Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment

NCT04058197 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: CD1001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 4

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.

Conditions

Chronic Cutaneous Ulcer; Sickle Cell Disease

Keywords

chronic sickle cell leg ulcer

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events [Safety / Tolerability]

  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.

  12 weeks

Secondary Outcomes (5)

• Degree of Wound closure

  12 weeks

• Partial wound closure incidence

  12 weeks

• Total wound closure incidence

  12 weeks

• Wound closure rate

  12 weeks

• Ulcer recurrence

  4 week follow-up post

Other Outcomes (6)

• Ulcer pain: Numeric Pain Rating Scale

  12 weeks

• Analgesic use

  12 weeks

• QOL: Health-related QOL in Chronic Wounds

  12 weeks

Study Arms (2)

Active

EXPERIMENTAL

Deferoxamine (DFO) Intradermal Delivery Patch (DIDP), 45mg DFO daily, up to 12 weeks

Drug: Deferoxamine Product

Placebo

PLACEBO COMPARATOR

Placebo

Other: Placebo

Interventions

Deferoxamine Product DRUG

Deferoxamine Intradermal Delivery Patch

Active

Placebo OTHER

Placebo Patch

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, \>18 years of age
• Diagnosed with Sickle Cell Disease (SS, SC, Sß-thalassemia, SD, SOArab)
• Cutaneous ulcer with a University of Texas Wound Classification System Grade 1A or 2A (= Wagner class 1or 2, without infection or ischemia)
• Ulcer on lower leg, ankle, or dorsum of foot
• Vascular status of site assessed and judged adequate for healing (per Section 4.2)
• Ulcer present ≥ 14 days but not \> 6 months at time of screening
• Ulcer 2.0 cm2 to 45.0 cm2 and \< 6.0 cm diameter at widest point after debridement, prior to randomization.
• Ulcer healed by \< 25% during the SOC run-in period prior to Visit 4 randomization.
• Subject is willing to use acceptable form of birth control (per Section 4.2) during trial and for one month thereafter

You may not qualify if:

• Active infection/purulence at ulcer site, based on Investigator's clinical judgement
• Current or history of osteomyelitis at or near site of ulcer
• Serum albumin \< 2.0 g/dL
• Treatment with systemic DFO within 7 days of study entry
• Serum ferritin \> 1000 ng/mL
• Subjects requiring, or expected to require, iron chelation therapy (systemic deferoxamine, deferasirox, or deferiprone) during the duration of the study.
• Subjects on dialysis or with evidence of nephrotic syndrome.
• Known bleeding or coagulation disorder that would preclude surgical debridement, as necessary.
• The subject has a major uncontrolled medical disorder, such as serious cardiovascular, renal, liver, pulmonary disease, or pulmonary disease (per Investigator discretion).
• Previous participation in another clinical trial within 30 days prior to study entry

Sponsors & Collaborators

• TauTona Group: lead

Study Sites (4)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Sonar Clinical Research LLC

Atlanta, Georgia, 30315, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 18, 2019
• First Posted: August 15, 2019
• Study Start: July 21, 2020
• Primary Completion: October 6, 2021
• Study Completion: October 6, 2021
• Last Updated: December 2, 2021

Record last verified: 2020-11

Locations

US (4)