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| Name | Class |
|---|---|
| Amicus Therapeutics | INDUSTRY |
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The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).
This was a Phase 2b, multi-center, randomized, double-blind, vehicle-controlled study to assess the efficacy and safety of SD-101 dermal cream (3% or 6%) on skin lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
SD-101 cream [containing 3% or 6% or vehicle (0%)] was applied topically, once a day to the entire body for a period of 90 days. Eligible participants had a target wound assessed at baseline. Selected target wound had to be at least a certain age and within a prespecified size range at study entry. 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 at Week 2, Month 1, Month 4, and Month 5 (at 14, 30, 60, and 90 days, respectively) to have the target wound previously identified at baseline re-assessed for the level of healing. In addition, changes in itching, pain, body surface area coverage of blisters and lesions, and scaring of the healed target wound were also assessed at each visit.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SD-101 Dermal Cream (6%) | Experimental | SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days. |
|
| SD-101 Dermal Cream (3%) | Experimental | SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days. |
|
| Vehicle (0%) | Placebo Comparator | Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SD-101 dermal cream (3%) | Drug | SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-3.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 3% and other excipients. |
| Measure | Description | Time Frame |
|---|---|---|
| Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment | The ARANZ SilhouetteStar™, a wound imaging, measurement, and documentation system providing accurate wound assessment, was used to measure the target wound at all visits. Information captured included photographic images, quantitative measures, and other target wound assessment data input to the device by the clinician, all obtained with no contact to the participant's skin. Information about the target wound's measurement history was available on this system so that the serial progression of the target wound status could also be calculated and presented. | Baseline to 1 Month |
| Measure | Description | Time Frame |
|---|---|---|
| Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment | The ARANZ SilhouetteStar™, a wound imaging, measurement, and documentation system providing accurate wound assessment, was used to measure the target wound at all visits. Information captured included photographic images, quantitative measures, and other target wound assessment data input to the device by the clinician, all obtained with no contact to the participant's skin. Information about the target wound's measurement history was available on this system so that the serial progression of the target wound status could also be calculated and presented. |
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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 |
|---|---|---|---|---|---|---|
| Palo Alto | California | 94304 | United States | |||
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| ID | Title | Description |
|---|---|---|
| FG000 | SD-101 Dermal Cream (6%) | SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days. |
| FG001 | SD-101 Dermal Cream (3%) | SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
| SD-101 Dermal Cream (6%) | Drug | 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. |
|
|
| Vehicle (SD-101 0%) | Drug | A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients. |
|
|
| Baseline to Month 2 and Month 3 |
| Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3 | The BSAI is a global measure of disease "spread" with weighting factors. Lesional skin consisted of area(s) that could contain any of the following: blisters, bullae, erosions, ulcerations, scabbing and eschars, as well as areas that are weeping, sloughing, oozing, crusted and denuded. The percentage, ranging from 0% to 100%, of affected body surface area was recorded for each defined body region (head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, and then summed for all body regions to calculate the BSAI. The BSAI affected with blisters and wounds was calculated at baseline and Month 3 to assess the total affected area. Percentage change from baseline was calculated as follows: Percentage change from baseline = 100*(Post-baseline value minus Baseline value) divided by Baseline value. Mean percentage change from baseline in BSAI is reported. Only participants with data available for analysis at the specified time point are presented. | Baseline, Month 3 |
| Participants Experiencing A Change From Baseline In Itching At Day 7 | The Itch Man Pruritus Assessment Tool was used to measure the intensity of itching. Itching was assessed and reported at Baseline and Day 7. For participants 6 months to 5 years of age, itching was assessed using the caretaker's response, while in participants 6 years and older, itching was self-reported. | Baseline, Day 7 |
| Change From Baseline In Pain At Day 7 | Pain was assessed at Baseline and Day 7. The presence and intensity of pain was assessed using the Face, Legs, Activity, Cry, Consolability (FLACC) Pain Scale for participants 6 months to 3 years of age. For participants aged 4 years and older, the Wong Faces Pain Scale was used. Scores were attributed for each of the 5 categories in the FLACC scale from 0 to 2, which resulted in a total score between 0 and 10. The Wong Faces Pain scale used 1 item to rate pain on a 0 to 10 scale. Higher score values indicated more pain. | Baseline, Day 7 |
| Participants With Scarring At Week 2, Month 1, Month 2, And Month 3 | In the event of a healed wound, where complete closure was confirmed, the extent of scarring was assessed as "Present" or "Absent" at all post-baseline visits (Week 2 and Months 1, 2, and 3). | Week 2, Month 1, Month 2, and Month 3 |
| Chicago |
| Illinois |
| 60611 |
| United States |
| St Louis | Missouri | 63110 | United States |
| Hackensack | New Jersey | 07601 | United States |
| Chapel Hill | North Carolina | 27516 | United States |
| San Antonio | Texas | 78218 | United States |
| Seattle | Washington | 98105 | United States |
| FG002 | Vehicle (0%) | Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days. |
| Received at Least 1 Dose of Study Drug | Intent-to-treat (ITT) Population |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Intent-to-treat (ITT) Population: Participants who provided informed consent, had been randomized, and had received at least 1 application of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | SD-101 Dermal Cream (6%) | SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days. |
| BG001 | SD-101 Dermal Cream (3%) | SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days. |
| BG002 | Vehicle (0%) | Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days. |
| BG003 | 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 |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Epidermolysis Bullosa Subtype | 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 | Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment | The ARANZ SilhouetteStar™, a wound imaging, measurement, and documentation system providing accurate wound assessment, was used to measure the target wound at all visits. Information captured included photographic images, quantitative measures, and other target wound assessment data input to the device by the clinician, all obtained with no contact to the participant's skin. Information about the target wound's measurement history was available on this system so that the serial progression of the target wound status could also be calculated and presented. | Intent-to-treat (ITT) Population: Participants who provided informed consent, had been randomized, and had received at least 1 application of study drug. | Posted | Count of Participants | Participants | Baseline to 1 Month |
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| Secondary | Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment | The ARANZ SilhouetteStar™, a wound imaging, measurement, and documentation system providing accurate wound assessment, was used to measure the target wound at all visits. Information captured included photographic images, quantitative measures, and other target wound assessment data input to the device by the clinician, all obtained with no contact to the participant's skin. Information about the target wound's measurement history was available on this system so that the serial progression of the target wound status could also be calculated and presented. | Intent-to-treat (ITT) Population: Participants who provided informed consent, had been randomized, and had received at least 1 application of study drug. | Posted | Count of Participants | Participants | Baseline to Month 2 and Month 3 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage Change From Baseline In Lesional Skin Based On Body Surface Area Index (BSAI) Measurements At Month 3 | The BSAI is a global measure of disease "spread" with weighting factors. Lesional skin consisted of area(s) that could contain any of the following: blisters, bullae, erosions, ulcerations, scabbing and eschars, as well as areas that are weeping, sloughing, oozing, crusted and denuded. The percentage, ranging from 0% to 100%, of affected body surface area was recorded for each defined body region (head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, and then summed for all body regions to calculate the BSAI. The BSAI affected with blisters and wounds was calculated at baseline and Month 3 to assess the total affected area. Percentage change from baseline was calculated as follows: Percentage change from baseline = 100*(Post-baseline value minus Baseline value) divided by Baseline value. Mean percentage change from baseline in BSAI is reported. Only participants with data available for analysis at the specified time point are presented. | Intent-to-treat (ITT) Population: Participants who provided informed consent, had been randomized, and had received at least 1 application of study drug. | Posted | Mean | Standard Deviation | percentage of BSAI | Baseline, Month 3 |
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| Secondary | Participants Experiencing A Change From Baseline In Itching At Day 7 | The Itch Man Pruritus Assessment Tool was used to measure the intensity of itching. Itching was assessed and reported at Baseline and Day 7. For participants 6 months to 5 years of age, itching was assessed using the caretaker's response, while in participants 6 years and older, itching was self-reported. | Intent-to-treat (ITT) Population: Participants who provided informed consent, had been randomized, and had received at least 1 application of study drug. | Posted | Count of Participants | Participants | Baseline, Day 7 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Pain At Day 7 | Pain was assessed at Baseline and Day 7. The presence and intensity of pain was assessed using the Face, Legs, Activity, Cry, Consolability (FLACC) Pain Scale for participants 6 months to 3 years of age. For participants aged 4 years and older, the Wong Faces Pain Scale was used. Scores were attributed for each of the 5 categories in the FLACC scale from 0 to 2, which resulted in a total score between 0 and 10. The Wong Faces Pain scale used 1 item to rate pain on a 0 to 10 scale. Higher score values indicated more pain. | Intent-to-treat (ITT) Population: Participants who provided informed consent, had been randomized, and had received at least 1 application of study drug. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Day 7 |
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| Secondary | Participants With Scarring At Week 2, Month 1, Month 2, And Month 3 | In the event of a healed wound, where complete closure was confirmed, the extent of scarring was assessed as "Present" or "Absent" at all post-baseline visits (Week 2 and Months 1, 2, and 3). | Intent-to-treat (ITT) Population: Participants who provided informed consent, had been randomized, had received at least 1 application of study drug, and whose target wounds healed while receiving study drug. | Posted | Count of Participants | Participants | Week 2, Month 1, Month 2, and Month 3 |
|
|
From baseline (Day 0) to 30 days after last application of study drug (up to a maximum of 4 months).
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| 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 Dermal Cream (6%) | SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days. | 0 | 15 | 0 | 15 | 9 | 15 |
| EG001 | SD-101 Dermal Cream (3%) | SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days. | 0 | 16 | 2 | 16 | 13 | 16 |
| EG002 | Vehicle (0%) | Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days. | 0 | 17 | 1 | 17 | 12 | 17 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bacterial tracheitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Catheter site cellulitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Feeding disorder | Metabolism and nutrition disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Catheter site infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Eye infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Application site pain | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Application site discharge | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Irritability | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Hair growth abnormal | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Miliaria | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Skin haemorrhage | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Increased bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Aphonia | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (16.1) | Systematic Assessment |
| |
| Excoriation | Injury, poisoning and procedural complications | MedDRA (16.1) | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA (16.1) | Systematic Assessment |
| |
| Wound complication | Injury, poisoning and procedural complications | MedDRA (16.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Corneal disorder | Eye disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Eye swelling | Eye disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Protein deficiency | Metabolism and nutrition disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Cerumen impaction | Ear and labyrinth disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Food allergy | Immune system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (16.1) | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Patient Advocacy | Amicus Therapeutics, Inc. | +1-609-662-2000 | clinicaltrials@amicusrx.com |
| 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 |
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| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Recessive Dystrophic |
|
| Junctional non-Herlitz |
|
| Title | Measurements |
|---|---|
|
Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.
|
|
SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days. |
| OG002 | Vehicle (0%) | Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days. |
|
|
| Units |
|---|
| Counts |
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| Participants |
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| Counts |
|---|
| Participants |
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| Absent |
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| Absent |
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| Absent |
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