What side effects are associated with this type of intervention?

Common treatments for neuropathic pain, such as pregabalin, gabapentin, duloxetine, and tramadol, have several known side effects. Pregabalin and gabapentin can cause dizziness, somnolence, peripheral edema, and weight gain. Duloxetine may lead to nausea, dry mouth, somnolence, and constipation. Tramadol, an opioid, can cause nausea, dizziness, constipation, and the risk of dependency and abuse. These side effects should be discussed with a healthcare provider to ensure the benefits outweigh the risks for each patient.

What prior FDA approvals exist?

Pregabalin, approved by the FDA for the treatment of neuropathic pain, works by modulating calcium channels to reduce neurotransmitter release. Gabapentin, another drug with a similar mechanism, is also FDA-approved for neuropathic pain. Both drugs are used to manage conditions such as diabetic neuropathy and postherpetic neuralgia. Additionally, duloxetine and tramadol have been approved for neuropathic pain, with duloxetine being a serotonin-norepinephrine reuptake inhibitor (SNRI) and tramadol acting as an opioid analgesic with SNRI properties. These medications provide a range of options for managing neuropathic pain through different mechanisms of action.

What other types of research exist?

Promising alternatives to pregabalin for neuropathic pain include gabapentin, which is another anticonvulsant with a similar mechanism of action. Duloxetine and milnacipran, both SNRIs, have shown efficacy in pain relief and may be considered. Additionally, non-pharmacologic treatments such as spinal cord stimulation and acupuncture have shown potential benefits in managing neuropathic pain. These alternatives offer different mechanisms of action and may provide relief for patients who do not respond to pregabalin.

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Gamma-Aminobutyric Acid (GABA) Analog

Pregabalin for Epidermolysis Bullosa

Phase 3

Waitlist Available

Research Sponsored by The Hospital for Sick Children

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age: > 8 - 40 years

Diagnosis of RDEB (by a dermatologist and/or EB specialist and/or genetic confirmation)

Must not have

Ongoing treatment with gabapentin, amitriptyline, duloxetine, nortriptyline, other tricyclics or SNRIs

Medical conditions that would be considered as contraindications for pregabalin treatment (ischemic heart disease, cardiac dysrhythmia, glaucoma, history of urinary retention)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 weeks

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Approved for 10 Other Conditions

All Individual Drugs Already Approved

Summary

This trial tests pregabalin, a medication that calms overactive nerves, on patients with RDEB who suffer from severe pain and itching. Pregabalin is an FDA-approved medication for treating various nerve pain conditions. The goal is to see if pregabalin can effectively reduce these symptoms and improve their quality of life.

Who is the study for?

This trial is for individuals aged 8-40 with RDEB, experiencing neuropathic pain and itch. Participants must have a confirmed diagnosis of RDEB, evidence of neuropathy, and significant daily pain and itch levels. Exclusions include recent Pregabalin or gabapentin use, ongoing treatment with certain antidepressants or SNRIs, specific medical conditions like heart disease or glaucoma, lactose intolerance, allergy to gelatin or the study drug itself.

What is being tested?

The trial tests Pregabalin's effectiveness in managing neuropathic pain and itch in RDEB patients compared to a placebo. Each participant will receive both the medication and placebo at different times (cross-over design), serving as their own control to provide data on dosage, efficacy, safety measures for future larger trials.

What are the potential side effects?

Pregabalin may cause dizziness, sleepiness; some people might experience swelling hands/feet or weight gain. Rarely it can lead to an allergic reaction which could be serious.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 8 and 40 years old.

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I have been diagnosed with RDEB by a specialist or through genetic testing.

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I experience significant numbness or pain due to nerve damage.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently taking medication for nerve pain or depression.

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I do not have heart disease, irregular heartbeat, glaucoma, or a history of urinary retention.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Difference in the mean pain scores between pregabalin and placebo group: VAS

Secondary study objectives

Changes in iscorEB (instrument for scoring clinical outcomes for research of EB), patient portion score in the intervention versus placebo

Interventional procedure

Difference in the average itch score as assessed using VAS between the first and the second treatment periods (period effect).

+5 more

Other study objectives

Correlation between itch score (as measured by VAS),iscorEB patient portion and QOLEB (for patients over 18 yrs) total scores.

Correlation between itch score( as measured by VAS) and iscorEB clinician and Epidermolysis Bullosa Disease Activity and Scarring Index(EBDASI)

Correlation between pain score (as measured by VAS), iscorEB patient portion score and QOLEB (for patients over 18 yrs) total scores.

+3 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Approved for 10 Other Conditions

This treatment demonstrated efficacy for 10 other conditions.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Pregabalin followed by placeboExperimental Treatment1 Intervention

The study has a crossover design. Participants in this arm will receive pregabalin during the first study treatment period for ten weeks and placebo during their second ten-week treatment period . The dose will depend on the participant's weight and phase of treatment period. Each treatment period consists of 4 weeks of escalating dose until the desired maximum , 4 weeks of active treatment and 2 weeks of titrating down.

Group II: Placebo followed by PregabalinExperimental Treatment1 Intervention

Participants will receive placebo during the first treatment period of the study(10 weeks) followed by 10 weeks of pregabalin treatment . The dose will depend on the participant's weight and phase of the treatment period . Each treatment period consists of 3 phases: 4 weeks of escalating dose until the desired maximum, 4 weeks of active treatment and 2 weeks of titrating down.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pregabalin

FDA approved

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for neuropathic pain, such as pregabalin and gabapentin, work by modulating calcium channels to reduce the release of neurotransmitters. This mechanism is important because it directly addresses the abnormal nerve signaling responsible for neuropathic pain, providing relief. These treatments not only alleviate pain but also improve sleep and overall quality of life, which are often significantly impacted in patients with neuropathic pain.

Painful Diabetic Neuropathy: Prevention or Suppression?Transdermal administration of aqueous pregabalin solution as a potential treatment option for patients with neuropathic pain to avoid central nervous system-mediated side effects.