Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment (D-SCOUT)
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|ClinicalTrials.gov Identifier: NCT04058197|
Recruitment Status : Not yet recruiting
First Posted : August 15, 2019
Last Update Posted : August 15, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|Chronic Cutaneous Ulcer Sickle Cell Disease||Drug: Deferoxamine Product Other: Placebo||Phase 1 Phase 2|
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. Additional PK samples will be collected for the three sentinel subjects. 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|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|
|Estimated Study Start Date :||August 2019|
|Estimated Primary Completion Date :||December 2020|
|Estimated Study Completion Date :||April 2021|
Deferoxamine (DFO) Intradermal Delivery Patch (DIDP), 45mg DFO daily, up to 12 weeks
Drug: Deferoxamine Product
Deferoxamine Intradermal Delivery Patch
Placebo Comparator: Placebo
- Incidence of Treatment-Emergent Adverse Events [Safety / Tolerability] [ Time Frame: 12 weeks ]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.
- Degree of Wound closure [ Time Frame: 12 weeks ]Percentage of wound closure
- Partial wound closure incidence [ Time Frame: 12 weeks ]Incidence of 80% closure
- Total wound closure incidence [ Time Frame: 12 weeks ]Incidence of 100% closure
- Wound closure rate [ Time Frame: 12 weeks ]rate of closure
- Ulcer recurrence [ Time Frame: 4 week follow-up post ]Incidence
- Ulcer pain: Numeric Pain Rating Scale [ Time Frame: 12 weeks ]Numeric Pain Rating Scale (McCaffery et al, 1989) Scale: 0-10 (0 = no pain, 10 = severe pain)
- Analgesic use [ Time Frame: 12 weeks ]Diary: Opioid analgesic use converted as morphine mg equivalents (MME). Non-steroidal use will be descriptive.
- QOL: Health-related QOL in Chronic Wounds [ Time Frame: 12 weeks ]Wound QOL: Health-related QOL in Chronic Wounds (Augustin et el, 2014; Blome et al, 2014) Response range: "not at all" -to- "very much"
- Pharmacokinetics (blood) [ Time Frame: 12 weeks ]Deferoxamine / Placebo Peak plasma concentration (Cmax)
- Pharmacokinetics (blood) [ Time Frame: 12 weeks ]Deferoxamine / Placebo Time of peak plasma concentration (Tmax)
- Pharmacokinetics (blood) [ Time Frame: 8 hours ]Deferoxamine / Placebo Plasma concentration over 8 hours
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04058197
|Contact: Kenneth Krantz, MD, PhDemail@example.com|
|Contact: Richard Erwin, BSfirstname.lastname@example.org|