Idiopathic Small Fiber NeuropathyPhase 2NCT02607254

Efficacy and Safety of Pregabalin in Treatment of Neuropathic Pain in Patients With Idiopathic Small Fiber Neuropathy

A Phase 2 study testing Pregabalin in people with Idiopathic Small Fiber Neuropathy, run by Johns Hopkins University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study is to assess safety and efficacy of treatment with pregabalin in patients with idiopathic small fiber neuropathy proven by skin biopsy.This is an enriched enrollment randomized withdrawal study that comprises 4 phases: a screening and selection phase, a washout period from previous pain… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Idiopathic Small Fiber Neuropathy
What is being tested?: Pregabalin
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 11 participants
Where: 1 siteUnited States
Study dates: Sep 2015to Jul 2018
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Subjects with idiopathic predominate-small fiber neuropathy
Subject must have chronic peripheral neuropathic pain for more than 3 months
A score >3 and <8 on Pain intensity scale for pain in prior week at first visit;
Show increase in pain intensity scores during the wash off period;
Age older than 18 years;

Who cannot join

Subjects with large-fiber predominant neuropathy
Subjects with HIV infection, trigeminal neuralgia (TGN), toxic neuropathy (e.g. chemotherapy exposure), paraneoplastic neuropathy, mono-gammopathy, inflammatory neuropathy, celiac disease, systemic lupus, peripheral vascular disease, connective tissue disorders, hepatitis C, Fabry disease, and diabetes;
Subjects with uncontrolled thyroid or B12 disorders
Subjects with Complex Regional Pain Syndrome
Allergy to Pregabalin
Subjects at risk of suicide or self harm

Show all 9Show fewer

Subjects with any clinically unstable cardiovascular, hematological, autoimmune, endocrine, renal, hepatic, renal, respiratory, or gastrointestinal disease; epilepsy, symptomatic peripheral vascular disease including intermittent claudication, pernicious anemia, untreated hypothyroidism, venous insufficiency, or spinal stenosis.
History of known analgesic, alcohol or illicit drug abuse within 12 months of first visit;
Pregnant females; breastfeeding females.

Trial locations and contacts

1 site in 1 country.

United States1 site

Johns Hopkins Hospital
Baltimore, Maryland, 21287

Other trials in United States

About this study

Read the detailed description

Patients who show any increase in their pain intensity score during the washout phase will be eligible for the single blind pregabalin treatment phase. Patients who respond to pregabalin, with at least 1 point improvement in neuropathic pain from baseline at the end of the single blind pregabalin treatment phase and meeting all other study requirements are considered eligible for participation in the withdrawal phase. These eligible patients are randomly assigned (1:1) to continue pregabalin or to be switched to placebo for a comparison of pregabalin efficacy and safety. Patients who have a worsening of average pain relative to the last week of the single blind pregabalin treatment period by more than one point and average pain level > =4 will be considered to have a loss of therapeutic response (LTR).

Conditions: Idiopathic Small Fiber Neuropathy
Being tested: Pregabalin, Placebo

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PregabalinDrug
Also called Lyrica
Pregabalin will be given to the patients, starting at 75 mg BID and increased weekly to reach 225 mg BID for 8 weeks. During withdrawal phase, dose of pregabalin will be kept the same for patients who are randomized to the pregabalin withdrawal group.
PlaceboDrug
Placebo will be given to the patients that are randomized to placebo during withdrawal phase.

Study groups in NCT02607254
Study group	Role	What they receive
Pregabalin Treatment phase	Experimental	All patients will be initially treated with pregabalin in a single blind fashion Receives: Drug: Pregabalin
Withdrawal phase	Experimental	After finishing the treatment phase, some patients will be randomized to the placebo or continue on pregabalin for the 4 weeks of withdrawal phase. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Visual Analogue Score for Pain Intensity.
The primary outcome is change in visual analogue score for pain, with 0 being no pain at all and 10 being the most severe pain and a higher score meaning more pain, after 8 weeks of treatment phase and 4 weeks of randomized withdrawal phase.
Measured: Baseline, at 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase

Secondary outcomes (3)

Brief Pain Inventory (BPI sf);
Brief pain inventory is a scoring system for average pain intensity on scale of 0-10 with higher number meaning more pain.
Measured: Baseline, at 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase
Sleep Quality as Assessed by Daily Sleep Interference Rating Scale (SIRS);
a scoring system describing sleep quality between 0-10 with 0 having no problem with sleep and 10 not being to sleep at all.
Measured: Baseline, at 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase
Patient Global Impression of Change (PGIC);
patient assign a number between 1-7 to the level of improvement, 1 showing substantial improvement and 7 showing no improvement at all.
Measured: At 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2015Jul 2018

First posted18 Nov 2015 (estimated)
Study startSep 2015
Primary completionJul 2018
Study completionJul 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Idiopathic Small Fiber NeuropathyStudies of the same condition, open and under way.
More trials run by Johns Hopkins UniversityThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Single blind
Who is masked: Participant
Enrolment: 11 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02607254
Sponsor's protocol ID: IRB00054498
Status last verified: Oct 2018
First submitted: 16 Nov 2015
Last updated: 4 Oct 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Study at a glance

From the registry record, last updated by the sponsor on 4 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Efficacy and Safety of Pregabalin in Treatment of Neuropathic Pain in Patients With Idiopathic Small Fiber Neuropathy
ClinicalTrials.gov ID: NCT02607254
Acronym / sponsor ID: IRB00054498
Conditions: Idiopathic Small Fiber Neuropathy
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 11 participants (actual)
Lead sponsor: Johns Hopkins UniversityUniversity, hospital or other
Collaborators: Pfizer
Study start: Sep 2015
Primary completion: Jul 2018 (actual)
Study completion: Jul 2018 (actual)
First posted: 18 Nov 2015 (estimated)
Results: Posted 4 Oct 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Single blind (participant)
Number of arms: 2

Arm	Type	Receives
Pregabalin Treatment phase	Experimental	Drug: PregabalinAll patients will be initially treated with pregabalin in a single blind fashion
Withdrawal phase	Experimental	Drug: PlaceboAfter finishing the treatment phase, some patients will be randomized to the placebo or continue on pregabalin for the 4 weeks of withdrawal phase.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Subjects with idiopathic predominate-small fiber neuropathy
Subject must have chronic peripheral neuropathic pain for more than 3 months
A score >3 and <8 on Pain intensity scale for pain in prior week at first visit;
Show increase in pain intensity scores during the wash off period;
Age older than 18 years;

Key exclusion criteria (9 listed)

Subjects with large-fiber predominant neuropathy
Subjects with HIV infection, trigeminal neuralgia (TGN), toxic neuropathy (e.g. chemotherapy exposure), paraneoplastic neuropathy, mono-gammopathy, inflammatory neuropathy, celiac disease, systemic lupus, peripheral vascular disease, connective tissue disorders, hepatitis C, Fabry disease, and diabetes;
Subjects with uncontrolled thyroid or B12 disorders
Subjects with Complex Regional Pain Syndrome
Allergy to Pregabalin

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
PregabalinAlso: LyricaPregabalin will be given to the patients, starting at 75 mg BID and increased weekly to reach 225 mg BID for 8 weeks. During withdrawal phase, dose of pregabalin will be kept the same for patients who are randomized to the pregabalin withdrawal group.	Drug	Pregabalin Treatment phase
PlaceboPlacebo will be given to the patients that are randomized to placebo during withdrawal phase.	Drug	Withdrawal phase

Primary outcome

Visual Analogue Score for Pain Intensity.
Time frame: Baseline, at 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase
The primary outcome is change in visual analogue score for pain, with 0 being no pain at all and 10 being the most severe pain and a higher score meaning more pain, after 8 weeks of treatment phase and 4 weeks of randomized withdrawal phase.

3 secondary outcomes

Brief Pain Inventory (BPI sf);
Time frame: Baseline, at 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase
Sleep Quality as Assessed by Daily Sleep Interference Rating Scale (SIRS);
Time frame: Baseline, at 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase
Patient Global Impression of Change (PGIC);
Time frame: At 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase

Results and safety

Results posted to the registry on 4 Oct 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Pregabalin Treatment/Pregabalin Withdrawal	3	3
Pregabalin Treatment/ Placebo Withdrawal	3	3
Pregabalin Treatment/No Withdrwal	5	5

Primary outcome: Visual Analogue Score for Pain Intensity.

Time frame: Baseline, at 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase. Mean (Standard Deviation). Unit: score on visual analogue score

Measure	Pregabalin Treatment/Pregabalin Withdrawal n = 3	Pregabalin Treatment/ Placebo Withdrawal n = 3	Pregabalin Treatment/No Withdrwal n = 5
Basline	7.1 (1.4)	6.9 (1.7)	7.3 (2.3)
After 8 weeks of Pregabalin	5.4 (0.8)	5.1 (1.1)	7.6 (2.9)
After 4 weeks of Withdrawal phase	6.1 (1.2)	7.4 (1.8)	NA (NA)

Population analysed: Patient with idiopathic small fiber neuropathy.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: 3 months. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Pregabalin Treatment/Pregabalin Withdrawal	0 of 3 (0%)	0 of 3 (0%)	0 of 3
Pregabalin Treatment/ Placebo Withdrawal	0 of 3 (0%)	0 of 3 (0%)	0 of 3
Pregabalin Treatment/No Withdrawal	0 of 5 (0%)	0 of 5 (0%)	0 of 5

Limitations and caveats (from the sponsor): There were low number of participants due to requirements of the study.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Mohammad Khoshnoodi, MD principal investigator

Johns Hopkins University

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pregabalin in people with Idiopathic Small Fiber Neuropathy, run by Johns Hopkins University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 Oct 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Visual Analogue Score for Pain Intensity. (time frame: Baseline, at 8 weeks after treatment phase and at 12 weeks for subjects completing the withdrawal phase). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02607254.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Oct 2018.