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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.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Epidermolysis Bullosa
Interventions Drug: SD-101-6.0 cream
Drug: Placebo (SD-101-0.0) cream
Enrollment 169
Recruitment Details Of 210 participants screened for this study, 169 participants were randomly assigned, on a 1:1 basis, to treatment with SD-101-6.0 or placebo at 31 study centers in 13 countries. The first participant was enrolled on 11 March 2015 and the last participant completed the study on 05 July 2017.
Pre-assignment Details Participants had to be >1 month of age with a diagnosis of simplex, recessive dystrophic, or junctional non-Herlitz Epidermolysis Bullosa (EB) and a target wound with a surface area of 10 to 50 cm^2 and ≥21 days old to be considered for participation. Participants who did not meet all inclusion/exclusion criteria were eligible for rescreening.
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days. Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days.
Period Title: Overall Study
Started 82 87
Received at Least 1 Dose of Study Drug 82 87
Completed 75 80
Not Completed 7 7
Reason Not Completed
Protocol Violation             0             1
Returned to previous therapeutic regimen             0             1
Adverse Event             5             2
Withdrawal by Subject             0             3
Elective medical treatment             1             0
Non-compliance             1             0
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream Total
Hide Arm/Group Description SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days. Placebo (SD-101-0.0) cream applied topically, once a day to the entire body for a period of 90 days. Total of all reporting groups
Overall Number of Baseline Participants 82 87 169
Hide Baseline Analysis Population Description
The Intent-to-treat (ITT) population included all randomized participants.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 82 participants 87 participants 169 participants
13.8  (13.15) 13.9  (13.12) 13.9  (13.09)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 82 participants 87 participants 169 participants
Female
33
  40.2%
48
  55.2%
81
  47.9%
Male
49
  59.8%
39
  44.8%
88
  52.1%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 82 participants 87 participants 169 participants
White/Caucasian
69
  84.1%
72
  82.8%
141
  83.4%
Black/African American
5
   6.1%
3
   3.4%
8
   4.7%
Asian
4
   4.9%
8
   9.2%
12
   7.1%
Other
1
   1.2%
1
   1.1%
2
   1.2%
Not Reported
3
   3.7%
3
   3.4%
6
   3.6%
EB type  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 82 participants 87 participants 169 participants
Simplex
10
  12.2%
8
   9.2%
18
  10.7%
Recessive dystrophic
57
  69.5%
62
  71.3%
119
  70.4%
Junctional non-Herlitz
15
  18.3%
17
  19.5%
32
  18.9%
1.Primary Outcome
Title Time To Complete Target Wound Closure Within 3 Months
Hide Description 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.
Time Frame From baseline to Month 3 visit
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with post-baseline wound closure data and whose target wound had closed within 3 months.
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description:
Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days.
Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days.
Overall Number of Participants Analyzed 39 45
Mean (Standard Deviation)
Unit of Measure: days
41.6  (25.50) 53.6  (28.59)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection SD-101-6.0 Cream, Placebo (SD-101-0.0) Cream
Comments Cox proportional hazards model compares treatment groups with baseline target wound size, target wound age, and EB type as covariates.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.985
Comments p-value is for Type 3 chi-square test for comparison between treatments.
Method Cox Model Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.004
Confidence Interval (2-Sided) 95%
0.651 to 1.549
Estimation Comments [Not Specified]
2.Primary Outcome
Title The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months
Hide Description 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.
Time Frame From baseline to Month 3 visit
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with post-baseline wound closure data and whose target wound had closed within 3 months.
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description:
Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days.
Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days.
Overall Number of Participants Analyzed 79 84
Measure Type: Number
Unit of Measure: percentage of participants
49.4 53.6
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection SD-101-6.0 Cream, Placebo (SD-101-0.0) Cream
Comments Comparison between treatment groups of complete closure of target wound within 3 months was performed using a logistic regression model with multiple imputation with EB type, baseline target wound size, and baseline target wound age as covariates.
Type of Statistical Test Other
Comments Multiple imputation was implemented by 2 steps. The first step used Markov Chain Monte Carlo (MCMC) to get monotonic missing data pattern. In the second step, a logistic regression model was used, with the following covariates included in the imputation model: treatment, EB type, baseline target wound size, target wound age, and non-missing data from earlier time points. The seed number was 010005 and the number of imputations was 5.
Statistical Test of Hypothesis P-Value 0.39
Comments p-value is from the logistic regression model for treatment comparison.
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.733
Confidence Interval (2-Sided) 95%
0.365 to 1.474
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits
Hide Description 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.
Time Frame From baseline to Month 1 and Month 2 visits
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on participants from the ITT population with post-baseline wound closure data.
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description:
Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days.
Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days.
Overall Number of Participants Analyzed 79 84
Measure Type: Number
Unit of Measure: percentage of participants
Month 1 31.6 22.6
Month 2 43.0 42.9
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection SD-101-6.0 Cream, Placebo (SD-101-0.0) Cream
Comments Logistic Regression Analysis at Month 1 visit. Comparison between treatment groups of complete closure of target wound within 1 month was performed using a logistic regression model with multiple imputation with EB type, baseline target wound size, and baseline target wound age as covariates.
Type of Statistical Test Other
Comments Multiple imputation was implemented by 2 steps. The first step used MCMC to get the monotonic missing data pattern. In the second step, a logistic regression model was used, with the following covariates included in the imputation method: treatment, EB type, baseline target wound size, target wound age, and non-missing data from earlier time points. The seed number was 01005 and the number of imputations was 5.
Statistical Test of Hypothesis P-Value 0.212
Comments p-value is from the logistic regression model for treatment comparison.
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 1.633
Confidence Interval (2-Sided) 95%
0.758 to 3.517
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection SD-101-6.0 Cream, Placebo (SD-101-0.0) Cream
Comments Logistic Regression Analysis at Month 2 visit. Comparison between treatment groups of complete closure of target wound within 2 months was performed using a logistic regression model with multiple imputation with EB type, baseline target wound size, and baseline target wound age as covariates.
Type of Statistical Test Other
Comments Multiple imputation was implemented by 2 steps. The first step used MCMC to get monotonic missing data pattern. In the second step, a logistic regression model was used, with the following covariates included in the imputation model: treatment, EB type, baseline target wound size, target wound age, and non-missing data from earlier time points. The seed number was 01005 and the number of imputations was 5.
Statistical Test of Hypothesis P-Value 0.802
Comments p-value is from the logistic regression model for treatment comparison.
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.891
Confidence Interval (2-Sided) 95%
0.436 to 1.821
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 3 Visit
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. 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.
Time Frame Baseline, Month 3 visit
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on the ITT population. The ITT population included all randomized participants.
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description:
Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days.
Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days.
Overall Number of Participants Analyzed 75 78
Least Squares Mean (Standard Error)
Unit of Measure: Percentage change in BSAI
-4.637  (1.404) -5.319  (1.354)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection SD-101-6.0 Cream, Placebo (SD-101-0.0) Cream
Comments Mixed Model Repeated Measures (MMRM) Analysis. The MMRM approach (using restricted maximum likelihood [REML] estimation) was used on each multiply-imputed data set. The model included treatment, baseline BSAI of lesional skin, EB type, visit, and visit-treatment interaction as the fixed effects.
Type of Statistical Test Other
Comments Multiple imputation was used by 2 steps. The first step used MCMC to get monotonic missing data pattern. In the second step, a linear regression model was used, with the following covariates included in the imputation model: treatment, EB type, baseline BSAI of lesional skin, and non-missing data from earlier time points. The seed number was 01005 and the number of imputations was 5.
Statistical Test of Hypothesis P-Value 0.706
Comments The p-value is calculated based on the hypothesis testing for the difference of least-squares (LS)-means between treatment and placebo.
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter LS means difference
Estimated Value 0.682
Confidence Interval (2-Sided) 95%
-2.873 to 4.238
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Change From Baseline In BSAI Of Total Body Wound Burden At Month 3 Visit
Hide Description 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.
Time Frame Baseline, Month 3 visit
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on the ITT population. The ITT population included all randomized participants.
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description:
Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days.
Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days.
Overall Number of Participants Analyzed 75 79
Least Squares Mean (Standard Error)
Unit of Measure: Percentage change in BSAI
-3.050  (0.816) -2.922  (0.813)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection SD-101-6.0 Cream, Placebo (SD-101-0.0) Cream
Comments MMRM Analysis. The MMRM approach (using REML estimation) was used on each multiply-imputed data set. The model included treatment, baseline BSAI of lesional skin, EB type, visit, and visit-treatment interaction as the fixed effects.
Type of Statistical Test Other
Comments Multiple imputation was used by 2 steps. The first step used MCMC to get monotonic missing data pattern. In the second step, a linear regression model was used, with the following covariates included in the imputation model: treatment, EB type, baseline BSAI of lesional skin, and non-missing data from earlier time points. The seed number was 01005 and the number of imputations was 5.
Statistical Test of Hypothesis P-Value 0.9
Comments The p-value is calculated based on the hypothesis testing for the difference of LS-means between treatment and placebo.
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter LS means difference
Estimated Value 0.128
Confidence Interval (2-Sided) 95%
-2.116 to 1.861
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Change From Baseline In Itching Score At Day 7
Hide Description 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.
Time Frame Baseline, Day 7
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on the ITT population. The ITT population included all randomized participants.
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description:
Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days.
Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days.
Overall Number of Participants Analyzed 77 79
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.5  (1.31) -0.3  (1.24)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection SD-101-6.0 Cream, Placebo (SD-101-0.0) Cream
Comments The proportion of participants experiencing improvement in itching versus non-improvement (including missing) was compared between the 2 treatment groups for Day 7 using the logistic regression model with baseline itching score, and EB type as covariates.
Type of Statistical Test Other
Comments Pre-specified.
Statistical Test of Hypothesis P-Value 0.262
Comments p-value is from the logistic regression model for treatment comparison.
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 1.445
Confidence Interval (2-Sided) 95%
0.759 to 2.752
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Change From Baseline In Pain Score At Day 7
Hide Description 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.
Time Frame Baseline, Day 7
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on the ITT population. The ITT population included all randomized participants.
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description:
Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days.
Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days.
Overall Number of Participants Analyzed 77 80
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.3  (2.57) -0.6  (3.07)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection SD-101-6.0 Cream, Placebo (SD-101-0.0) Cream
Comments The proportion of participants experiencing improvement in pain versus non-improvement (including missing) was compared between the 2 treatment groups for Day 7 using the logistic regression model with baseline pain score and EB type as covariates.
Type of Statistical Test Other
Comments Pre-specified.
Statistical Test of Hypothesis P-Value 0.098
Comments p-value is from the logistic regression model for treatment comparison.
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.596
Confidence Interval (2-Sided) 95%
0.323 to 1.1
Estimation Comments [Not Specified]
Time Frame From baseline up to 3 months.
Adverse Event Reporting Description Adverse events (AEs) were defined as treatment-emergent AEs if the AE occurred on or after the first date of application of study drug.
 
Arm/Group Title SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Hide Arm/Group Description SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days. Placebo (SD-101-0.0) cream applied topically, once a day to the entire body for a period of 90 days.
All-Cause Mortality
SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Affected / at Risk (%) Affected / at Risk (%)
Total   0/82 (0.00%)   1/87 (1.15%) 
Show Serious Adverse Events Hide Serious Adverse Events
SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Affected / at Risk (%) Affected / at Risk (%)
Total   4/82 (4.88%)   8/87 (9.20%) 
Blood and lymphatic system disorders     
Anemia  1  0/82 (0.00%)  1/87 (1.15%) 
Cardiac disorders     
Cardiopulmonary failure  1  0/82 (0.00%)  1/87 (1.15%) 
Gastrointestinal disorders     
Dysphagia  1  0/82 (0.00%)  1/87 (1.15%) 
Gastritis  1  0/82 (0.00%)  1/87 (1.15%) 
Oesophageal stenosis  1  0/82 (0.00%)  1/87 (1.15%) 
General disorders     
Pyrexia  1  2/82 (2.44%)  0/87 (0.00%) 
Infections and infestations     
Influenza  1  0/82 (0.00%)  2/87 (2.30%) 
Cystitis  1  0/82 (0.00%)  1/87 (1.15%) 
Gastroenteritis  1  0/82 (0.00%)  1/87 (1.15%) 
Staphylococcal skin infection  1  0/82 (0.00%)  1/87 (1.15%) 
Injury, poisoning and procedural complications     
Wound  1  1/82 (1.22%)  0/87 (0.00%) 
Metabolism and nutrition disorders     
Dehydration  1  0/82 (0.00%)  1/87 (1.15%) 
Psychiatric disorders     
Aggression  1  1/82 (1.22%)  0/87 (0.00%) 
Renal and urinary disorders     
Acute kidney injury  1  0/82 (0.00%)  1/87 (1.15%) 
Respiratory, thoracic and mediastinal disorders     
Acute respiratory failure  1  0/82 (0.00%)  1/87 (1.15%) 
Vascular disorders     
Circulatory collapse  1  0/82 (0.00%)  1/87 (1.15%) 
1
Term from vocabulary, MedDRA (19.1)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
SD-101-6.0 Cream Placebo (SD-101-0.0) Cream
Affected / at Risk (%) Affected / at Risk (%)
Total   49/82 (59.76%)   46/87 (52.87%) 
Blood and lymphatic system disorders     
Anemia  1  3/82 (3.66%)  2/87 (2.30%) 
Gastrointestinal disorders     
Vomiting  1  2/82 (2.44%)  2/87 (2.30%) 
General disorders     
Pyrexia  1  5/82 (6.10%)  9/87 (10.34%) 
Pain  1  1/82 (1.22%)  4/87 (4.60%) 
Infections and infestations     
Nasopharyngitis  1  11/82 (13.41%)  3/87 (3.45%) 
Upper respiratory tract infection  1  4/82 (4.88%)  9/87 (10.34%) 
Skin infection  1  3/82 (3.66%)  9/87 (10.34%) 
Wound infection  1  6/82 (7.32%)  5/87 (5.75%) 
Staphyloccocal skin infection  1  1/82 (1.22%)  6/87 (6.90%) 
Ear infection  1  1/82 (1.22%)  4/87 (4.60%) 
Pharyngitis  1  1/82 (1.22%)  4/87 (4.60%) 
Skin bacterial infection  1  1/82 (1.22%)  4/87 (4.60%) 
Wound infection staphylococcal  1  1/82 (1.22%)  4/87 (4.60%) 
Injury, poisoning and procedural complications     
Wound  1  2/82 (2.44%)  3/87 (3.45%) 
Fall  1  3/82 (3.66%)  1/87 (1.15%) 
Musculoskeletal and connective tissue disorders     
Pain in extremity  1  2/82 (2.44%)  2/87 (2.30%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  4/82 (4.88%)  4/87 (4.60%) 
Rhinorrhoea  1  2/82 (2.44%)  4/87 (4.60%) 
Skin and subcutaneous tissue disorders     
Pruritus  1  9/82 (10.98%)  8/87 (9.20%) 
Blister  1  3/82 (3.66%)  2/87 (2.30%) 
Rash  1  2/82 (2.44%)  3/87 (3.45%) 
1
Term from vocabulary, MedDRA (19.1)
Indicates events were collected by systematic assessment
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: NCT02384460     History of Changes
Other Study ID Numbers: SD-005
2014-002288-14 ( EudraCT Number )
R01-005095-01 ( Other Identifier: Orphan Product Grant )
First Submitted: February 13, 2015
First Posted: March 10, 2015
Results First Submitted: September 19, 2018
Results First Posted: October 16, 2018
Last Update Posted: November 15, 2018