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NCT02670330

Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

Terminated Phase 3 Results posted Last updated 27 September 2019
What this trial tests

Phase 3 trial testing SD-101-6.0 cream in Epidermolysis Bullosa in 152 participants. Terminated before completion.

Timeline
9 June 2015
Primary endpoint
3 September 2018
3 September 2018

Quick facts

Lead sponsorScioderm, Inc.
PhasePhase 3
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment152
Start date9 June 2015
Primary completion3 September 2018
Estimated completion3 September 2018
Sites30 locations across France, Netherlands, Serbia, Austria, United Kingdom, Germany, Israel, Poland

Drugs / interventions tested

Conditions studied

Sponsor

Scioderm, Inc. — full company profile →

Who can join

1 Month and older, any sex, with Epidermolysis Bullosa. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number Of Participants With Treatment Emergent Adverse Events (TEAEs) Primary · 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.

Any TEAE
GroupValue95% CI
SD-101-6.0 to SD-101-6.051
Placebo to SD-101-6.058
Any TEAE Related To Study Drug
GroupValue95% CI
SD-101-6.0 to SD-101-6.08
Placebo to SD-101-6.017
Any Fatal TEAE
GroupValue95% CI
SD-101-6.0 to SD-101-6.00
Placebo to SD-101-6.00
Any Serious TEAE
GroupValue95% CI
SD-101-6.0 to SD-101-6.011
Placebo to SD-101-6.014
Any TEAE Leading To Discontinuation
GroupValue95% CI
SD-101-6.0 to SD-101-6.02
Placebo to SD-101-6.03
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30 Secondary · Baseline, 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 phys

Month 1
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-0.76± 4.185
Placebo to SD-101-6.0-0.54± 5.398
Month 3
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.87± 5.999
Placebo to SD-101-6.0-1.21± 6.457
Month 6
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.77± 6.015
Placebo to SD-101-6.0-1.35± 6.813
Month 9
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-2.48± 9.436
Placebo to SD-101-6.00.31± 10.117
Month 12
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-3.35± 9.566
Placebo to SD-101-6.00.44± 10.327
Month 15
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.90± 7.337
Placebo to SD-101-6.02.15± 12.546
Month 18
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-2.46± 5.275
Placebo to SD-101-6.0-4.24± 8.207
Month 21
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-3.06± 5.869
Placebo to SD-101-6.0-1.58± 10.442
Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30 Secondary · Baseline, 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

Month 1
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.75± 4.891
Placebo to SD-101-6.00.07± 4.044
Month 3
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.52± 5.343
Placebo to SD-101-6.0-0.80± 3.001
Month 6
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.54± 4.322
Placebo to SD-101-6.0-0.48± 3.595
Month 9
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.82± 6.083
Placebo to SD-101-6.0-0.42± 3.586
Month 12
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.38± 5.497
Placebo to SD-101-6.0-0.13± 3.211
Month 15
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-0.15± 4.511
Placebo to SD-101-6.00.26± 3.030
Month 18
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.12± 3.053
Placebo to SD-101-6.0-1.31± 4.665
Month 21
GroupValue95% CI
SD-101-6.0 to SD-101-6.0-1.44± 3.150
Placebo to SD-101-6.0-0.28± 5.507

Adverse events — posted to ClinicalTrials.gov

Time frame: From baseline to 30 days after last application of study drug (up to a maximum of 37 months).. Reporting threshold: 2%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

SD-101-6.0 to SD-101-6.0
Serious: 11/75 (15%)
Deaths: 0/75
Placebo to SD-101-6.0
Serious: 14/77 (18%)
Deaths: 0/77

Serious adverse events (34 terms)

ReactionSystemSD-101-6.0 to SD-101-6.0Placebo to SD-101-6.0
AnaemiaBlood and lymphatic system disorders
Oesophageal stenosisGastrointestinal disorders
Skin infectionInfections and infestations
PericarditisCardiac disorders
Congenital megaureterCongenital, familial and genetic disorders
KeratitisEye disorders
Abdominal painGastrointestinal disorders
ConstipationGastrointestinal disorders
DysphagiaGastrointestinal disorders
FaecalomaGastrointestinal disorders
Intestinal obstructionGastrointestinal disorders
Drug hypersensitivityImmune system disorders
Gastroenteritis clostridialInfections and infestations
Implant site infectionInfections and infestations
InfectionInfections and infestations
Otitis mediaInfections and infestations
Skin bacterial infectionInfections and infestations
Staphylococcal skin infectionInfections and infestations
Wound infectionInfections and infestations
Wound infection bacterialInfections and infestations
Joint dislocationInjury, poisoning and procedural complications
Post procedural fistulaInjury, poisoning and procedural complications
Procedural painInjury, poisoning and procedural complications
Procedural vomitingInjury, poisoning and procedural complications
Stoma site extravasationInjury, poisoning and procedural complications
Other adverse events (49 terms — click to expand)

ReactionSystemSD-101-6.0 to SD-101-6.0Placebo to SD-101-6.0
Skin infectionInfections and infestations
PyrexiaGeneral disorders
NasopharyngitisInfections and infestations
Wound infectionInfections and infestations
PruritusSkin and subcutaneous tissue disorders
AnaemiaBlood and lymphatic system disorders
ConstipationGastrointestinal disorders
BronchitisInfections and infestations
Upper respiratory tract infectionInfections and infestations
Wound infection bacterialInfections and infestations
Oesophageal stenosisGastrointestinal disorders
Pharyngitis streptococcalInfections and infestations
SinusitisInfections and infestations
Skin bacterial infectionInfections and infestations
Staphylococcal skin infectionInfections and infestations
CoughRespiratory, thoracic and mediastinal disorders
Epidermolysis bullosaCongenital, familial and genetic disorders
Abdominal painGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
Gastrooesophageal reflux diseaseGastrointestinal disorders
VomitingGastrointestinal disorders
CellulitisInfections and infestations
GastroenteritisInfections and infestations
InfluenzaInfections and infestations
Otitis mediaInfections and infestations
Wound infection staphylococcalInfections and infestations
Procedural painInjury, poisoning and procedural complications
WoundInjury, poisoning and procedural complications
AnxietyPsychiatric disorders
LymphadenopathyBlood and lymphatic system disorders
Abdominal pain upperGastrointestinal disorders
HaematemesisGastrointestinal disorders
Oesophageal dilatationGastrointestinal disorders
ToothacheGastrointestinal disorders
PainGeneral disorders
Systemic inflammatory response syndromeGeneral disorders
CystitisInfections and infestations
Eye infectionInfections and infestations
PyodermaInfections and infestations
RhinitisInfections and infestations

Most-reported serious reactions: Anaemia, Oesophageal stenosis, Skin infection, Pericarditis, Congenital megaureter, Keratitis, Abdominal pain, Constipation.

Data from ClinicalTrials.gov NCT02670330 adverse events section.

Sponsor's own description

The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

Verify or expand the search:

Other trials of SD-101-6.0 cream

Trials testing the same drug.

Other recruiting trials for Epidermolysis Bullosa

Currently open trials in the same condition.

Other Scioderm, Inc. trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02670330.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing