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Trial Outcomes & Findings for Safety and Efficacy of Sodium Nitrite in Sickle Cell Disease (NCT NCT01033227)

NCT ID: NCT01033227

Last Updated: 2017-10-17

Results Overview

The primary end points will be to determine if a) a 48-hour sodium nitrite infusion is tolerated without a decrease in mean arterial blood pressure by 15mmHg for greater than 2 hours or development of methemoglobin greater than 5% and b) a 48-hour sodium nitrite infusion is safe as determined by monitoring for adverse events

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

5 participants

Primary outcome timeframe

48 hours from start of infusion

Results posted on

2017-10-17

Participant Flow

Subjects were recruited for the study during regular clinic visits.

Once consent signed subjects would not be randomized into the treatment arm until (if or when) they had their admission to the hospital for vaso-occlusive crisis (VOC).

Participant milestones

Participant milestones
Measure
No Drug
This group did not receive anything additional in the no drug arm. The treatment group received the study drug and the non treatment group received no drug.
Sodium Nitrite Injection, USP
Administration if sodium nitrite injection, USP sodium nitrite injection, usp: Sodium nitrite injection, USP will be administered in blocks of six subjects (3 sodium nitrite and 3 no drug). A total of five dose levels are planned, pending safety starting. Drug will be given by continuous infusion infusion for 48 hours starting at 6 nmol/min/kg (10% of the maximal tolerated dose).
Overall Study
STARTED
3
2
Overall Study
COMPLETED
3
2
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Efficacy of Sodium Nitrite in Sickle Cell Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
No Drug
n=3 Participants
Sodium Nitrite Injection, USP
n=2 Participants
Administration if sodium nitrite injection, USP sodium nitrite injection, usp: Sodium nitrite injection, USP will be administered in blocks of six subjects (3 sodium nitrite and 3 no drug). A total of five dose levels are planned, pending safety starting. Drug will be given by continuous infusion infusion for 48 hours starting at 6 nmol/min/kg (10% of the maximal tolerated dose).
Total
n=5 Participants
Total of all reporting groups
Age, Categorical
<=18 years
3 Participants
n=5 Participants
1 Participants
n=7 Participants
4 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Continuous
16 years
n=5 Participants
15.5 years
n=7 Participants
16 years
n=5 Participants
Sex: Female, Male
Female
3 Participants
n=5 Participants
2 Participants
n=7 Participants
5 Participants
n=5 Participants
Sex: Female, Male
Male
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Region of Enrollment
United States
3 participants
n=5 Participants
2 participants
n=7 Participants
5 participants
n=5 Participants
Sickle Cell Disease hb SS
3 Participants
n=5 Participants
1 Participants
n=7 Participants
4 Participants
n=5 Participants
Sbeta+thalassemia
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 48 hours from start of infusion

The primary end points will be to determine if a) a 48-hour sodium nitrite infusion is tolerated without a decrease in mean arterial blood pressure by 15mmHg for greater than 2 hours or development of methemoglobin greater than 5% and b) a 48-hour sodium nitrite infusion is safe as determined by monitoring for adverse events

Outcome measures

Outcome measures
Measure
No Drug
n=3 Participants
Will not receive study drug, there is no placebo in this study. The patient will know they are not receiving the study drug.
Sodium Nitrite Injection, USP
n=2 Participants
Administration of sodium nitrite injection, USP
48 Hour Sodium Nitrite Infusion Safety as Determined by Number of Participants With No Adverse Events
3 Participants
2 Participants

SECONDARY outcome

Timeframe: 48 hours

Population: Data were \*not collected\* and the Outcome was never analyzed, study terminated

a) reduced the duration and intensity of pain; b) reduced total narcotic analgesic consumption; and c) reduced length of hospitalization.

Outcome measures

Outcome data not reported

Adverse Events

No Drug

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Sodium Nitrite Injection, USP

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

Dr. Thomas Coates

Children's Hospital Los Angeles

Phone: 323 361 3841

Results disclosure agreements

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