Brief Summary
Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP).
Those subjects who pass Screening will enter into the 2-week Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the 2-week Run-in Period will be randomized into active and control groups (2 active to 1 placebo) and enter the 12-week Treatment Period. At each visit during the Treatment Period, the target ulcer will be measured by digital photographic planimetry, the Principal Investigator will assess the wound qualitative attributes, and the DIDP (or placebo patch) will be placed as the primary wound dressing. At each visit the subject will also receive/review a daily diary to document pain , study drug compliance, and analgesic use.
Those subjects who pass Screening will enter into the 2-week Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the 2-week Run-in Period will be randomized into active and control groups (2 active to 1 placebo) and enter the 12-week Treatment Period. At each visit during the Treatment Period, the target ulcer will be measured by digital photographic planimetry, the Principal Investigator will assess the wound qualitative attributes, and the DIDP (or placebo patch) will be placed as the primary wound dressing. At each visit the subject will also receive/review a daily diary to document pain , study drug compliance, and analgesic use.
Brief Title
Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment
Detailed Description
Approximately 60 subjects will be enrolled to allow for up to 48 male or female subjects with SCD and cutaneous ulcers to complete this double-blind, placebo-controlled study.
A sentinel group of 3 subjects will be enrolled and evaluated for safety (while still blinded). The remaining subjects will be enrolled in a 2:1 ratio, active:placebo.
Those subjects who pass Screening will enter into the Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the Run-in Period will be randomized and enter the 12-week Treatment Period.
The DIDP (or placebo) will be replaced daily at home. The subject will visit the clinic on an approximately weekly basis for study assessments, to include imaging and planimetric wound measurements and qualitative wound assessments. Clinical laboratory samples will be collected at Treatment Baseline, Treatment Weeks 4, 8 and 12, or at End of Study (EOS) visit if sooner. A blood sample for PK testing will also be collected at these timepoints. During this Treatment Period, if at any time the wound has met the criterion for 100% healing, the subject will immediately go into the 4-week Follow-up Period.
During the Follow-up Period, the subject will come to the clinic at 1-week intervals. At these visits, the area of the wound will continue to be protected with a protective dressing. Clinical laboratory samples will be collected at the Termination Visit.
Ulcer pain will be assessed by the subject daily and recorded in a diary, along with a record of analgesic use. At each visit, study staff will assist the subjects to assign an overall ulcer pain score for the week.
Quality of Life Assessment will be performed at Baseline prior to dosing and at End of Treatment.
Safety will be assessed based upon known adverse outcomes of Deferoxamine (DFO) therapy. Skin will be examined for evidence of rash and skin irritation.
A sentinel group of 3 subjects will be enrolled and evaluated for safety (while still blinded). The remaining subjects will be enrolled in a 2:1 ratio, active:placebo.
Those subjects who pass Screening will enter into the Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the Run-in Period will be randomized and enter the 12-week Treatment Period.
The DIDP (or placebo) will be replaced daily at home. The subject will visit the clinic on an approximately weekly basis for study assessments, to include imaging and planimetric wound measurements and qualitative wound assessments. Clinical laboratory samples will be collected at Treatment Baseline, Treatment Weeks 4, 8 and 12, or at End of Study (EOS) visit if sooner. A blood sample for PK testing will also be collected at these timepoints. During this Treatment Period, if at any time the wound has met the criterion for 100% healing, the subject will immediately go into the 4-week Follow-up Period.
During the Follow-up Period, the subject will come to the clinic at 1-week intervals. At these visits, the area of the wound will continue to be protected with a protective dressing. Clinical laboratory samples will be collected at the Termination Visit.
Ulcer pain will be assessed by the subject daily and recorded in a diary, along with a record of analgesic use. At each visit, study staff will assist the subjects to assign an overall ulcer pain score for the week.
Quality of Life Assessment will be performed at Baseline prior to dosing and at End of Treatment.
Safety will be assessed based upon known adverse outcomes of Deferoxamine (DFO) therapy. Skin will be examined for evidence of rash and skin irritation.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Chronic Cutaneous Ulcer
Sickle Cell Disease
Eligibility Criteria
Inclusion Criteria:
1. Male or female, \>18 years of age
2. Diagnosed with Sickle Cell Disease (SS, SC, Sß-thalassemia, SD, SOArab)
3. Cutaneous ulcer with a University of Texas Wound Classification System Grade 1A or 2A (= Wagner class 1or 2, without infection or ischemia)
4. Ulcer on lower leg, ankle, or dorsum of foot
5. Vascular status of site assessed and judged adequate for healing (per Section 4.2)
6. Ulcer present ≥ 14 days but not \> 6 months at time of screening
7. Ulcer 2.0 cm2 to 45.0 cm2 and \< 6.0 cm diameter at widest point after debridement, prior to randomization.
8. Ulcer healed by \< 25% during the SOC run-in period prior to Visit 4 randomization.
9. Subject is willing to use acceptable form of birth control (per Section 4.2) during trial and for one month thereafter
Exclusion Criteria:
1. Active infection/purulence at ulcer site, based on Investigator's clinical judgement
2. Current or history of osteomyelitis at or near site of ulcer
3. Serum albumin \< 2.0 g/dL
4. Treatment with systemic DFO within 7 days of study entry
5. Serum ferritin \> 1000 ng/mL
6. Subjects requiring, or expected to require, iron chelation therapy (systemic deferoxamine, deferasirox, or deferiprone) during the duration of the study.
7. Subjects on dialysis or with evidence of nephrotic syndrome.
8. Known bleeding or coagulation disorder that would preclude surgical debridement, as necessary.
9. The subject has a major uncontrolled medical disorder, such as serious cardiovascular, renal, liver, pulmonary disease, or pulmonary disease (per Investigator discretion).
10. Any condition that in the Investigator's opinion would warrant exclusion from the study or prevent the subject from completing the study
11. Previous participation in another clinical trial within 30 days prior to study entry
1. Male or female, \>18 years of age
2. Diagnosed with Sickle Cell Disease (SS, SC, Sß-thalassemia, SD, SOArab)
3. Cutaneous ulcer with a University of Texas Wound Classification System Grade 1A or 2A (= Wagner class 1or 2, without infection or ischemia)
4. Ulcer on lower leg, ankle, or dorsum of foot
5. Vascular status of site assessed and judged adequate for healing (per Section 4.2)
6. Ulcer present ≥ 14 days but not \> 6 months at time of screening
7. Ulcer 2.0 cm2 to 45.0 cm2 and \< 6.0 cm diameter at widest point after debridement, prior to randomization.
8. Ulcer healed by \< 25% during the SOC run-in period prior to Visit 4 randomization.
9. Subject is willing to use acceptable form of birth control (per Section 4.2) during trial and for one month thereafter
Exclusion Criteria:
1. Active infection/purulence at ulcer site, based on Investigator's clinical judgement
2. Current or history of osteomyelitis at or near site of ulcer
3. Serum albumin \< 2.0 g/dL
4. Treatment with systemic DFO within 7 days of study entry
5. Serum ferritin \> 1000 ng/mL
6. Subjects requiring, or expected to require, iron chelation therapy (systemic deferoxamine, deferasirox, or deferiprone) during the duration of the study.
7. Subjects on dialysis or with evidence of nephrotic syndrome.
8. Known bleeding or coagulation disorder that would preclude surgical debridement, as necessary.
9. The subject has a major uncontrolled medical disorder, such as serious cardiovascular, renal, liver, pulmonary disease, or pulmonary disease (per Investigator discretion).
10. Any condition that in the Investigator's opinion would warrant exclusion from the study or prevent the subject from completing the study
11. Previous participation in another clinical trial within 30 days prior to study entry
Inclusion Criteria
Inclusion Criteria:
1. Male or female, \>18 years of age
2. Diagnosed with Sickle Cell Disease (SS, SC, Sß-thalassemia, SD, SOArab)
3. Cutaneous ulcer with a University of Texas Wound Classification System Grade 1A or 2A (= Wagner class 1or 2, without infection or ischemia)
4. Ulcer on lower leg, ankle, or dorsum of foot
5. Vascular status of site assessed and judged adequate for healing (per Section 4.2)
6. Ulcer present ≥ 14 days but not \> 6 months at time of screening
7. Ulcer 2.0 cm2 to 45.0 cm2 and \< 6.0 cm diameter at widest point after debridement, prior to randomization.
8. Ulcer healed by \< 25% during the SOC run-in period prior to Visit 4 randomization.
9. Subject is willing to use acceptable form of birth control (per Section 4.2) during trial and for one month thereafter
1. Male or female, \>18 years of age
2. Diagnosed with Sickle Cell Disease (SS, SC, Sß-thalassemia, SD, SOArab)
3. Cutaneous ulcer with a University of Texas Wound Classification System Grade 1A or 2A (= Wagner class 1or 2, without infection or ischemia)
4. Ulcer on lower leg, ankle, or dorsum of foot
5. Vascular status of site assessed and judged adequate for healing (per Section 4.2)
6. Ulcer present ≥ 14 days but not \> 6 months at time of screening
7. Ulcer 2.0 cm2 to 45.0 cm2 and \< 6.0 cm diameter at widest point after debridement, prior to randomization.
8. Ulcer healed by \< 25% during the SOC run-in period prior to Visit 4 randomization.
9. Subject is willing to use acceptable form of birth control (per Section 4.2) during trial and for one month thereafter
Gender
All
Gender Based
false
Keywords
chronic sickle cell leg ulcer
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT04058197
Org Class
Industry
Org Full Name
TauTona Group
Org Study Id
CD1001
Overall Status
Withdrawn
Phases
Phase 1
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Randomized, Double-Blind, Placebo-Controlled Pilot Study of the Safety and Efficacy of Deferoxamine Intradermal Delivery Patch (DIDP) in Chronic Sickle Cell Leg Ulcers
Primary Outcomes
Outcome Description
Incidence of systemic and local adverse events of DIDP applied to non-healing cutaneous leg ulcers. Changes from baseline characteristics that are treatment-related as measured by physical examinations, clinical laboratory, skin and other physiologic assessments.
Outcome Measure
Incidence of Treatment-Emergent Adverse Events [Safety / Tolerability]
Outcome Time Frame
12 weeks
Secondary Outcomes
Outcome Description
Percentage of wound closure
Outcome Time Frame
12 weeks
Outcome Measure
Degree of Wound closure
Outcome Description
Incidence of 80% closure
Outcome Time Frame
12 weeks
Outcome Measure
Partial wound closure incidence
Outcome Description
Incidence of 100% closure
Outcome Time Frame
12 weeks
Outcome Measure
Total wound closure incidence
Outcome Description
rate of closure
Outcome Time Frame
12 weeks
Outcome Measure
Wound closure rate
Outcome Description
Incidence
Outcome Time Frame
4 week follow-up post
Outcome Measure
Ulcer recurrence
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Roman Zinder
Investigator Email
rzinder@montefiore.org
Investigator Phone