Epidermolysis BullosaPhase 2NCT00311766
A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa
A Phase 2 study testing Thymosin Beta 4 in people with Epidermolysis Bullosa, run by RegeneRx Biopharmaceuticals, Inc..
Trial 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). Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- Thymosin Beta 4
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- Feb 2006to Nov 2012
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.)
About this study
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
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).
Read the 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.
- Conditions
- Epidermolysis Bullosa
- Being tested
- Thymosin Beta 4, Placebo
- Keywords
- Epidermolysis Bullosa, cutaneous wound-healing, chronic wound-healing, Thymosin Beta 4, laminin-5
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thymosin Beta 4Drug
Topical administration, 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days
PlaceboDrug
Topical administration, 0.00% Thymosin Beta 4 gel qd up to 56 days
| Study group | Role | What they receive |
|---|---|---|
| 1 | Placebo comparator | 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 Receives: Drug: Placebo |
| 2 | Active comparator | Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days Receives: Drug: Thymosin Beta 4 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: 70 days
Secondary outcomes (1)
Number of Participants Whose Wounds Have Healed
Wound healing means that the wound has closed without any drainage
Measured: 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2006 (estimated)
- Study startFeb 2006
- Primary completionJul 2010
- Study completionNov 2012
Why it stopped: Lack of patient availability and expiration of study drug
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- RegeneRx Biopharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David R Crockford, Study director, RegeneRx Biopharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00311766
- Sponsor's protocol ID
- SSEB
- Other IDs
- 0003031
- Status last verified
- Apr 2014
- First submitted
- 4 Apr 2006
- Last updated
- 12 May 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00311766
- Acronym / sponsor ID
- SSEB
- Conditions
- Epidermolysis Bullosa
- Keywords
- Epidermolysis Bullosa, cutaneous wound-healing, chronic wound-healing, Thymosin Beta 4, laminin-5
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of patient availability and expiration of study drug
- Enrolment
- 30 participants (actual)
- Lead sponsor
- RegeneRx Biopharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2006
- Primary completion
- Jul 2010 (actual)
- Study completion
- Nov 2012 (actual)
- First posted
- 6 Apr 2006 (estimated)
- Results
- Posted 10 Jun 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Placebo comparator | Drug: PlaceboPlacebo 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 |
| 2 | Active comparator | Drug: Thymosin Beta 4Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
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 |
|---|---|---|
| Thymosin Beta 4Topical administration, 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days | Drug | 2 |
| PlaceboTopical administration, 0.00% Thymosin Beta 4 gel qd up to 56 days | Drug | 1 |
Primary outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 70 days
1 secondary outcome
Number of Participants Whose Wounds Have Healed
Time frame: 56 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2014.
- 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.
David R Crockford study director
RegeneRx Biopharmaceuticals, Inc.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thymosin Beta 4 in people with Epidermolysis Bullosa, run by RegeneRx Biopharmaceuticals, Inc..
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 8 inclusion and 9 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 12 May 2014. Status can change between updates, so confirm with the study contact before making plans.
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 10 Jun 2013). 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 Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: 70 days). 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 12 May 2014.