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A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa

Primary Purpose

Epidermolysis Bullosa

Status
Terminated
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Thymosin Beta 4
Placebo
Sponsored by
RegeneRx Biopharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Epidermolysis Bullosa focused on measuring Epidermolysis Bullosa, cutaneous wound-healing, chronic wound-healing, Thymosin Beta 4, laminin-5

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.)

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Placebo Comparator

    Active Comparator

    Arm Label

    1

    2

    Arm Description

    Placebo comparator gel does not contain any active drug. Topical administration of 0.0% Thymosin Beta 4 gel, once a day (qd) up to 56 days

    Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days

    Outcomes

    Primary Outcome Measures

    Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Secondary Outcome Measures

    Number of Participants Whose Wounds Have Healed
    Wound healing means that the wound has closed without any drainage

    Full Information

    First Posted
    April 4, 2006
    Last Updated
    April 25, 2014
    Sponsor
    RegeneRx Biopharmaceuticals, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00311766
    Brief Title
    A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa
    Official Title
    A Randomized, Double-Blind, Placebo-Controlled, Dose-Response Study of the Safety and Efficacy of Thymosin Beta 4 in the Treatment of Patients With Epidermolysis Bullosa
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2014
    Overall Recruitment Status
    Terminated
    Why Stopped
    Lack of patient availability and expiration of study drug
    Study Start Date
    February 2006 (undefined)
    Primary Completion Date
    July 2010 (Actual)
    Study Completion Date
    November 2012 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    RegeneRx Biopharmaceuticals, Inc.

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Epidermolysis Bullosa
    Keywords
    Epidermolysis Bullosa, cutaneous wound-healing, chronic wound-healing, Thymosin Beta 4, laminin-5

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Parallel Assignment
    Masking
    ParticipantCare ProviderInvestigatorOutcomes Assessor
    Allocation
    Randomized
    Enrollment
    30 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    1
    Arm Type
    Placebo Comparator
    Arm Description
    Placebo comparator gel does not contain any active drug. Topical administration of 0.0% Thymosin Beta 4 gel, once a day (qd) up to 56 days
    Arm Title
    2
    Arm Type
    Active Comparator
    Arm Description
    Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days
    Intervention Type
    Drug
    Intervention Name(s)
    Thymosin Beta 4
    Intervention Description
    Topical administration, 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days
    Intervention Type
    Drug
    Intervention Name(s)
    Placebo
    Intervention Description
    Topical administration, 0.00% Thymosin Beta 4 gel qd up to 56 days
    Primary Outcome Measure Information:
    Title
    Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
    Time Frame
    70 days
    Secondary Outcome Measure Information:
    Title
    Number of Participants Whose Wounds Have Healed
    Description
    Wound healing means that the wound has closed without any drainage
    Time Frame
    56 days

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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.)
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    David R Crockford
    Organizational Affiliation
    RegeneRx Biopharmaceuticals, Inc.
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Learn more about this trial

    A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa

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