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Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02670330
Recruitment Status : Terminated (The Sponsor voluntarily recalled SD-101 and terminated the study due to GMP deficiencies identified during an FDA inspection at the site of the manufacturer.)
First Posted : February 1, 2016
Results First Posted : August 2, 2019
Last Update Posted : September 27, 2019
Sponsor:
Collaborator:
Amicus Therapeutics
Information provided by (Responsible Party):
Scioderm, Inc.

Tracking Information
First Submitted Date  ICMJE July 28, 2015
First Posted Date  ICMJE February 1, 2016
Results First Submitted Date  ICMJE July 10, 2019
Results First Posted Date  ICMJE August 2, 2019
Last Update Posted Date September 27, 2019
Actual Study Start Date  ICMJE June 9, 2015
Actual Primary Completion Date September 3, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 10, 2019)
Number Of Participants With Treatment Emergent Adverse Events (TEAEs) [ Time Frame: From baseline to 30 days after last application of study drug (up to a maximum of 37 months) ]
TEAEs were defined as adverse events that started or worsened on or after baseline visit.
Original Primary Outcome Measures  ICMJE
 (submitted: January 27, 2016)
Demonstrate continued long term safety of Zorblisa assessed via monitoring of local tolerability at the application sites, occurrence of adverse events and physical examinations. Results will be summarized [ Time Frame: up to 630 days ]
The primary objective is to demonstrate the longterm safety of ZORBLISA in patients, with Simplex, Recessive Dystrophic, and Junctional non-Herlitz Epidermolysis Bullosa. Results will be summarized using descriptive statistics. Summary statistics to be used include proportion of patients with complete closure of their target lesion at each visit, mean and standard deviation of BSAI compared with baseline at each visit, and incidence rates of specific adverse events. Additional evaluations may be made to compare responses of patients in this extension study with their responses in SD-005. For those patients randomized to SD-101-6.0 in SD-005, the use of summary statistics as described in the previous paragraph will provide an understanding of the long term effect of SD-101-6.0 in these patients. For those patients randomized to the control arm in SD-005, summary statistics will be used to compare their response to SD-101-6.0 in SD-006 with their response to control in SD-005.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 10, 2019)
  • Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30 [ Time Frame: Baseline, up to Month 30 ]
    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. The percentage, ranging from 0% to 100%, of affected body surface area (BSA) was 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 to calculate the BSAI that would range from 0% to 100%. The BSA for lesional skin was to be assessed by the same study physician on each visit for a particular participant. The mean change from baseline in BSAI was assessed every 3 months. Only participants with data available for analysis at each time point are presented.
  • Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30 [ Time Frame: Baseline, up to Month 30 ]
    A wound was defined as an open area on the skin (that is, epidermal covering disrupted). Total body wound burden was calculated using BSAI; the percentage, ranging from 0% to 100%, of affected BSA was 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 to calculate the BSAI that would range from 0% to 100%. The BSAI for total body wound burden was to be assessed by the same study physician at each visit for a particular participant. The mean change from baseline in total body wound burden was assessed every 3 months. Only participants with data available for analysis at each time point are presented.
Original Secondary Outcome Measures  ICMJE
 (submitted: January 27, 2016)
Changes in Body Surface Area (BSA) of lesional skin and wound burden using BSAI [ Time Frame: Months 1, 3, 6, 9 12,15,18 and 21 ]
The secondary objectives are to assess the efficacy of ZORBLISA in terms of the change in Body Surface Area (BSA) of lesional skin and wound burden; as well as closure of unhealed target wounds from the SD-005 study. Change in lesional skin based on BSA estimates compared to Baseline will be measured using the Body Surface Area Index (BSAI). Change in total body wound coverage based on BSA estimates compared to Baseline will be measured using the BSAI.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
Official Title  ICMJE An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisa™ (SD-101-6.0) in Patients With Epidermolysis Bullosa
Brief Summary The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
Detailed Description

This was an open label, multi-center extension study to assess the long-term safety of topically applied SD-101-6.0 in participants with simplex, recessive dystrophic, and junctional non-Herlitz EB. SD-101-6.0 was applied topically once a day to the entire body. The planned duration of treatment with SD-101-6.0 for Study SD-006 was up to 48 months, with a safety follow-up period of 30 days; however, the study was terminated early by the sponsor. The maximum study duration completed by at least 1 participant, treatment and safety follow-up, was 37 months.

Participants who successfully completed Study SD-005 had the option to rollover into Study SD-006. The screening/baseline visit (Visit 1) occurred at Visit 5 (approximately 90 days from baseline) of Study SD-005. The Body Surface Area (BSA) assessments of lesional skin and wound burden performed at Visit 5 (approximately 90 days from baseline) for Study SD-005 were utilized as the baseline assessments for Study SD-006. Participants returned for follow-up visits at Month 1 then every 3 months.

At each visit, assessments included BSA of lesional skin and wound burden. For target wounds that are not closed by the end of Study SD-005, the ARANZ picture and calculation of target wound area at the final visit for Study SD-005 was used as the baseline area size of the target wound for Study SD-006. These unhealed target wounds from Study SD-005 were assessed via ARANZ SilhouetteStar™ at each subsequent scheduled visit until the target wound was documented as closed. Closed wounds were assessed for scarring.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Epidermolysis Bullosa
Intervention  ICMJE Drug: SD-101-6.0 cream
SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-6.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 6% and other excipients.
Other Names:
  • Zorblisa
  • SD-101
Study Arms  ICMJE Experimental: Experimental: SD-101-6.0 cream
All participants (or their caregivers) applied SD-101-6.0 cream topically once a day to the entire body for a period of up to 36 months.
Intervention: Drug: SD-101-6.0 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 Terminated
Actual Enrollment  ICMJE
 (submitted: September 28, 2018)
152
Original Estimated Enrollment  ICMJE
 (submitted: January 27, 2016)
130
Actual Study Completion Date  ICMJE September 3, 2018
Actual Primary Completion Date September 3, 2018   (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.
  • Participants who completed the SD-005 study (on study drug at Visit 5, approximately 90 days from baseline).

Exclusion Criteria:

  • Participants who did not meet the entry criteria outlined above.
  • Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at the final visit for Study SD-005 for female participants of childbearing potential and repeated at screening/baseline visit of Study SD-006 if these visits did not occur on the same day).
  • Female participants of childbearing potential who were not abstinent or 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,   Lithuania,   Netherlands,   Poland,   Serbia,   Spain,   United Kingdom,   United States
Removed Location Countries Italy
 
Administrative Information
NCT Number  ICMJE NCT02670330
Other Study ID Numbers  ICMJE SD-006
2014-005679-96 ( EudraCT Number )
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 September 2019

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