Iontophoresis of Treprostinil to Enhance Wound Healing in Diabetic Foot Skin Ulcers

NCT ID: NCT03654989

Last Updated: 2023-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-01-28

Study Completion Date

2021-12-07

Brief Summary

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Assess the effect of iontophoresis of treprostinil on wound closure over 12 weeks, in patients with DFU.

In the present study the investigators aim at establishing the proof-of-concept of iontophoresis of treprostinil as a potential treatment of diabetic foot ulcers in humans. The main hypothesis is that in patients with DFUs, the pharmacodynamic effect of a PGI2 analogue potentiates the effect of low-intensity current on microvascular function, tissue oxygenation and healing.

Detailed Description

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Diabetic foot ulcers (DFUs) represent a serious public health problem associated with significant morbidity and health costs. Despite optimal etiologic treatment and local care, amputation is frequent, stressing the need for new treatments. Tissue ischemia is the primary cause for nonhealing DFUs. The cutaneous microcirculation, by providing tissue perfusion, fluid hemostasis, and delivery of oxygen and nutrients, plays a critical role in the pathophysiology and impaired healing of DFUs.

The investigators therefore hypothesize that the skin microcirculation, and more specifically the prostacyclin (PGI2) pathway, is an interesting target for the local treatment of DFUs. Indeed, besides its potent vasodilator effect, PGI2 plays a role in the promotion of fibroblast migration and angiogenesis in wound models.

However, the benefit of systemic (i.e. IV or SC) treatments is counterbalanced by potentially serious vasodilatation-induced side effects (e.g. severe headaches, flushing, tachycardia and hypotension). These properties are dose-limiting and are associated with safety issues and increased costs. The paradox is that impaired microvasculature prevents the drug, when administered intravenously, from diffusing properly to the wound. Elevated doses are therefore needed, leading to adverse drug reactions.

The originality of this approach is to locally deliver negatively charged PGI2 analogues into and around the wound under the influence of a low-intensity current, through a method called iontophoresis. Iontophoresis enables a controlled delivery of ionized drugs into/through the skin under the influence of low-intensity current. In addition, endogenous electrical signals in the wound are known to play a role in healing, by increasing the directed migration of keratinocytes, fibroblasts and neutrophils. Exogenous electric stimulation would mimic this phenomenon with a positive impact on wound healing. In summary, both the drug and its vehicle could therefore work synergistically, while delivering the drug locally therefore limiting side effects due to systemic diffusion.

This is a prospective, monocentric, controlled, randomized, double-blinded phase I/II study The main objective is to assess the effect of iontophoresis of treprostinil on wound closure over 12 weeks, in patients with DFU. The investigators will compare wound closure, expressed as the percentage change of the wound area over time (12-week follow-up), between 3 groups: iontophoresis of treprostinil, iontophoresis of placebo, and standard of care. Wound area will be assessed with a digital camera and image analysis software.

Secondary objectives are:

* To assess the effect of iontophoresis of treprostinil on complete healing over 3 months
* To assess the effect of iontophoresis of treprostinil on the time to complete healing
* To assess the effect of iontophoresis of treprostinil on skin perfusion at the site of the ulcer and around the wound
* To evaluate the effect of iontophoresis of treprostinil on skin oxygenation around the lesion and on healed skin
* To assess the pharmacokinetics (PK) of topical administration of treprostinil over damaged (wounded)
* To evaluate the safety of the procedure

The study will be divided into two consecutive parts:

Part 1: 8 to 24 patients with DFU (depending on the total number of doses tested, and the number of doses per patient) will be included in a single ascending dose (SAD) safety study of treprostinil iontophoresis.

Part 2: 36 patients with DFU associated with microvascular dysfunction (+/- neuropathy) will be randomized into three groups to receive either: 1. Treprostinil iontophoresis; 2. Placebo iontophoresis; 3. Standard care. Drug administration (placebo or treprostinil), but not standard care, will be double-blind. After a 10-day treatment, follow-up includes 6 visits over 10 weeks.

Conditions

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Diabetic Foot Ulcer

Keywords

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Iontophoresis Treprostinil Diabetic Foot Ulcer Wound Healing

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

2 sub-studies:

* Preliminary safety study, Phase I: single ascending dose study that aims at establishing the safety, tolerability, and PK of the procedure in patients with active ulcers; we propose an accelerated titration design with 7 doses of gel. The accelerated phase (first four doses) uses single-patient cohorts per dose. After these doses, a standard 3+3 design will be performed. Any occurrence of dose-limiting toxicity (DLT) during the accelerated phase halts the accelerated titration and the cohort is expended to the standard 3+3 design. Two instances of DLT at a dose level halt escalation, and D-1 is the maximum tolerated dose.
* Proof-of-concept study, Phase II: Thirty-six patients with DFU associated with microvascular dysfunction will be randomized into three groups: Treprostinil iontophoresis; Placebo iontophoresis; Standard care. Drug administration, but not standard care, will be double-blind. After a 10-day treatment, follow-up includes 6 visits over 10 weeks.
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Outcome Assessors
Double blind (for the two groups treated with iontophoresis of treprostinil or placebo); open for the standard of care group.

The preparation of syringes of gel containing treprostinil or placebo will be centralized. Both gels will be physically identical, and investigators will not have access to the randomization list or to preparation records. This ensures proper blinding at treatment initiation. Treatment kits will subsequently be given to research nurses to continue the treatment at home. Nurses will not have access to the randomization list or preparation records either, which guarantees proper blinding throughout the 10-day treatment.

Unblinding will be done in case of any suspicion of an unexpected serious adverse reaction, prior to the declaration of the event to the competent authorities. Unblinding may be done 24/24h by the Pharmacy department.

Study Groups

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Treprostinil iontophoresis

Gel of treprostinil 1 mg/mL (target concentration)

* Part 1: 1 administration/day, on separate days, with 72h between two doses. Ascending doses are 0.025 mg/mL, 0.05mg/mL, 0.1 mg/mL, 0.25 mg/mL, 0.5 mg/mL, 0.7 mg/mL, and 1 mg/mL. The intensity will be set at 120 µA during 60 minutes, i.e. a total current of 17.3 mC/cm².
* Part 2: 1 administration/day at the maximum tolerated dose (MTD) for 10 days; dressing will be changed by a trained nurse every 2 days. The intensity will be set at 120 µA during 60 minutes, i.e. a total current of 17.3 mC/cm².

Group Type EXPERIMENTAL

Treprostinil iontophoresis

Intervention Type DRUG

We will administer treprostinil at increasing doses by a iontophoresis.

Remodulin® Placebo iontophoresis

Placebo will be made from the placebo of Remodulin® incorporated into hydrogel (Suprasorb® G). The route and frequency of administration will be the same as for the investigational drug (topical administration by iontophoresis).

Group Type PLACEBO_COMPARATOR

Remodulin® placebo iontophoresis

Intervention Type DEVICE

Placebo iontophoresis will be performed using Remodulin® placebo (United Therapeutics) delivered with Axion GmbH electrodes connected to a PeriIont generator (Perimed).

* Part 1: 1 administration/day, on separate days, with 72h between two doses. The intensity will be set at 120 µA during 60 minutes, i.e. a total current of 17.3 mC/cm².
* Part 2: 1 administration/day for 10 days. The intensity will be set at 120 µA during 60 minutes, i.e. a total current of 17.3 mC/cm².

Standard care

subjects randomized to the standard of care group (no iontophoresis) will only undergo standard blood test at visit 0 or 1, unless tests \<1 month before inclusion are available This group is not double blind Standard care consists on debridement and dressings

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Treprostinil iontophoresis

We will administer treprostinil at increasing doses by a iontophoresis.

Intervention Type DRUG

Remodulin® placebo iontophoresis

Placebo iontophoresis will be performed using Remodulin® placebo (United Therapeutics) delivered with Axion GmbH electrodes connected to a PeriIont generator (Perimed).

* Part 1: 1 administration/day, on separate days, with 72h between two doses. The intensity will be set at 120 µA during 60 minutes, i.e. a total current of 17.3 mC/cm².
* Part 2: 1 administration/day for 10 days. The intensity will be set at 120 µA during 60 minutes, i.e. a total current of 17.3 mC/cm².

Intervention Type DEVICE

Eligibility Criteria

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Inclusion Criteria

* Patients with type 2 diabetes according to the criteria of the American Diabetes Association (ADA), with one or more foot ulcer of microvascular or mixed etiology:

* The ulcer size must be ≥1 cm² and \<20 cm²
* Grade 1A, 1C, 2A or 2C (University of Texas Classification of Diabetic Foot)
* Patient affiliated to social security insurance or beneficiary of social security insurance.

Exclusion Criteria

* History of hypersensitivity reaction to treprostinil
* Pulmonary veno-occlusive disease (PVOD)
* Systemic treatment with any PGI2 analogue in the past two months.
* Critical ischemia of the lower limb, defined as leg pain at rest associated with ankle pressure \<50 mmHg.
* Infected wound, treated with antibiotics in the past 15 days.
* Active or uncontrolled cardiovascular disease as follows:
* Myocardial infarction, or angina within 6 months of study participation
* Arrhythmia (uncontrolled, highly symptomatic, requires treatment or life-threatening).
* Congestive heart failure.
* Stroke or transient ischemic attack within 3 months of study participation
* Uncontrolled hypertension: systolic blood pressure\> 180 mmHg or diastolic blood pressure\> 105 mmHg (2 abnormal readings during visit)
* Valvular heart disease
* Severe liver disease (Child-Pugh C) at the time of enrollment
* Active gastroduodenal ulcer
* Intracerebral hemorrhage
* Trauma or any clinical event susceptible to be responsible for hemorrhage within 6 months of study participation
* Renal disease (creatinine \> 2 mg/dL and/or estimated glomerular filtration rate\<30 mL/min, history of dialysis)
* Unstable diabetes that has resulted in hyperosmolar coma or ketoacidosis, and/or documented increase or decrease in HbA1c of more than 2.0% within the previous 3 months.
* Pregnancy or Lactation
* Females of childbearing potential not using an effective form of birth control as determined by the investigators.
* Participant involved in another interventional clinical study
* Person deprived of liberty by judicial order
* Person under guardianship or curatorship
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Grenoble

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cracowski Jean-luc, Professor

Role: PRINCIPAL_INVESTIGATOR

Clinical pharmacology unit, grenoble alpes university hospital

Locations

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CHU Grenoble Alpes

Grenoble, , France

Site Status

Countries

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France

Other Identifiers

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38RC17.163

Identifier Type: -

Identifier Source: org_study_id