Epidermolysis BullosaPhase 3NCT02670330
Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
A Phase 3 study testing SD-101-6.0 cream in people with Epidermolysis Bullosa, run by Scioderm, Inc. at 30 sites in 12 countries.
Trial summary
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB). Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- SD-101-6.0 cream
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 152 participants
- Where
- 30 sitesin 12 countries
- Study dates
- 9 Jun 2015to 3 Sep 2018
- Phase
- Phase 3
- 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 1 month 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 was under the age of 18 but capable of providing assent, signed assent from the participant.
- Participant (or caretaker) must have been willing to comply with all protocol requirements.
- Participants who completed the SD-005 study (on study drug at Visit 5, approximately 90 days from baseline).
Who cannot join
- Participants who did not meet the entry criteria outlined above.
- Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at the final visit for Study SD-005 for female participants of childbearing potential and repeated at screening/baseline visit of Study SD-006 if these visits did not occur on the same day).
- Female participants of childbearing potential who were not abstinent or not practicing a medically acceptable method of contraception.
Trial locations and contacts
30 sites in 12 countries.
30 sites in 12 countries
United States15 sites
Study site
Aurora, Colorado, 80045
Study site
Chapel Hill, North Carolina, 27516
Study site
Charleston, South Carolina, 29425-5780
Study site
Chicago, Illinois, 60611
Study site
Cincinnati, Ohio, 45229-3039
Study site
Columbia, Missouri, 65212
Study site
East Setauket, New York, 11733
Study site
Minneapolis, Minnesota, 55455
Study site
Philadelphia, Pennsylvania, 19104
Study site
Phoenix, Arizona, 85016
Study site
Redwood City, California, 94063
Study site
San Antonio, Texas, 78218
Study site
Seattle, Washington, 98105
Study site
St Louis, Missouri, 63110
Study site
Washington D.C., District of Columbia, 20016
France3 sites
Study site
Nice, 1-06202
Study site
Paris, 75015
Study site
Toulouse
Australia2 sites
Study site
Kogarah, New South Wales, 2217
Study site
Parkville, Victoria, 3052
Germany2 sites
Study site
Freiburg im Breisgau, 79104
Study site
Hanover, 30173
About this study
Official title: An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisa™ (SD-101-6.0) in Patients With Epidermolysis Bullosa
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
Read the detailed description
This was an open label, multi-center extension study to assess the long-term safety of topically applied SD-101-6.0 in participants with simplex, recessive dystrophic, and junctional non-Herlitz EB. SD-101-6.0 was applied topically once a day to the entire body. The planned duration of treatment with SD-101-6.0 for Study SD-006 was up to 48 months, with a safety follow-up period of 30 days; however, the study was terminated early by the sponsor. The maximum study duration completed by at least 1 participant, treatment and safety follow-up, was 37 months.
Participants who successfully completed Study SD-005 had the option to rollover into Study SD-006. The screening/baseline visit (Visit 1) occurred at Visit 5 (approximately 90 days from baseline) of Study SD-005. The Body Surface Area (BSA) assessments of lesional skin and wound burden performed at Visit 5 (approximately 90 days from baseline) for Study SD-005 were utilized as the baseline assessments for Study SD-006. Participants returned for follow-up visits at Month 1 then every 3 months.
At each visit, assessments included BSA of lesional skin and wound burden. For target wounds that are not closed by the end of Study SD-005, the ARANZ picture and calculation of target wound area at the final visit for Study SD-005 was used as the baseline area size of the target wound for Study SD-006. These unhealed target wounds from Study SD-005 were assessed via ARANZ SilhouetteStar™ at each subsequent scheduled visit until the target wound was documented as closed. Closed wounds were assessed for scarring.
- Conditions
- Epidermolysis Bullosa
- Being tested
- SD-101-6.0 cream
- Keywords
- Amicus Therapeutics, Scioderm, Inc., Simplex, Recessive Dystrophic, Junctional non-Herlitz, Epidermolysis Bullosa, 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-101-6.0 creamDrug
Also called Zorblisa, SD-101
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 |
|---|---|---|
| Experimental: SD-101-6.0 cream | Experimental | All participants (or their caregivers) applied SD-101-6.0 cream topically once a day to the entire body for a period of up to 36 months. Receives: Drug: SD-101-6.0 cream |
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)
TEAEs were defined as adverse events that started or worsened on or after baseline visit.
Measured: From baseline to 30 days after last application of study drug (up to a maximum of 37 months)
Secondary outcomes (2)
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30
Lesional skin was defined as areas that contained any of the following: blisters, erosions, ulcerations, scabbing, bullae, or eschars, as well as areas that were weeping, sloughing, oozing, crusted, or denuded. The percentage, ranging from 0% to 100%, of affected body surface area (BSA) was recorded for each defined body region (that is, head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, then summed for all body regions to calculate the BSAI that would range from 0% to 100%. The BSA for lesional skin was to be assessed by the same study physician on each visit for a particular participant. The mean change from baseline in BSAI was assessed every 3 months. Only participants with data available for analysis at each time point are presented.
Measured: Baseline, up to Month 30
Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30
A wound was defined as an open area on the skin (that is, epidermal covering disrupted). Total body wound burden was calculated using BSAI; the percentage, ranging from 0% to 100%, of affected BSA was recorded for each defined body region (that is, head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, then summed for all body regions to calculate the BSAI that would range from 0% to 100%. The BSAI for total body wound burden was to be assessed by the same study physician at each visit for a particular participant.
The mean change from baseline in total body wound burden was assessed every 3 months. Only participants with data available for analysis at each time point are presented.
Measured: Baseline, up to Month 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2016 (estimated)
- Study start9 Jun 2015
- Primary completion3 Sep 2018
- Study completion3 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.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 152 participants (actual)
Sponsor and investigators
- Lead sponsor
- Scioderm, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Amicus Therapeutics
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Amicus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02670330
- Sponsor's protocol ID
- SD-006
- Other IDs
- 2014-005679-96, 2014
- Status last verified
- Sep 2019
- First submitted
- 28 Jul 2015
- Last updated
- 27 Sep 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisa™ (SD-101-6.0) in Patients With Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT02670330
- Acronym / sponsor ID
- SD-006
- Conditions
- Epidermolysis Bullosa
- Keywords
- Amicus Therapeutics, Scioderm, Inc., Simplex, Recessive Dystrophic, Junctional non-Herlitz, Epidermolysis Bullosa, SD-101 6%, SD-101-6.0
- Phase
- Phase 3
- 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
- 152 participants (actual)
- Lead sponsor
- Scioderm, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Amicus Therapeutics
- Study start
- 9 Jun 2015 (actual)
- Primary completion
- 3 Sep 2018 (actual)
- Study completion
- 3 Sep 2018 (actual)
- First posted
- 1 Feb 2016 (estimated)
- Results
- Posted 2 Aug 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
Detailed description
This was an open label, multi-center extension study to assess the long-term safety of topically applied SD-101-6.0 in participants with simplex, recessive dystrophic, and junctional non-Herlitz EB. SD-101-6.0 was applied topically once a day to the entire body. The planned duration of treatment with SD-101-6.0 for Study SD-006 was up to 48 months, with a safety follow-up period of 30 days; however, the study was terminated early by the sponsor. The maximum study duration completed by at least 1 participant, treatment and safety follow-up, was 37 months.
Participants who successfully completed Study SD-005 had the option to rollover into Study SD-006. The screening/baseline visit (Visit 1) occurred at Visit 5 (approximately 90 days from baseline) of Study SD-005. The Body Surface Area (BSA) assessments of lesional skin and wound burden performed at Visit 5 (approximately 90 days from baseline) for Study SD-005 were utilized as the baseline assessments for Study SD-006. Participants returned for follow-up visits at Month 1 then every 3 months.
At each visit, assessments included BSA of lesional skin and wound burden. For target wounds that are not closed by the end of Study SD-005, the ARANZ picture and calculation of target wound area at the final visit for Study SD-005 was used as the baseline area size of the target wound for Study SD-006. These unhealed target wounds from Study SD-005 were assessed via ARANZ SilhouetteStar™ at each subsequent scheduled visit until the target wound was documented as closed. Closed wounds were assessed for scarring.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental: SD-101-6.0 cream | Experimental | Drug: SD-101-6.0 creamAll participants (or their caregivers) applied SD-101-6.0 cream topically once a day to the entire body for a period of up to 36 months. |
Who may qualify
- Ages
- 1 month 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 was under the age of 18 but capable of providing assent, signed assent from the participant.
- Participant (or caretaker) must have been willing to comply with all protocol requirements.
- Participants who completed the SD-005 study (on study drug at Visit 5, approximately 90 days from baseline).
Key exclusion criteria (3 listed)
- Participants who did not meet the entry criteria outlined above.
- Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at the final visit for Study SD-005 for female participants of childbearing potential and repeated at screening/baseline visit of Study SD-006 if these visits did not occur on the same day).
- Female participants of childbearing potential who were not abstinent or 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-101-6.0 creamAlso: Zorblisa, SD-101SD-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 | Experimental: SD-101-6.0 cream |
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 37 months)
TEAEs were defined as adverse events that started or worsened on or after baseline visit.
2 secondary outcomes
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30
Time frame: Baseline, up to Month 30
Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30
Time frame: Baseline, up to Month 30
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Sep 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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
30 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing SD-101-6.0 cream in people with Epidermolysis Bullosa, run by Scioderm, Inc. at 30 sites in 12 countries.
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 27 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, France, Australia, Germany, Austria and 7 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 2 Aug 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 37 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Sep 2019.