Trial Outcomes & Findings for Intranasal Fentanyl for Initial Treatment of a Vaso-occlusive Crisis (NCT NCT01482091)

NCT ID: NCT01482091

Last Updated: 2016-08-19

Results Overview

Change in pain score between 0 and 20 minutes using the Wong Baker FACES pain scale (WBFPS). The WBFPRS has six faces, with each face representing an increasing severity of pain the more rightward it is on the scale (0 is the lowest score , which represents the least amount of pain, while 10 is the highest score which represents the greatest level of pain).. Each face has an even number underneath it, consecutively. To calculate the change, the reported pain score at 20 minutes was subtracted from the reported baseline pain score. Thus, the higher change in pain score is indicative of a GREATER change in pain (i.e. greater decrease in pain at 20 minutes compared to baseline). The greatest possible changes in pain would be a 10 (pain score of 10 at baseline and 0 at 20 minutes) representing a DECREASE in pain between the two time points, and -10 (pain score of 0 at baseline and 10 at 20 minutes) representing a INCREASE in pain between the two time points.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 124 participants
• Primary outcome timeframe: Baseline and 20 minutes after administration of study drug
• Results posted on: 2016-08-19

Participant Flow

Subjects were PRE-CONSENTED for the study in the outpatient hematology clinic, inpatient floors prior to discharge or a separate emergency department (ED) visit prior to discharge. This was done to prevent decision making while under emotional duress of a vaso-occlusive crisis (VOC). PRE-CONSENT occurred from 12.12.2011-1.8.2015

124 subjects were consented for the study. The study drug was only administered one time in the ED if subjects met all inclusion and exclusion criteria when they arrived with a VOC. Only 49 of the 124 consented subjects received the study drug.

Participant milestones

Measure	Intranasal Saline Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Overall Study STARTED	25	24
Overall Study COMPLETED	25	24
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Intranasal Fentanyl for Initial Treatment of a Vaso-occlusive Crisis

Baseline characteristics by cohort

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device	Total n=49 Participants Total of all reporting groups
Age, Continuous	12.5 years STANDARD_DEVIATION 5.1 • n=5 Participants	10.6 years STANDARD_DEVIATION 5.3 • n=7 Participants	11.6 years STANDARD_DEVIATION 5.2 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	13 Participants n=7 Participants	19 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants	11 Participants n=7 Participants	30 Participants n=5 Participants
weight	45 kg n=5 Participants	28 kg n=7 Participants	37 kg n=5 Participants
Type of Sickle Cell Disease SS	19 participants n=5 Participants	20 participants n=7 Participants	39 participants n=5 Participants
Type of Sickle Cell Disease SC	6 participants n=5 Participants	3 participants n=7 Participants	9 participants n=5 Participants
Type of Sickle Cell Disease SB	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Duration of VOC <1/2 day	7 participants n=5 Participants	9 participants n=7 Participants	16 participants n=5 Participants
Duration of VOC 1/2-1day	5 participants n=5 Participants	7 participants n=7 Participants	12 participants n=5 Participants
Duration of VOC 1-2 day	6 participants n=5 Participants	4 participants n=7 Participants	10 participants n=5 Participants
Duration of VOC >2 day	7 participants n=5 Participants	4 participants n=7 Participants	11 participants n=5 Participants
Location of VOC Abdomen	6 participants n=5 Participants	10 participants n=7 Participants	16 participants n=5 Participants
Location of VOC Back	12 participants n=5 Participants	12 participants n=7 Participants	24 participants n=5 Participants
Location of VOC Chest	4 participants n=5 Participants	7 participants n=7 Participants	11 participants n=5 Participants
Location of VOC Head	1 participants n=5 Participants	4 participants n=7 Participants	5 participants n=5 Participants
Location of VOC Arms	5 participants n=5 Participants	9 participants n=7 Participants	14 participants n=5 Participants
Location of VOC Legs	12 participants n=5 Participants	10 participants n=7 Participants	22 participants n=5 Participants
Oral analgesic within 2h prior to study drug	2 participants n=5 Participants	3 participants n=7 Participants	5 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 20 minutes after administration of study drug

Change in pain score between 0 and 20 minutes using the Wong Baker FACES pain scale (WBFPS). The WBFPRS has six faces, with each face representing an increasing severity of pain the more rightward it is on the scale (0 is the lowest score , which represents the least amount of pain, while 10 is the highest score which represents the greatest level of pain).. Each face has an even number underneath it, consecutively.

To calculate the change, the reported pain score at 20 minutes was subtracted from the reported baseline pain score. Thus, the higher change in pain score is indicative of a GREATER change in pain (i.e. greater decrease in pain at 20 minutes compared to baseline). The greatest possible changes in pain would be a 10 (pain score of 10 at baseline and 0 at 20 minutes) representing a DECREASE in pain between the two time points, and -10 (pain score of 0 at baseline and 10 at 20 minutes) representing a INCREASE in pain between the two time points.

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Change in Pain Score 20 Minutes After Administration of Study Drug	1 units on a scale Interval 0.0 to 2.0	2 units on a scale Interval 0.5 to 4.0

SECONDARY outcome

Timeframe: Every 5 minutes until 30 minutes after study drug administration

Number of participants who had bradycardia

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Presence of Bradycardia	0 participants	0 participants

SECONDARY outcome

Timeframe: Participants will be followed for the duration of their ED visit, an expected average of 6 hours

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=25 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Presence of Headache	0 participants	4 participants

SECONDARY outcome

Timeframe: This will be assessed at either discharge from the ED or admission to an inpatient unit, an expected average of 6 hours after triage

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Admission Rate	16 participants	13 participants

SECONDARY outcome

Timeframe: Time from triage until either discharge from the ED or admission to an inpatient unit, an expected average of 6 hours

Given multiple confounding factors, reliable data was not able to be obtained for this outcome measure

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Participants will be followed for the duration of their ED visit, an expected average of 6 hours

Given multiple confounding and extraneous factors, reliable data was not able to be obtained for this outcome measure

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Time from triage to adminstration of study drug

Outcome measures

Measure	Intranasal Saline n=24 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=25 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Time to Study Drug Administration	47 minutes Standard Deviation 28	43 minutes Standard Deviation 36

SECONDARY outcome

Timeframe: Baseline and 10 minutes after administration of study drug

Change in pain score between 0 and 10 minutes using the Wong Baker FACES pain scale (WBFPS). The WBFPRS has six faces, with each face representing an increasing severity of pain the more rightward it is on the scale (0 is the lowest score , which represents the least amount of pain, while 10 is the highest score which represents the greatest level of pain).. Each face has an even number underneath it, consecutively.

To calculate the change, the reported pain score at 10 minutes was subtracted from the reported baseline pain score. Thus, the higher change in pain score is indicative of a GREATER change in pain (i.e. greater decrease in pain at 10 minutes compared to baseline). The greatest possible changes in pain would be a 10 (pain score of 10 at baseline and 0 at 10 minutes) representing a DECREASE in pain between the two time points, and -10 (pain score of 0 at baseline and 10 at 10 minutes) representing a INCREASE in pain between the two time points.

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Change in Pain Score at 10 Minutes	0 units on a scale Interval 0.0 to 2.0	2 units on a scale Interval 0.0 to 4.0

SECONDARY outcome

Timeframe: Baseline and 30 minutes after administration of study drug

Change in pain score between 0 and 30 minutes using the Wong Baker FACES pain scale (WBFPS). The WBFPRS has six faces, with each face representing an increasing severity of pain the more rightward it is on the scale (0 is the lowest score, which represents the least amount of pain, while 10 is the highest score which represents the greatest level of pain).. Each face has an even number underneath it, consecutively.

To calculate the change, the reported pain score at 30 minutes was subtracted from the reported baseline pain score. Thus, the higher change in pain score is indicative of a GREATER change in pain (i.e. greater decrease in pain at 30 minutes compared to baseline). The greatest possible changes in pain would be a 10 (pain score of 10 at baseline and 0 at 30 minutes) representing a DECREASE in pain between the two time points, and -10 (pain score of 0 at baseline and 10 at 30 minutes) representing a INCREASE in pain between the two time points.

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Change in Pain Score at 30 Minutes	2 units on a scale Interval 0.0 to 2.0	2 units on a scale Interval 0.0 to 4.0

SECONDARY outcome

Timeframe: Baseline and immediately prior to IV insertion

Due to confounding factors we were unable to obtain reliable data for this outcome

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Every 5 minutes until 30 minutes after study drug administration

Participants who had respiratory distress within 30 min of study drug administration

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Respiratory Distress	0 participants	0 participants

SECONDARY outcome

Timeframe: Every 5 minutes until 30 minutes after study drug administration

Participants who had hypotension within 30 min of study drug administration

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Hypotension	2 participants	2 participants

SECONDARY outcome

Timeframe: Every 5 minutes until 30 minutes after study drug administration

Number of participants who had hypoxia within 30 min of study drug adminsitration

Outcome measures

Measure	Intranasal Saline n=25 Participants Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 Participants Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Hypoxia	0 participants	3 participants

Adverse Events

Intranasal Saline

Serious events: 2 serious events

Other events: 9 other events

Deaths: 0 deaths

Intranasal Fentnayl

Serious events: 5 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Measure	Intranasal Saline n=25 participants at risk Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 participants at risk Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/25	12.5% 3/24
Cardiac disorders Hypotension	8.0% 2/25	8.3% 2/24

Other adverse events

Measure	Intranasal Saline n=25 participants at risk Normal Saline: A single dose of equivalent volume of 0.9% Normal Saline will be administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device.	Intranasal Fentnayl n=24 participants at risk Fentanyl Citrate: A single dose of fentanyl citrate (2 mcg/kg; max 100mcg) administered intranasally. Half of the volume will be administered in each nare. The medication will be administered using a mucosal atomization device
Nervous system disorders Sleepiness	24.0% 6/25	41.7% 10/24
Nervous system disorders Headache	0.00% 0/25	16.7% 4/24
Skin and subcutaneous tissue disorders Itching	4.0% 1/25	8.3% 2/24
Gastrointestinal disorders Nausea	4.0% 1/25	8.3% 2/24
Skin and subcutaneous tissue disorders Nasal Pain	0.00% 0/25	12.5% 3/24
Respiratory, thoracic and mediastinal disorders Acute Chest Syndrome	8.0% 2/25	8.3% 2/24
Blood and lymphatic system disorders Blood Transfusion	0.00% 0/25	8.3% 2/24

Additional Information

Dr. Daniel Fein

Albert Einstein College of Medicine/Children's Hospital at Montefiore

Phone: 718-920-5312

Email: dfein@montefiore.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place