Trial Outcomes & Findings for Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan (NCT NCT02985710)

Last Updated: 2021-10-07

Results Overview

The internal software of the Sudoscan will allow analysis of skin conductance in all patients and quantification into normal or abnormal. Correlation of an abnormal conductance measurement with clinical symptoms and signs of small fiber neuropathy will be evaluated for accuracy of Sudoscan measurements

Recruitment status

COMPLETED

Study phase

Target enrollment

102 participants

Primary outcome timeframe

12 months

Results posted on

2021-10-07

Participant Flow

Participant milestones

Participant milestones
Measure	Sudoscan Only Patients in this arm will only undergoing testing with Sudoscan. Sudoscan: The Sudoscan is a non invasive procedure and similar to standing on a scale to be weighed. Sudoscan measurements are made in compliance with the manufacturer's recommended procedures. The measurement is rapid and non-invasive, and requires no advance preparation.	Sudoscan Plus Patients in this arm will get Sudoscan testing as well as a skin biopsy and QSART testing. Sudoscan: The Sudoscan is a non invasive procedure and similar to standing on a scale to be weighed. Sudoscan measurements are made in compliance with the manufacturer's recommended procedures. The measurement is rapid and non-invasive, and requires no advance preparation. Skin biopsy: For subjects that give additional consent, skin biopsy will be done in standard fashion under sterile conditions. Assessment of nerve fiber density typically involves a 3-mm punch biopsy of skin from the leg (10 cm above the external malleolus). QSART: QSRT (quantitative sudomotor axon reflex test) testing involves having a technician wipe the subject's arms and leg with alcohol, then tissue to wipe it dry. Electrodes filled with acetylcholine are put on three areas of the leg and one on the wrist, stimulators are turned on and sweat responses are measured.
Overall Study STARTED	69	33
Overall Study COMPLETED	65	33
Overall Study NOT COMPLETED	4	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sudoscan Only n=65 Participants Patients in this arm will only undergoing testing with Sudoscan. Sudoscan: The Sudoscan is a non invasive procedure and similar to standing on a scale to be weighed. Sudoscan measurements are made in compliance with the manufacturer's recommended procedures. The measurement is rapid and non-invasive, and requires no advance preparation.	Sudoscan Plus n=33 Participants Patients in this arm will get Sudoscan testing as well as a skin biopsy and QSART testing. Sudoscan: The Sudoscan is a non invasive procedure and similar to standing on a scale to be weighed. Sudoscan measurements are made in compliance with the manufacturer's recommended procedures. The measurement is rapid and non-invasive, and requires no advance preparation. Skin biopsy: For subjects that give additional consent, skin biopsy will be done in standard fashion under sterile conditions. Assessment of nerve fiber density typically involves a 3-mm punch biopsy of skin from the leg (10 cm above the external malleolus). QSART: QSRT (quantitative sudomotor axon reflex test) testing involves having a technician wipe the subject's arms and leg with alcohol, then tissue to wipe it dry. Electrodes filled with acetylcholine are put on three areas of the leg and one on the wrist, stimulators are turned on and sweat responses are measured.	Total n=98 Participants Total of all reporting groups
Age, Categorical <=18 years	9 Participants n=5 Participants	0 Participants n=7 Participants	9 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	50 Participants n=5 Participants	27 Participants n=7 Participants	77 Participants n=5 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Age, Continuous	42.4 years STANDARD_DEVIATION 17.02 • n=5 Participants	43.6 years STANDARD_DEVIATION 15.35 • n=7 Participants	42.5 years STANDARD_DEVIATION 16.18 • n=5 Participants
Sex: Female, Male Female	45 Participants n=5 Participants	22 Participants n=7 Participants	67 Participants n=5 Participants
Sex: Female, Male Male	20 Participants n=5 Participants	11 Participants n=7 Participants	31 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	64 Participants n=5 Participants	33 Participants n=7 Participants	97 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	65 Participants n=5 Participants	33 Participants n=7 Participants	98 Participants n=5 Participants
Small fiber polyneuropathy questionnaire	51 Participants n=5 Participants	20 Participants n=7 Participants	71 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Sudoscan Only n=65 Participants Patients in this arm will only undergoing testing with Sudoscan. Sudoscan: The Sudoscan is a non invasive procedure and similar to standing on a scale to be weighed. Sudoscan measurements are made in compliance with the manufacturer's recommended procedures. The measurement is rapid and non-invasive, and requires no advance preparation.	Sudoscan Plus n=33 Participants Patients in this arm will get Sudoscan testing as well as a skin biopsy and QSART testing. Sudoscan: The Sudoscan is a non invasive procedure and similar to standing on a scale to be weighed. Sudoscan measurements are made in compliance with the manufacturer's recommended procedures. The measurement is rapid and non-invasive, and requires no advance preparation. Skin biopsy: For subjects that give additional consent, skin biopsy will be done in standard fashion under sterile conditions. Assessment of nerve fiber density typically involves a 3-mm punch biopsy of skin from the leg (10 cm above the external malleolus). QSART: QSRT (quantitative sudomotor axon reflex test) testing involves having a technician wipe the subject's arms and leg with alcohol, then tissue to wipe it dry. Electrodes filled with acetylcholine are put on three areas of the leg and one on the wrist, stimulators are turned on and sweat responses are measured.
Number of Patients With Abnormal Electrochemical Skin Conductance in the Different Study Groups Who Have Clinical Symptoms Consistent With Small Fiber Polyneuropathy.	42 Participants	12 Participants

SECONDARY outcome

Timeframe: 48 months

Distribution of the electrochemical skin conductance (ESC) in both hands and feet was reviewed as an indirect measure of the subject's capacity to sweat. Normative scales of adult sweat function are preloaded in the device and compared to actual measurements to determine if a subject's sweat response is reduced, which is associated with neuropathy. A positive outcome is the identification of a lower ESC in either hands or feet that distinguishes Fabry disease from other disorders and would be a reflection of the presence of SFPN.

Outcome measures

Outcome measures
Measure	Sudoscan Only n=65 Participants Patients in this arm will only undergoing testing with Sudoscan. Sudoscan: The Sudoscan is a non invasive procedure and similar to standing on a scale to be weighed. Sudoscan measurements are made in compliance with the manufacturer's recommended procedures. The measurement is rapid and non-invasive, and requires no advance preparation.	Sudoscan Plus n=33 Participants Patients in this arm will get Sudoscan testing as well as a skin biopsy and QSART testing. Sudoscan: The Sudoscan is a non invasive procedure and similar to standing on a scale to be weighed. Sudoscan measurements are made in compliance with the manufacturer's recommended procedures. The measurement is rapid and non-invasive, and requires no advance preparation. Skin biopsy: For subjects that give additional consent, skin biopsy will be done in standard fashion under sterile conditions. Assessment of nerve fiber density typically involves a 3-mm punch biopsy of skin from the leg (10 cm above the external malleolus). QSART: QSRT (quantitative sudomotor axon reflex test) testing involves having a technician wipe the subject's arms and leg with alcohol, then tissue to wipe it dry. Electrodes filled with acetylcholine are put on three areas of the leg and one on the wrist, stimulators are turned on and sweat responses are measured.
Number of Patients in Whom a Specific Skin Conductance (ESC) Signature Pattern Was Detected in the Hands and/or Feet on Sudoscan Measurements.	21 Participants	16 Participants

Adverse Events

Sudoscan Only

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Sudoscan Plus

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

PI

Massachusetts General Hospital

Phone: 6177260580

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60