body Surface Area Affected by Atopic Dermatitis	17.98 % of total body surface area STANDARD_DEVIATION 7.581
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	0 Participants
Race (NIH/OMB) Black or African American	5 Participants
Race (NIH/OMB) More than one race	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	23 Participants
Sex: Female, Male Female	15 Participants
Sex: Female, Male Male	13 Participants
Weight	36.22 kg STANDARD_DEVIATION 30.599

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 28
other Total, other adverse events	11 / 28
serious Total, serious adverse events	0 / 28

Outcome results

Primary

Mean Change in Blood Pressure From Baseline at Day 28

Blood pressure will be obtained (mmHg). Clinical significance of blood pressure will be determined at the investigator's discretion. Change from Day 28 to Baseline.