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ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT02384460
Recruitment Status : Completed
First Posted : March 10, 2015
Results First Posted : October 16, 2018
Last Update Posted : November 15, 2018
Sponsor:
Collaborator:
Amicus Therapeutics
Information provided by (Responsible Party):
Scioderm, Inc.

Tracking Information
First Submitted Date  ICMJE February 13, 2015
First Posted Date  ICMJE March 10, 2015
Results First Submitted Date  ICMJE September 19, 2018
Results First Posted Date  ICMJE October 16, 2018
Last Update Posted Date November 15, 2018
Actual Study Start Date  ICMJE March 11, 2015
Actual Primary Completion Date July 5, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 19, 2018)
  • Time To Complete Target Wound Closure Within 3 Months [ Time Frame: From baseline to Month 3 visit ]
    Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Time to target wound closure was measured from the date of the first administration of the study drug to the date of target wound closure. Participants were censored if they did not have a response within 3 months, or withdrew earlier before the confirmation of their target wound closing. This primary end point displays the mean time to complete target wound closure, analyzed using a Kaplan-Meier approach.
  • The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months [ Time Frame: From baseline to Month 3 visit ]
    Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. Participants were considered responders if they experienced complete wound closure at the Week 2 or Months 1, 2, or 3 visits. If a target wound was documented to have closed at a given visit, it was considered closed at all subsequent visits. This primary end point displays the percentage of participants from the ITT population who had complete target wound closure by the end of the study period (that is, 3 months). Analysis was performed on participants with post-baseline wound closure data.
Original Primary Outcome Measures  ICMJE
 (submitted: March 9, 2015)
Complete closure of the target wound [ Time Frame: Within 2 months ]
Complete target wound closure is defined as skin re-epithelialization without drainage. The primary efficacy analysis will compare the proportion of patients achieving this endpoint within 2 months.
Change History Complete list of historical versions of study NCT02384460 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 19, 2018)
  • Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits [ Time Frame: From baseline to Month 1 and Month 2 visits ]
    Target wounds were monitored at each study visit for complete closure, defined as skin re-epithelialization without drainage. The percentage of participants who completed target wound closure at the Month 1 and Month 2 study visits is displayed. If a target wound was documented to have closed at a given visit, it was considered closed at all subsequent visits.
  • Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit [ Time Frame: Baseline, Month 3 visit ]
    Lesional skin was defined as areas that contained any of the following: blisters, erosions, ulcerations, scabbing, bullae, or eschars, as well as areas that were weeping, sloughing, oozing, crusted, or denuded. BSAI was calculated as a percentage, ranging from 0% to 100%, of affected body surface area, recorded for each defined body region (that is, head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, then summed for all body regions.
  • Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit [ Time Frame: Baseline, Month 3 visit ]
    Total body wound burden was calculated using BSAI. A wound defined as an open area on the skin (that is, epidermal covering disrupted). BSAI was calculated as a percentage, ranging from 0% to 100%, of affected body surface area, recorded for each defined body region (that is, head/neck, upper limbs, lower limbs, trunk [includes groin]), and multiplied by the weighting factor, then summed for all body regions.
  • Change From Baseline In Itching Score At Day 7 [ Time Frame: Baseline, Day 7 ]
    Itching was assessed using the 5-point Itch Man Pruritus Assessment Tool. For participants up to 5 years of age, itching was assessed using caretaker's response and participants 6 years of age and older self-reported their itching assessments based on the following scores: 0=Comfortable, no itch; 1=itches a little, does not interfere with activity; 2=itches more, sometimes interferes with activity; 3=itches a lot, difficult to be still, concentrate; 4=itches most terribly, impossible to sit still or concentrate. Itching scores were categorized into 3 groups based on improvement; Improved or No Itching, Not Improved, and Missing. An itching score reduction from baseline greater than or equal to 1 point on the scale was classed as improved.
  • Change From Baseline In Pain Score At Day 7 [ Time Frame: Baseline, Day 7 ]
    Change in pain assessed at Day 7 compared to baseline was measured using the Face, Legs, Activity, Cry, and Consolability (FLACC) behavioral scale for participants 1 month to 3 years of age. Each of the 5 FLACC categories was scored from 0 to 2, which resulted in a total score between 0 and 10 with 0=Relaxed and comfortable, 1 to 3=Mild discomfort, 4 to 6=Moderate pain, and 7 to 10=Severe discomfort/pain. For participants 4 years of age and older, the "Wong Faces Pain Scale" was used. This scale shows a series of faces ranging from a happy face at 0, which represents "no hurt," to a crying face at 10, which represents "hurts worst". Pain scores were categorized into 3 groups based on improvement: Improved or No Pain, Not Improved, and Missing. A pain score reduction from baseline greater than or equal to 2 points on the scale was classed as improved.
Original Secondary Outcome Measures  ICMJE
 (submitted: March 9, 2015)
  • Median time to complete target wound closure [ Time Frame: Within 3 months ]
  • Change in lesional skin [ Time Frame: Month 3 ]
    Based on BSA estimates at month 3, compared to baseline
  • Change in itching using Itch Man Pruritus Assessment Tool [ Time Frame: Day 7 ]
    Assessed at Day 7 using Itch Man Pruritus Assessment Tool, compared to baseline
  • Change in pain using "FLACC scale" for patients up to 3 years of age and using "Wrong Faces Pain Scale" for patients 4 years of age and older [ Time Frame: Day 7 ]
    Assessed at Day 7, compared to baseline using the "FLACC scale" for patients up to 3 years of age and for patients 4 years of age and older the "Wong Faces Pain Scale" will be utilized.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: March 9, 2015)
  • Estimation of Total Body Wound Burden [ Time Frame: 2 weeks; months 1, 2 and 3 ]
    Assessed at 2 weeks and Months 1, 2 and 3; compared to baseline
  • Change in lesional skin [ Time Frame: week 2; months 1 and 2 ]
    based on BSA estimates, compared to baseline
  • Presence of scarring of healed target wound [ Time Frame: once target wound has healed assessed at day 7 ]
  • Change in itching and pain [ Time Frame: Days 1 to 6; week 2; months 1, 2 and 3 ]
    Assessed at Days 1 to 6, Week 2 and Months 1, 2 and 3; compared to baseline
  • Proportion of patients experiencing target wound closure [ Time Frame: Week 2; Months 1 and 3 ]
 
Descriptive Information
Brief Title  ICMJE ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
Official Title  ICMJE A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa
Brief Summary The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding Source - United States Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).
Detailed Description This was a Phase 3, multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of SD-101-6.0 cream versus placebo (SD-101-0.0) cream on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB). Epidermolysis Bullosa is a rare group of inherited disorders that typically manifest at birth as blistering and lesion formation on the skin in response to little or no apparent trauma. In this study, SD-101-6.0 cream or placebo (SD-101-0.0) cream was to be applied topically, once a day to the entire body for a period of 90 days. Participants had 1 target wound selected at baseline by the investigator. The selected target wound was required to have been present for at least 21 days. Photographic confirmation of the target wound location was collected at baseline, and the picture saved from the first visit was used to confirm location of the target wound at subsequent visits. The participant returned to the study site for Visit 2 (approximately 14 days from baseline), Visit 3 (approximately 30 days from baseline), Visit 4 (approximately 60 days from baseline), and Visit 5 (approximately 90 days from baseline) to have the target wound assessed for the level of healing. In addition, itching, pain, body surface area, target wound closure, and scarring of healed target wound were assessed at each visit. The ARANZ SilhouetteStar™ was used to measure the target wound at all visits.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Epidermolysis Bullosa
Intervention  ICMJE
  • Drug: SD-101-6.0 cream
    applied topically once a day for 90 days
    Other Name: SD-101
  • Drug: Placebo (SD-101-0.0) cream
    applied topically once a day for 90 days
    Other Name: placebo
Study Arms  ICMJE
  • Experimental: SD-101-6.0 cream
    SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days
    Intervention: Drug: SD-101-6.0 cream
  • Placebo Comparator: Placebo (SD-101-0.0) cream
    SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days
    Intervention: Drug: Placebo (SD-101-0.0) cream
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 19, 2018)
169
Original Estimated Enrollment  ICMJE
 (submitted: March 9, 2015)
130
Actual Study Completion Date  ICMJE July 5, 2017
Actual Primary Completion Date July 5, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Informed Consent form signed by the participant or participant's legal representative; if the participant was under the age of 18 but capable of providing assent, signed assent from the participant.
  • Participant (or caretaker) must have been willing to comply with all protocol requirements.
  • Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
  • Participant must have had 1 target wound (size 10 to 50 cm^2) at study entry.
  • Participants 1 month and older.
  • Target wound must have been present for at least 21 days.

Exclusion Criteria:

  • Participants who did not meet the entry criteria outlined above.
  • Selected target wound did not have clinical evidence of local infection.
  • Use of any investigational drug within the 30 days before enrollment.
  • Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.
  • Use of systemic or topical steroidal therapy within the 30 days before enrollment. (Inhaled steroids and ophthalmic drops containing steroids were allowed).
  • Use of systemic antibiotics within the 7 days before enrollment.
  • Current or former malignancy.
  • Arterial or venous disorder resulting in ulcerated lesions.
  • Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at screening and every 30 days until the final visit for female participants of childbearing potential).
  • Females of childbearing potential who were not abstinent and not practicing a medically acceptable method of contraception.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Month and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Austria,   France,   Germany,   Israel,   Italy,   Lithuania,   Netherlands,   Poland,   Serbia,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02384460
Other Study ID Numbers  ICMJE SD-005
2014-002288-14 ( EudraCT Number )
R01-005095-01 ( Other Identifier: Orphan Product Grant )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Scioderm, Inc.
Study Sponsor  ICMJE Scioderm, Inc.
Collaborators  ICMJE Amicus Therapeutics
Investigators  ICMJE
Study Director: Medical Monitor Amicus Therapeutics
PRS Account Scioderm, Inc.
Verification Date October 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP