Epidermolysis Bullosa
Conditions
Keywords
Epidermolysis Bullosa, cutaneous wound-healing, chronic wound-healing, Thymosin Beta 4, laminin-5
Brief summary
The purpose of this study is to investigate a treatment to enhance the healing of acute and chronic nonhealing cutaneous wounds, such as the erosions experienced by patients with Epidermolysis Bullosa (EB), by the known activity of thymosin beta 4 (Tβ4). Funding Source - FDA Office of Orphan Product Development (OOPD).
Detailed description
EB is a group of genetic diseases characterized by skin-blistering and lesion-formation after minor trauma to the skin. This family of disorders, most of which are inherited, range in severity from mild to the severely disabling and life-threatening. Tβ4 is a synthetically-produced copy of a naturally-occurring 43 amino acid peptide that has wound healing and anti-inflammatory properties and can up-regulate the expression of laminin-5.
Interventions
DRUGThymosin Beta 4
Topical administration, 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days
DRUGPlacebo
Topical administration, 0.00% Thymosin Beta 4 gel qd up to 56 days
Sponsors
RegeneRx Biopharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Informed Consent form signed by the patient or patient's legal representative; also, if the patient is under the age of majority but capable of providing assent, signed assent from the patient * Diagnosis of junctional or dystrophic EB. * Patients who present with Hallopeau-Siemens subtype may be enrolled. * At least one active, unroofed EB erosion on the limb or on the trunk. * Lesion size 5 to 50 cm2, inclusive. * Stable lesion present for 14-60 days before enrollment. * More that one member in a family can be enrolled as long as that member is treated to a different cohort with an assurance that the study medication will not be shared. * No clinically significant abnormalities (Grade 2 or higher on the National Cancer Institute \[NCI\] toxicity scale) on Screening laboratory tests, except for the following specific laboratory threshold result: albumin must be 2 g/dL or higher; hemoglobin must be 8 g/dL or higher.
Exclusion criteria
* Clinical evidence of local infection of the index (targeted) lesion. * Use of any investigational drug within the 30 days before enrollment. * Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment. * Use of systemic or topical steroidal therapy within the 30 days before enrollment.Inhaled steroids are allowed. * Use of systemic antibiotics within the 7 days before enrollment. * Current or former malignancy. * Arterial or venous disorder resulting in ulcerated wounds. * Diabetes mellitus. * Pregnancy or breastfeeding during the study. (A serum pregnancy test will be performed at Screening for female patients of childbearing potential.)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | 70 days |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Whose Wounds Have Healed | 56 days | Wound healing means that the wound has closed without any drainage |
Participant flow
Recruitment details
First Patient In:22 February 2006; Last Patient In: 6 July 2010 Locations: Medical Centers
Pre-assignment details
No wash out or run-in periods in this study
Participants by arm
| Arm | Count |
|---|---|
| Placebo Topical administration of placebo gel, 0% Thymosin Beta 4, qd up to 56 days
Placebo : Topical administration, 0.00% qd up to 56 days | 8 |
| Thymosin Beta 4 Topical administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days
Thymosin Beta 4 : Topical administration, 0.01%, 0.03%, and 0.1% gel, qd up to 56 days | 22 |
| Total | 30 |
Baseline characteristics
| Characteristic | Placebo | Thymosin Beta 4 | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 4 Participants | 12 Participants | 16 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 4 Participants | 10 Participants | 14 Participants |
| Region of Enrollment United States | 8 participants | 22 participants | 30 participants |
| Sex: Female, Male Female | 2 Participants | 8 Participants | 10 Participants |
| Sex: Female, Male Male | 6 Participants | 14 Participants | 20 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 8 | 1 / 22 |
| serious Total, serious adverse events | 0 / 8 | 0 / 22 |
Outcome results
Primary
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 70 days
Population: The population for safety analysis included all patients who were randomized and received at least one dose of study medication and had at one safety parameter recorded.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Placebo | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Adverse Events | 0 Participants |
| Placebo | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Serious Adverse Events | 0 Participants |
| Thymosin Beta 4 | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Adverse Events | 1 Participants |
| Thymosin Beta 4 | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Serious Adverse Events | 0 Participants |
Secondary
Number of Participants Whose Wounds Have Healed
Wound healing means that the wound has closed without any drainage
Time frame: 56 days
Population: The population for efficacy analysis will be the Full Analysis (FA) population. The FA analysis included all patients who were randomized and received at least one dose of study medication and who had at least one baseline efficacy parameter recorded.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Number of Participants Whose Wounds Have Healed | 5 participants |
| Thymosin Beta 4 | Number of Participants Whose Wounds Have Healed | 8 participants |
Comparison: The primary population was the Full Analysis(FA)population. The FA population included all patients who were randomized and received at least one dose of study medication and who had at least one baseline efficacy parameter recordedp-value: >0.05Fisher Exact