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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-002288-14 | EudraCT Number | ||
| R01-005095-01 | Other Identifier | Orphan Product Grant |
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| Name | Class |
|---|---|
| Amicus Therapeutics | INDUSTRY |
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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).
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. 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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SD-101-6.0 cream | Experimental | 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 | Placebo Comparator | SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SD-101-6.0 cream | Drug | applied topically once a day for 90 days |
|
| Measure | Description | Time Frame |
|---|---|---|
| Time To Complete Target Wound Closure Within 3 Months | 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. | From baseline to Month 3 visit |
| The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months | 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. | From baseline to Month 3 visit |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Amicus Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix | Arizona | 85016 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32693833 | Derived | Murrell DF, Paller AS, Bodemer C, Browning J, Nikolic M, Barth JA, Lagast H, Krusinska E, Reha A; ESSENCE Study Group. Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study. Orphanet J Rare Dis. 2020 Jul 21;15(1):190. doi: 10.1186/s13023-020-01435-3. | |
| 32576219 | Derived |
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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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | SD-101-6.0 Cream | Participants applied SD-101-6.0 cream topically, once a day to the entire body for a period of 90 days. |
| FG001 | Placebo (SD-101-0.0) Cream | Participants applied Placebo (SD-101-0.0) cream topically, once a day to the entire body for a period of 90 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 20, 2017 | Sep 19, 2018 |
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| Placebo (SD-101-0.0) cream | Drug | applied topically once a day for 90 days |
|
|
| From baseline to Month 1 and Month 2 visits |
| Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At 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. | Baseline, Month 3 visit |
| Change From Baseline In BSAI Of Total Body Wound Burden At 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. | Baseline, Month 3 visit |
| Change From Baseline In Itching Score At 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. | Baseline, Day 7 |
| Change From Baseline In Pain Score At 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. | Baseline, Day 7 |
| Redwood City |
| California |
| 94063 |
| United States |
| Aurora | Colorado | 80045 | United States |
| Washington D.C. | District of Columbia | 20016 | United States |
| Miami | Florida | 33136 | United States |
| Miami | Florida | 33155 | United States |
| Chicago | Illinois | 60611 | United States |
| Minneapolis | Minnesota | 55455 | United States |
| Columbia | Missouri | 65212 | United States |
| St Louis | Missouri | 63110 | United States |
| Lebanon | New Hampshire | 03756 | United States |
| Hackensack | New Jersey | 07601 | United States |
| East Setauket | New York | 11733 | United States |
| New York | New York | 10032 | United States |
| Chapel Hill | North Carolina | 27516 | United States |
| Cincinnati | Ohio | 45229-3039 | United States |
| Philadelphia | Pennsylvania | 19104 | United States |
| Charleston | South Carolina | 29425-5780 | United States |
| Austin | Texas | 78723 | United States |
| San Antonio | Texas | 78218 | United States |
| Seattle | Washington | 98105 | United States |
| Kogarah | New South Wales | 2217 | Australia |
| Parkville | Victoria | 3050 | Australia |
| Parkville | Victoria | 3052 | Australia |
| Salzburg | 5020 | Austria |
| Nice | 0-06200 | France |
| Paris | 75015 | France |
| Toulouse | France |
| Freiburg im Breisgau | Germany |
| Hanover | Germany |
| Tel Aviv | Israel |
| Milan | 20122 | Italy |
| Kaunas | Lithuania |
| Groningen | 9713 GZ | Netherlands |
| Warsaw | Koszykowa | Poland |
| Belgrade | Serbia |
| Madrid | Spain |
| London | WC1N 3JH | United Kingdom |
| Paller AS, Browning J, Nikolic M, Bodemer C, Murrell DF, Lenon W, Krusinska E, Reha A, Lagast H, Barth JA; ESSENCE Study Group. Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study). Orphanet J Rare Dis. 2020 Jun 23;15(1):158. doi: 10.1186/s13023-020-01419-3. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
The Intent-to-treat (ITT) population included all randomized participants.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | Placebo (SD-101-0.0) Cream | Placebo (SD-101-0.0) cream applied topically, once a day to the entire body for a period of 90 days. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| EB type | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Time To Complete Target Wound Closure Within 3 Months | 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. | ITT population with post-baseline wound closure data and whose target wound had closed within 3 months. | Posted | Mean | Standard Deviation | days | From baseline to Month 3 visit |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | The Percentage Of Participants Experiencing Complete Closure Of Their Target Wound Within 3 Months | 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. | ITT population with post-baseline wound closure data and whose target wound had closed within 3 months. | Posted | Number | percentage of participants | From baseline to Month 3 visit |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At 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. | Analysis was performed on participants from the ITT population with post-baseline wound closure data. | Posted | Number | percentage of participants | From baseline to Month 1 and Month 2 visits |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At 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. | Analysis was performed on the ITT population. The ITT population included all randomized participants. | Posted | Least Squares Mean | Standard Error | Percentage change in BSAI | Baseline, Month 3 visit |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In BSAI Of Total Body Wound Burden At 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. | Analysis was performed on the ITT population. The ITT population included all randomized participants. | Posted | Least Squares Mean | Standard Error | Percentage change in BSAI | Baseline, Month 3 visit |
|
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| Secondary | Change From Baseline In Itching Score At 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. | Analysis was performed on the ITT population. The ITT population included all randomized participants. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Day 7 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Pain Score At 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. | Analysis was performed on the ITT population. The ITT population included all randomized participants. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Day 7 |
|
From baseline up to 3 months.
Adverse events (AEs) were defined as treatment-emergent AEs if the AE occurred on or after the first date of application of study drug.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. | 0 | 82 | 4 | 82 | 49 | 82 |
| EG001 | Placebo (SD-101-0.0) Cream | Placebo (SD-101-0.0) cream applied topically, once a day to the entire body for a period of 90 days. | 1 | 87 | 8 | 87 | 46 | 87 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cardiopulmonary failure | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oesophageal stenosis | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Staphylococcal skin infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Aggression | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Circulatory collapse | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Skin bacterial infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Staphyloccocal skin infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Wound infection staphylococcal | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Patient Advocacy | Amicus Therapeutics, Inc. | +1-609-662-2000 | clinicaltrials@amicusrx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 22, 2018 | Sep 19, 2018 | SAP_001.pdf |
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| ID | Term |
|---|---|
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
Not provided
Not provided
| Male |
|
| Black/African American |
|
| Asian |
|
| Other |
|
| Not Reported |
|
| Recessive dystrophic |
|
| Junctional non-Herlitz |
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