AzTrials
AzTrials

Epidermolysis BullosaPhase 2NCT02014376

Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa

A Phase 2 study testing SD-101 dermal cream (3%) and SD-101 Dermal Cream (6%) in people with Epidermolysis Bullosa, run by Scioderm, Inc. at 7 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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). 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 (3%) and SD-101 Dermal Cream (6%)
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
48 participants
Where
7 sitesUnited States
Study dates
6 Jan 2014to 24 Jun 2014
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 the participant's legal representative; if the participant is under the age of majority but capable of providing assent, signed assent from the participant.
  • Participant (or caretaker) was willing to comply with all protocol requirements.
  • Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
  • Participants 6 months of age and older.
  • Participants had 1 target wound within a prespecified size range at study entry.
  • Target wound was at least 21 days or older.

Who cannot join

  • Participants who did not meet all the entry criteria outlined in inclusion criteria.
  • Selected target wound had clinical evidence of local infection.
  • Use of any investigational drug within 30 days before enrollment.
  • Use of immunotherapy or cytotoxic chemotherapy within 60 days before enrollment.
  • Use of systemic or topical steroidal therapy within 30 days before enrollment (inhaled steroids and ophthalmic drops containing steroids were allowed).
  • Use of systemic antibiotics within 7 days before enrollment.
Show all 12Show fewer
  • Current or former malignancy.
  • Arterial or venous disorder resulting in ulcerated wounds.
  • Diabetes mellitus.
  • Pregnancy or breastfeeding during the study (a urine pregnancy test was performed at screening for female participants of childbearing potential).
  • Females of childbearing potential who were not abstinent and not practicing a medically acceptable method of contraception.
  • Known history of cardiac, hepatic, or renal disease.

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

Other trials in United States

About this study

Official title: A Phase 2b, Multi-Center, Randomized, Double-Blind, Vehicle-Controlled Study of the Effectiveness and Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa

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).

Read the detailed description

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.

Being tested
SD-101 dermal cream (3%), SD-101 Dermal Cream (6%), Vehicle (SD-101 0%)
Keywords
Epidermolysis Bullosa, Simplex, Recessive Dystrophic, Junctional non-Herlitz, Amicus Therapeutics, Scioderm, Inc., SD-101 6%, SD-101-6.0, Allantoin 6%, SD-101 3%, SD-101-3.0, Allantoin 3%, 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 (3%)Drug

    Also called SD-101, SD-101-3.0, Zorblisa

    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.

  • 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.

  • Vehicle (SD-101 0%)Drug

    Also called Placebo Comparator, SD-101-0.0

    A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.

Study groups in NCT02014376
Study groupRoleWhat they receive
SD-101 Dermal Cream (6%)ExperimentalSD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.
Receives: Drug: SD-101 Dermal Cream (6%)
SD-101 Dermal Cream (3%)ExperimentalSD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days.
Receives: Drug: SD-101 dermal cream (3%)
Vehicle (0%)Placebo comparatorVehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.
Receives: Drug: Vehicle (SD-101 0%)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Baseline to 1 Month

Secondary outcomes (5)

  1. 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.

    Measured: Baseline to Month 2 and Month 3

  2. 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.

    Measured: Baseline, Month 3

  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.

    Measured: Baseline, Day 7

  4. 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.

    Measured: Baseline, Day 7

  5. 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).

    Measured: Week 2, Month 1, Month 2, and Month 3

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Dec 2013 (estimated)
  2. Study start6 Jan 2014
  3. Primary completion24 Jun 2014
  4. Study completion24 Jun 2014

Resources

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
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
48 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02014376
Sponsor's protocol ID
SD-003
Status last verified
Jan 2020
First submitted
12 Dec 2013
Last updated
13 Jan 2020
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing SD-101 dermal cream (3%) and 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 6 inclusion and 12 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: finished, as of the sponsor's last update on 13 Jan 2020. 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. Some participants in this study may receive a placebo. 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 13 Jan 2020). 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?

Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment (time frame: Baseline to 1 Month). 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 NCT02014376.

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 13 Jan 2020.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in