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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.

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Epidermolysis Bullosa
Intervention Drug: SD-101-6.0 cream
Enrollment 152
Recruitment Details 152 participants with epidermolysis bullosa (EB) were enrolled between 9 June 2015 and 5 July 2017 in this open-label, multi-center extension study. All enrolled participants had previously completed Study SD-005 (NCT02384460).
Pre-assignment Details Analysis groups were defined based on prior treatment in Study SD-005: 77 participants who received placebo were allocated to the 'Placebo to Zorblisa™ (SD-101-6.0)' group and 75 participants who received SD-101-6.0 were allocated to the 'SD-101-6.0 to SD-101-6.0' group. All participants received SD-101-6.0 upon enrolling in Study SD-006.
Arm/Group Title SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0
Hide Arm/Group Description Participants who received SD-101-6.0 in Study SD-005 continued to receive SD-101-6.0 in this open label extension study. SD-101-6.0 was applied topically once a day to the entire body. Participants who received placebo in Study SD-005 received SD-101-6.0 in this open-label extension study. SD-101-6.0 was applied topically once a day to the entire body.
Period Title: Overall Study
Started 75 77
Received At Least 1 Dose Of Study Drug [1] 75 77
Completed 0 0
Not Completed 75 77
Reason Not Completed
Protocol Deviation             1             0
Study Terminated By Sponsor             36             43
Adverse Event             1             3
Withdrawal by Subject             34             29
Lost to Follow-up             3             2
[1]
Safety Population
Arm/Group Title SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0 Total
Hide Arm/Group Description Participants who received SD-101-6.0 in Study SD-005 continued to receive SD-101-6.0 in this open label extension study. SD-101-6.0 was applied topically once a day to the entire body. Participants who received placebo in Study SD-005 received SD-101-6.0 in this open-label extension study. SD-101-6.0 was applied topically once a day to the entire body. Total of all reporting groups
Overall Number of Baseline Participants 75 77 152
Hide Baseline Analysis Population Description
Intent-to-treat (ITT) population: all participants who rolled over from Study SD-005.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 75 participants 77 participants 152 participants
14.18  (13.381) 14.36  (13.599) 14.27  (13.447)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 75 participants 77 participants 152 participants
Female
28
  37.3%
45
  58.4%
73
  48.0%
Male
47
  62.7%
32
  41.6%
79
  52.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 75 participants 77 participants 152 participants
Hispanic or Latino
5
   6.7%
13
  16.9%
18
  11.8%
Not Hispanic or Latino
67
  89.3%
60
  77.9%
127
  83.6%
Unknown or Not Reported
3
   4.0%
4
   5.2%
7
   4.6%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 75 participants 77 participants 152 participants
White/Caucasian
65
  86.7%
63
  81.8%
128
  84.2%
Black or African-American
4
   5.3%
3
   3.9%
7
   4.6%
Asian
3
   4.0%
7
   9.1%
10
   6.6%
American Indian or Alaskan Native
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific
0
   0.0%
0
   0.0%
0
   0.0%
Other
1
   1.3%
1
   1.3%
2
   1.3%
Unknown
2
   2.7%
3
   3.9%
5
   3.3%
EB Type  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 75 participants 77 participants 152 participants
Simplex
9
  12.0%
7
   9.1%
16
  10.5%
Recessive Dystrophic
53
  70.7%
56
  72.7%
109
  71.7%
Junctional Non-Herlitz
13
  17.3%
14
  18.2%
27
  17.8%
1.Primary Outcome
Title Number Of Participants With Treatment Emergent Adverse Events (TEAEs)
Hide Description TEAEs were defined as adverse events that started or worsened on or after baseline visit.
Time Frame From baseline to 30 days after last application of study drug (up to a maximum of 37 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who applied/were administered the study drug at least once.
Arm/Group Title SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0
Hide Arm/Group Description:
Participants who received SD-101-6.0 in Study SD-005 continued to receive SD-101-6.0 in this open label extension study. SD-101-6.0 was applied topically once a day to the entire body.
Participants who received placebo in Study SD-005 received SD-101-6.0 in this open-label extension study. SD-101-6.0 was applied topically once a day to the entire body.
Overall Number of Participants Analyzed 75 77
Measure Type: Count of Participants
Unit of Measure: Participants
Any TEAE
51
  68.0%
58
  75.3%
Any TEAE Related To Study Drug
8
  10.7%
17
  22.1%
Any Fatal TEAE
0
   0.0%
0
   0.0%
Any Serious TEAE
11
  14.7%
14
  18.2%
Any TEAE Leading To Discontinuation
2
   2.7%
3
   3.9%
2.Secondary Outcome
Title Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30
Hide Description 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.
Time Frame Baseline, up to Month 30
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: all participants who rolled over from Study SD-005 and had data available for analysis at each specified time point.
Arm/Group Title SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0
Hide Arm/Group Description:
Participants who received SD-101-6.0 in Study SD-005 continued to receive SD-101-6.0 in this open label extension study. SD-101-6.0 was applied topically once a day to the entire body.
Participants who received placebo in Study SD-005 received SD-101-6.0 in this open-label extension study. SD-101-6.0 was applied topically once a day to the entire body.
Overall Number of Participants Analyzed 75 77
Mean (Standard Deviation)
Unit of Measure: Percentage of BSAI
Month 1 Number Analyzed 70 participants 71 participants
-0.76  (4.185) -0.54  (5.398)
Month 3 Number Analyzed 69 participants 66 participants
-1.87  (5.999) -1.21  (6.457)
Month 6 Number Analyzed 64 participants 60 participants
-1.77  (6.015) -1.35  (6.813)
Month 9 Number Analyzed 61 participants 56 participants
-2.48  (9.436) 0.31  (10.117)
Month 12 Number Analyzed 52 participants 50 participants
-3.35  (9.566) 0.44  (10.327)
Month 15 Number Analyzed 35 participants 29 participants
-1.90  (7.337) 2.15  (12.546)
Month 18 Number Analyzed 25 participants 26 participants
-2.46  (5.275) -4.24  (8.207)
Month 21 Number Analyzed 18 participants 16 participants
-3.06  (5.869) -1.58  (10.442)
Month 24 Number Analyzed 11 participants 7 participants
-1.63  (5.647) -0.64  (5.981)
Month 27 Number Analyzed 6 participants 6 participants
-1.87  (4.405) -0.16  (6.514)
Month 30 Number Analyzed 3 participants 5 participants
-2.77  (5.465) 3.37  (10.706)
3.Secondary Outcome
Title Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30
Hide Description

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.

Time Frame Baseline, up to Month 30
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population: all participants who rolled over from Study SD-005 and had data available for analysis at each specified time point.
Arm/Group Title SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0
Hide Arm/Group Description:
Participants who received SD-101-6.0 in Study SD-005 continued to receive SD-101-6.0 in this open label extension study. SD-101-6.0 was applied topically once a day to the entire body.
Participants who received placebo in Study SD-005 received SD-101-6.0 in this open-label extension study. SD-101-6.0 was applied topically once a day to the entire body.
Overall Number of Participants Analyzed 75 77
Mean (Standard Deviation)
Unit of Measure: Percentage of BSAI
Month 1 Number Analyzed 70 participants 71 participants
-1.75  (4.891) 0.07  (4.044)
Month 3 Number Analyzed 69 participants 66 participants
-1.52  (5.343) -0.80  (3.001)
Month 6 Number Analyzed 64 participants 60 participants
-1.54  (4.322) -0.48  (3.595)
Month 9 Number Analyzed 61 participants 56 participants
-1.82  (6.083) -0.42  (3.586)
Month 12 Number Analyzed 52 participants 50 participants
-1.38  (5.497) -0.13  (3.211)
Month 15 Number Analyzed 35 participants 29 participants
-0.15  (4.511) 0.26  (3.030)
Month 18 Number Analyzed 25 participants 26 participants
-1.12  (3.053) -1.31  (4.665)
Month 21 Number Analyzed 18 participants 16 participants
-1.44  (3.150) -0.28  (5.507)
Month 24 Number Analyzed 11 participants 7 participants
-0.68  (3.890) -0.01  (2.898)
Month 27 Number Analyzed 6 participants 6 participants
-0.43  (2.924) 0.31  (3.615)
Month 30 Number Analyzed 3 participants 5 participants
-1.55  (2.883) 1.69  (5.933)
Time Frame From baseline to 30 days after last application of study drug (up to a maximum of 37 months).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0
Hide Arm/Group Description Participants who received SD-101-6.0 in Study SD-005 continued to receive SD-101-6.0 in this open label extension study. SD-101-6.0 was applied topically once a day to the entire body. Participants who received placebo in Study SD-005 received SD-101-6.0 in this open-label extension study. SD-101-6.0 was applied topically once a day to the entire body.
All-Cause Mortality
SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0
Affected / at Risk (%) Affected / at Risk (%)
Total   0/75 (0.00%)   0/77 (0.00%) 
Hide Serious Adverse Events
SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0
Affected / at Risk (%) Affected / at Risk (%)
Total   11/75 (14.67%)   14/77 (18.18%) 
Blood and lymphatic system disorders     
Anaemia  1  1/75 (1.33%)  2/77 (2.60%) 
Cardiac disorders     
Pericarditis  1  1/75 (1.33%)  0/77 (0.00%) 
Congenital, familial and genetic disorders     
Congenital megaureter  1  1/75 (1.33%)  0/77 (0.00%) 
Eye disorders     
Keratitis  1  1/75 (1.33%)  0/77 (0.00%) 
Gastrointestinal disorders     
Abdominal pain  1  0/75 (0.00%)  1/77 (1.30%) 
Constipation  1  1/75 (1.33%)  1/77 (1.30%) 
Dysphagia  1  1/75 (1.33%)  0/77 (0.00%) 
Faecaloma  1  1/75 (1.33%)  0/77 (0.00%) 
Intestinal obstruction  1  0/75 (0.00%)  1/77 (1.30%) 
Oesophageal stenosis  1  1/75 (1.33%)  2/77 (2.60%) 
Immune system disorders     
Drug hypersensitivity  1  0/75 (0.00%)  1/77 (1.30%) 
Infections and infestations     
Gastroenteritis clostridial  1  0/75 (0.00%)  1/77 (1.30%) 
Implant site infection  1  1/75 (1.33%)  0/77 (0.00%) 
Infection  1  1/75 (1.33%)  0/77 (0.00%) 
Otitis media  1  0/75 (0.00%)  1/77 (1.30%) 
Skin bacterial infection  1  0/75 (0.00%)  1/77 (1.30%) 
Skin infection  1  0/75 (0.00%)  2/77 (2.60%) 
Staphylococcal skin infection  1  0/75 (0.00%)  1/77 (1.30%) 
Wound infection  1  0/75 (0.00%)  1/77 (1.30%) 
Wound infection bacterial  1  1/75 (1.33%)  0/77 (0.00%) 
Injury, poisoning and procedural complications     
Joint dislocation  1  0/75 (0.00%)  1/77 (1.30%) 
Post procedural fistula  1  0/75 (0.00%)  1/77 (1.30%) 
Procedural pain  1  0/75 (0.00%)  1/77 (1.30%) 
Procedural vomiting  1  0/75 (0.00%)  1/77 (1.30%) 
Stoma site extravasation  1  0/75 (0.00%)  1/77 (1.30%) 
Stoma site inflammation  1  0/75 (0.00%)  1/77 (1.30%) 
Investigations     
Body temperature increased  1  0/75 (0.00%)  1/77 (1.30%) 
Metabolism and nutrition disorders     
Dehydration  1  1/75 (1.33%)  1/77 (1.30%) 
Feeding intolerance  1  1/75 (1.33%)  0/77 (0.00%) 
Malnutrition  1  0/75 (0.00%)  1/77 (1.30%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Intraductal papillary-mucinous carcinoma of pancreas  1  0/75 (0.00%)  1/77 (1.30%) 
Squamous cell carcinoma  1  1/75 (1.33%)  1/77 (1.30%) 
Psychiatric disorders     
Intermittent explosive disorder  1  1/75 (1.33%)  0/77 (0.00%) 
Skin and subcutaneous tissue disorders     
Skin disorder  1  1/75 (1.33%)  0/77 (0.00%) 
1
Term from vocabulary, MedDRA (19.1)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
SD-101-6.0 to SD-101-6.0 Placebo to SD-101-6.0
Affected / at Risk (%) Affected / at Risk (%)
Total   44/75 (58.67%)   51/77 (66.23%) 
Blood and lymphatic system disorders     
Anaemia  1  5/75 (6.67%)  5/77 (6.49%) 
Lymphadenopathy  1  1/75 (1.33%)  2/77 (2.60%) 
Congenital, familial and genetic disorders     
Epidermolysis bullosa  1  3/75 (4.00%)  0/77 (0.00%) 
Gastrointestinal disorders     
Abdominal pain  1  1/75 (1.33%)  3/77 (3.90%) 
Abdominal pain upper  1  0/75 (0.00%)  2/77 (2.60%) 
Constipation  1  5/75 (6.67%)  3/77 (3.90%) 
Diarrhoea  1  2/75 (2.67%)  3/77 (3.90%) 
Gastrooesophageal reflux disease  1  3/75 (4.00%)  0/77 (0.00%) 
Haematemesis  1  0/75 (0.00%)  2/77 (2.60%) 
Oesophageal dilatation  1  2/75 (2.67%)  0/77 (0.00%) 
Oesophageal stenosis  1  3/75 (4.00%)  4/77 (5.19%) 
Toothache  1  2/75 (2.67%)  0/77 (0.00%) 
Vomiting  1  2/75 (2.67%)  3/77 (3.90%) 
General disorders     
Pain  1  2/75 (2.67%)  2/77 (2.60%) 
Pyrexia  1  5/75 (6.67%)  10/77 (12.99%) 
Systemic inflammatory response syndrome  1  0/75 (0.00%)  2/77 (2.60%) 
Infections and infestations     
Bronchitis  1  5/75 (6.67%)  1/77 (1.30%) 
Cellulitis  1  3/75 (4.00%)  0/77 (0.00%) 
Cystitis  1  2/75 (2.67%)  0/77 (0.00%) 
Eye infection  1  2/75 (2.67%)  1/77 (1.30%) 
Gastroenteritis  1  0/75 (0.00%)  3/77 (3.90%) 
Influenza  1  2/75 (2.67%)  3/77 (3.90%) 
Nasopharyngitis  1  10/75 (13.33%)  6/77 (7.79%) 
Otitis media  1  3/75 (4.00%)  0/77 (0.00%) 
Pharyngitis streptococcal  1  2/75 (2.67%)  4/77 (5.19%) 
Pyoderma  1  2/75 (2.67%)  0/77 (0.00%) 
Rhinitis  1  2/75 (2.67%)  1/77 (1.30%) 
Sinusitis  1  4/75 (5.33%)  1/77 (1.30%) 
Skin bacterial infection  1  4/75 (5.33%)  4/77 (5.19%) 
Skin infection  1  6/75 (8.00%)  12/77 (15.58%) 
Staphylococcal skin infection  1  4/75 (5.33%)  4/77 (5.19%) 
Upper respiratory tract infection  1  4/75 (5.33%)  5/77 (6.49%) 
Urinary tract infection  1  2/75 (2.67%)  1/77 (1.30%) 
Viral rash  1  0/75 (0.00%)  2/77 (2.60%) 
Viral upper respiratory tract infection  1  1/75 (1.33%)  2/77 (2.60%) 
Wound infection  1  3/75 (4.00%)  6/77 (7.79%) 
Wound infection bacterial  1  2/75 (2.67%)  5/77 (6.49%) 
Wound infection pseudomonas  1  2/75 (2.67%)  2/77 (2.60%) 
Wound infection staphylococcal  1  2/75 (2.67%)  3/77 (3.90%) 
Injury, poisoning and procedural complications     
Corneal abrasion  1  2/75 (2.67%)  2/77 (2.60%) 
Procedural pain  1  3/75 (4.00%)  0/77 (0.00%) 
Wound  1  3/75 (4.00%)  0/77 (0.00%) 
Wound complication  1  1/75 (1.33%)  2/77 (2.60%) 
Psychiatric disorders     
Anxiety  1  1/75 (1.33%)  3/77 (3.90%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  4/75 (5.33%)  3/77 (3.90%) 
Rhinorrhoea  1  2/75 (2.67%)  1/77 (1.30%) 
Skin and subcutaneous tissue disorders     
Excessive granulation tissue  1  2/75 (2.67%)  1/77 (1.30%) 
Pruritus  1  3/75 (4.00%)  6/77 (7.79%) 
Skin lesion  1  2/75 (2.67%)  2/77 (2.60%) 
1
Term from vocabulary, MedDRA (19.1)
Indicates events were collected by systematic assessment
Results are presented for all participants who received SD-101-6.0 before the early termination of the study on 4 June 2018. The maximum study duration completed by at least 1 participant was 37 months.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Patient Advocacy
Organization: Amicus Therapeutics, Inc.
Phone: +1-609-662-2000
EMail: clinicaltrials@amicusrx.com
Layout table for additonal information
Responsible Party: Scioderm, Inc.
ClinicalTrials.gov Identifier: NCT02670330    
Other Study ID Numbers: SD-006
2014-005679-96 ( EudraCT Number )
First Submitted: July 28, 2015
First Posted: February 1, 2016
Results First Submitted: July 10, 2019
Results First Posted: August 2, 2019
Last Update Posted: September 27, 2019