Epidermolysis BullosaPhase 2NCT02090283
Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
A Phase 2 study testing SD-101 dermal cream (6%) in people with Epidermolysis Bullosa, run by Scioderm, Inc. at 7 sites in United States.
Trial summary
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB). Funding Source: FDA Office of Orphan Products Development Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- SD-101 dermal cream (6%)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 7 sitesUnited States
- Study dates
- 26 Mar 2014to 14 Sep 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Informed consent form signed by the participant or participant's legal representative; if the participant is under the age of 18 but capable of providing assent, signed assent from the participant.
- Participant (or caretaker) must be willing to comply with all protocol requirements.
- Participant must have successfully completed the SD-003 study.
Who cannot join
- Participants who do not meet the inclusion criteria.
- Pregnancy or breastfeeding during the study. A urine pregnancy test will be performed at the final visit for SD-003 for female participants of childbearing potential.
- Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Study site
Chapel Hill, North Carolina, 27516
Study site
Chicago, Illinois, 60611
Study site
Hackensack, New Jersey, 07601
Study site
Palo Alto, California, 94304
Study site
San Antonio, Texas, 78218
Study site
Seattle, Washington, 98105
Study site
St Louis, Missouri, 63110
About this study
Official title: An Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB).
Funding Source: FDA Office of Orphan Products Development
Read the detailed description
This was an open-label extension study to assess the continued safety of topically applied SD-101 dermal cream (6%) in participants with Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB.
SD-101 dermal cream (6%) was applied topically, once a day to the entire body for the duration of the study. Participants who successfully completed the entire SD-003 study (NCT02014376) had the option to roll over into the SD-004 study (NCT02090283). The baseline visit occurred at the final visit date for the SD-003 study. The body surface area (BSA) coverage of blisters and lesions assessment made at the final SD-003 study visit were used as the baseline information at the baseline visit for the SD-004 study. Participants returned to the study site for the following 13 visits (36 months) to have BSA assessed. BSA was assessed at all subsequent scheduled study center visits. Scheduled study center visits occurred every 6 months after Month 36 (Month 42, 48, and so on). After completion of Month 36, the next participant visit (Month 39) was a phone call from the site to the participant. Telephone visits occurred every 6 months thereafter (Month 45, 51, and so on) and included assessment of adverse events and concomitant medications only. For female participants of childbearing potential, a urine pregnancy test was performed at Month 6 and every 6 months up to and including the final study visit.
- Conditions
- Epidermolysis Bullosa
- Being tested
- SD-101 dermal cream (6%)
- Keywords
- Amicus Therapeutics, Scioderm, Inc., Recessive Dystrophic, Junctional non-Herlitz, Epidermolysis Bullosa, Simplex, SD-101 6%, SD-101-6.0, Allantoin 6%, Zorblisa
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SD-101 dermal cream (6%)Drug
Also called SD-101, SD-101-6.0, Zorblisa
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.
| Study group | Role | What they receive |
|---|---|---|
| SD-101 Dermal Cream (6%) | Experimental | All participants applied SD-101 dermal cream topically, once a day, to the entire body for the duration of the study. Receives: Drug: SD-101 dermal cream (6%) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number Of Participants With Treatment-Emergent Adverse Events (TEAEs)
Treatment-emergent adverse events were defined as adverse events that started or worsened on or after baseline visit, which occurred at the final visit date for the SD-003 study. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Measured: From baseline to 30 days after last application of study drug (up to a maximum of 54 months)
Secondary outcomes (1)
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 24
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, and then summed for all body regions to calculate the BSAI. 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 (final visit from the SD-003 study) in BSAI was assessed every 3 months. Only participants with data available for analysis at the specified time point are presented.
Measured: Baseline, Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2014 (estimated)
- Study start26 Mar 2014
- Primary completion14 Sep 2018
- Study completion14 Sep 2018
Why it stopped: 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Scioderm, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Amicus Therapeutics, Food and Drug Administration (FDA)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Amicus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02090283
- Sponsor's protocol ID
- SD-004
- Other IDs
- 1R01FD005093-01
- Status last verified
- Oct 2019
- First submitted
- 12 Mar 2014
- Last updated
- 4 Nov 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Paller A., Browning J., Aslam R., et al. Efficacy and Safety Results From a 24-Month, Open-Label Extension of a Phase 2b Dose-Ranging Study of SD-101 Cream in Patients With Epidermolysis Bullosa. Abstract presented at European Academy of Dermatology and Venereology, 26th Congress, Geneva, Switzerland, September 13-17, 2017.
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT02090283
- Acronym / sponsor ID
- SD-004
- Conditions
- Epidermolysis Bullosa
- Keywords
- Amicus Therapeutics, Scioderm, Inc., Recessive Dystrophic, Junctional non-Herlitz, Epidermolysis Bullosa, Simplex, SD-101 6%, SD-101-6.0
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 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.
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Scioderm, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Amicus Therapeutics, Food and Drug Administration (FDA)
- Study start
- 26 Mar 2014 (actual)
- Primary completion
- 14 Sep 2018 (actual)
- Study completion
- 14 Sep 2018 (actual)
- First posted
- 18 Mar 2014 (estimated)
- Results
- Posted 4 Nov 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB).
Funding Source: FDA Office of Orphan Products Development
Detailed description
This was an open-label extension study to assess the continued safety of topically applied SD-101 dermal cream (6%) in participants with Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB.
SD-101 dermal cream (6%) was applied topically, once a day to the entire body for the duration of the study. Participants who successfully completed the entire SD-003 study (NCT02014376) had the option to roll over into the SD-004 study (NCT02090283). The baseline visit occurred at the final visit date for the SD-003 study. The body surface area (BSA) coverage of blisters and lesions assessment made at the final SD-003 study visit were used as the baseline information at the baseline visit for the SD-004 study. Participants returned to the study site for the following 13 visits (36 months) to have BSA assessed. BSA was assessed at all subsequent scheduled study center visits. Scheduled study center visits occurred every 6 months after Month 36 (Month 42, 48, and so on). After completion of Month 36, the next participant visit (Month 39) was a phone call from the site to the participant. Telephone visits occurred every 6 months thereafter (Month 45, 51, and so on) and included assessment of adverse events and concomitant medications only. For female participants of childbearing potential, a urine pregnancy test was performed at Month 6 and every 6 months up to and including the final study visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SD-101 Dermal Cream (6%) | Experimental | Drug: SD-101 dermal cream (6%)All participants applied SD-101 dermal cream topically, once a day, to the entire body for the duration of the study. |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Informed consent form signed by the participant or participant's legal representative; if the participant is under the age of 18 but capable of providing assent, signed assent from the participant.
- Participant (or caretaker) must be willing to comply with all protocol requirements.
- Participant must have successfully completed the SD-003 study.
Key exclusion criteria (3 listed)
- Participants who do not meet the inclusion criteria.
- Pregnancy or breastfeeding during the study. A urine pregnancy test will be performed at the final visit for SD-003 for female participants of childbearing potential.
- Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SD-101 dermal cream (6%)Also: SD-101, SD-101-6.0, ZorblisaSD-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. | Drug | SD-101 Dermal Cream (6%) |
Primary outcome
Number Of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: From baseline to 30 days after last application of study drug (up to a maximum of 54 months)
Treatment-emergent adverse events were defined as adverse events that started or worsened on or after baseline visit, which occurred at the final visit date for the SD-003 study. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
1 secondary outcome
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 24
Time frame: Baseline, Month 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publicationPaller A., Browning J., Aslam R., et al. Efficacy and Safety Results From a 24-Month, Open-Label Extension of a Phase 2b Dose-Ranging Study of SD-101 Cream in Patients With Epidermolysis Bullosa. Abstract presented at European Academy of Dermatology and Venereology, 26th Congress, Geneva, Switzerland, September 13-17, 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Amicus Therapeutics
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SD-101 dermal cream (6%) in people with Epidermolysis Bullosa, run by Scioderm, Inc. at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 4 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Nov 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number Of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: From baseline to 30 days after last application of study drug (up to a maximum of 54 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02090283, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2019; AzTrials last refreshed its copy on 30 Oct 2026.