Trial Outcomes & Findings for Management of Severe Acute Malnutrition in SCD, in Northern Nigeria (NCT NCT03634488)

NCT ID: NCT03634488

Last Updated: 2024-03-27

Results Overview

The primary outcome is the proportion of eligible individuals who adhere to therapy (Ready-to-use therapeutic food and hydroxyurea). The adherence rate for hydroxyurea was determined based on mean corpuscular volume (MCV) values at exit (12 weeks).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

132 participants

Primary outcome timeframe

Feasibility over 12-week Period [Time Frame: 3 months]

Results posted on

2024-03-27

Participant Flow

The first participant was enrolled in the "Management of Severe Acute Malnutrition in Children With Sickle Cell Disease Greater Than 5 Years of Age Living in Northern Nigeria" study in August 2021. Recruitment took place at Aminu Kano Teaching Hospital and Murtala Muhammad Specialist Hospital in Kano, Nigeria. Enrollment for children with SCD (either the Ready-to-Use Therapeutic Food arm or the Ready-to-Use Therapeutic Food and Hydroxyurea arm) and siblings without SCD ended in May 2022.

The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia.

Participant milestones

Participant milestones
Measure
SCD: Ready-to-use Therapeutic Food and Hydroxyurea
At least 50 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive Ready-to-use therapeutic food (500-1,000 daily calories) and hydroxyurea (20mg/kg/day) Hydroxyurea (20mg/kg/day): Treatment of severe malnutrition in children with SCA in northern Nigeria Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
SCD: Ready-to-use Therapeutic Food Alone
At least 50 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive Ready-to-use therapeutic food alone Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Siblings Without SCD
Up to 100 non-SCD siblings(5-12 years old) and severe malnutrition will be enrolled. Ready-to-use therapeutic food: Treatment of severe malnutrition in children without SCD in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Overall Study
STARTED
56
54
22
Overall Study
COMPLETED
54
54
22
Overall Study
NOT COMPLETED
2
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
SCD: Ready-to-use Therapeutic Food and Hydroxyurea
At least 50 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive Ready-to-use therapeutic food (500-1,000 daily calories) and hydroxyurea (20mg/kg/day) Hydroxyurea (20mg/kg/day): Treatment of severe malnutrition in children with SCA in northern Nigeria Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
SCD: Ready-to-use Therapeutic Food Alone
At least 50 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive Ready-to-use therapeutic food alone Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Siblings Without SCD
Up to 100 non-SCD siblings(5-12 years old) and severe malnutrition will be enrolled. Ready-to-use therapeutic food: Treatment of severe malnutrition in children without SCD in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Overall Study
Death
1
0
0
Overall Study
A participant was administratively withdrawn due to a calculated BMI z-score >-3.0.
1
0
0

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
SCD - Ready-to-use Therapeutic Food and Hydroxyurea
n=55 Participants
Children (5-12 years old) with SCA and severe malnutrition randomly allocated to receive Ready-to-use therapeutic food and hydroxyurea (20mg/kg/day) hydroxyurea (20mg/kg/day): Treatment of severe malnutrition in children with SCA in northern Nigeria Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
SCD - Ready-to-use Therapeutic Food Alone
n=54 Participants
Children (5-12 years old) with SCA and severe malnutrition randomly allocated to receive Ready-to-use therapeutic food alone Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Siblings Without SCD
n=22 Participants
non-SCD siblings(5-12 years old) with severe malnutrition. Ready-to-use therapeutic food: Treatment of severe malnutrition in children without SCD in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Total
n=131 Participants
Total of all reporting groups
Age, Continuous
10.2 years
n=55 Participants
10.5 years
n=54 Participants
8.2 years
n=22 Participants
10.1 years
n=131 Participants
Sex: Female, Male
Female
26 Participants
n=55 Participants
27 Participants
n=54 Participants
14 Participants
n=22 Participants
67 Participants
n=131 Participants
Sex: Female, Male
Male
29 Participants
n=55 Participants
27 Participants
n=54 Participants
8 Participants
n=22 Participants
64 Participants
n=131 Participants
Race and Ethnicity Not Collected
0 Participants
Race and Ethnicity were not collected from any participant.
Race/Ethnicity, Customized
Fulani
4 Participants
n=55 Participants
2 Participants
n=54 Participants
0 Participants
n=22 Participants
6 Participants
n=131 Participants
Race/Ethnicity, Customized
Hausa
49 Participants
n=55 Participants
51 Participants
n=54 Participants
22 Participants
n=22 Participants
122 Participants
n=131 Participants
Race/Ethnicity, Customized
Kanuri
1 Participants
n=55 Participants
0 Participants
n=54 Participants
0 Participants
n=22 Participants
1 Participants
n=131 Participants
Race/Ethnicity, Customized
Other
1 Participants
n=55 Participants
1 Participants
n=54 Participants
0 Participants
n=22 Participants
2 Participants
n=131 Participants
Weight, kg, mean (std. dev.)
18.4 kg
STANDARD_DEVIATION 3.5 • n=55 Participants
18.7 kg
STANDARD_DEVIATION 3.5 • n=54 Participants
18.1 kg
STANDARD_DEVIATION 4.2 • n=22 Participants
18.5 kg
STANDARD_DEVIATION 3.6 • n=131 Participants
Height, cm, mean (std. dev.)
123.5 centimeters
STANDARD_DEVIATION 10.1 • n=55 Participants
124.1 centimeters
STANDARD_DEVIATION 9.5 • n=54 Participants
123.5 centimeters
STANDARD_DEVIATION 12.3 • n=22 Participants
123.7 centimeters
STANDARD_DEVIATION 10.2 • n=131 Participants
Body Mass Index, kg/m^2, mean (std. dev.)
12 kg/m^2
STANDARD_DEVIATION 0.6 • n=55 Participants
12.1 kg/m^2
STANDARD_DEVIATION 0.6 • n=54 Participants
11.7 kg/m^2
STANDARD_DEVIATION 0.6 • n=22 Participants
12 kg/m^2
STANDARD_DEVIATION 0.6 • n=131 Participants
Body Mass Index z-score, mean (std. dev.)
-3.64 Z-score
STANDARD_DEVIATION 0.49 • n=55 Participants
-3.66 Z-score
STANDARD_DEVIATION 0.44 • n=54 Participants
-3.6 Z-score
STANDARD_DEVIATION 0.5 • n=22 Participants
-3.65 Z-score
STANDARD_DEVIATION 0.46 • n=131 Participants
Hemoglobin, g/dl, mean (std. dev.)
7.4 g/dl
STANDARD_DEVIATION 1 • n=55 Participants
7.3 g/dl
STANDARD_DEVIATION 1.1 • n=54 Participants
10.8 g/dl
STANDARD_DEVIATION 1.0 • n=22 Participants
7.9 g/dl
STANDARD_DEVIATION 1.7 • n=131 Participants

PRIMARY outcome

Timeframe: 6 months

Population: Children aged 5-12 years with SCA were evaluated for eligibility. Siblings of enrolled participants aged 5-12 years old without SCD were also evaluated for eligibility.

Recruitment Feasibility: The primary outcome is the proportion of eligible individuals that agree to be included, referred to as the recruitment rate. Children with severe malnutrition who qualified and agreed to participate were invited to sign a consent and assent for study recruitment to this study.

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=111 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=22 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Enrollment Rate at the End of the 6-month Recruitment Period
Unable to randomize
1 participants
0 participants
Enrollment Rate at the End of the 6-month Recruitment Period
Eligible
111 participants
22 participants
Enrollment Rate at the End of the 6-month Recruitment Period
Randomized
110 participants
22 participants

PRIMARY outcome

Timeframe: 12 weeks

Population: The analysis included participants with sickle cell anemia and severe acute malnutrition. The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia.

The primary outcome is the proportion of participants who completed the 12-week trial, known as the retention rate for the trial.

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=56 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=54 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
n=22 Participants
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Retention Over 12-week Period
Voluntarily withdrew from the trial
0 participants
0 participants
0 participants
Retention Over 12-week Period
Involuntary withdrawal from the trial
1 participants
0 participants
0 participants

PRIMARY outcome

Timeframe: 12 weeks

Population: The final analysis included 54 participants with sickle cell anemia from each arm with sickle cell anemia and severe acute malnutrition, along with 22 non-SCD siblings with severe acute malnutrition.

Adherence to the ready-to-use therapeutic food was evaluated based on the percentage of empty food sachets returned at each visit.

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=54 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=54 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
n=22 Participants
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Percentage of Ready-to-use Therapeutic Food Sachets Returned as Empty.
99.02 percentage of empty sachets
Interval 99.0 to 99.04
99.03 percentage of empty sachets
Interval 99.0 to 99.05
99.02 percentage of empty sachets
Interval 99.01 to 99.04

PRIMARY outcome

Timeframe: 12 weeks

Population: Per-protocol analysis of participants.

Adherence to monthly visits was assessed based on the number of missed visits

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=216 Research Visits
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=216 Research Visits
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
n=88 Research Visits
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Number of Missed Visits
0 missed visits
0 missed visits
0 missed visits

PRIMARY outcome

Timeframe: 12 weeks

Population: The final analysis included 54 participants with sickle cell anemia and severe acute malnutrition randomized to receive hydroxyurea in addition to ready-to-use therapeutic food. The other arms did not receive hydroxyurea, and therefore, adherence to hydroxyurea cannot be evaluated.

Adherence to hydroxyurea was evaluated based on the percentage of hydroxyurea pills returned for the group randomized to both ready-to-use therapeutic food and hydroxyurea.

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=54 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Percentage of Hydroxyurea Pills Returned
4.4 percentage of hydroxyurea pills returned
Interval 0.0 to 8.27

PRIMARY outcome

Timeframe: 12 weeks

Population: The final analysis included 54 participants from each arm with sickle cell anemia and severe acute malnutrition. We did not include the siblings' change in mean corpuscular volume - since none of the siblings without sickle cell disease received hydroxyurea.

Adherence to hydroxyurea was evaluated based on change in mean corpuscular volume

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=54 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=54 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Change in Mean Corpuscular Volume
5.4 fl
Standard Deviation 12.7
-1.0 fl
Standard Deviation 10.9

PRIMARY outcome

Timeframe: Baseline to 12 weeks

Population: The final analysis included 54 participants from each arm with sickle cell anemia and severe acute malnutrition. We did not measure fetal hemoglobin in children without SCD.

The primary outcome is the proportion of eligible individuals who adhere to therapy (Ready-to-use therapeutic food and hydroxyurea). The adherence rate for hydroxyurea was determined based on the change in fetal hemoglobin level percentage.

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=54 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=54 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Change in Fetal Hemoglobin Level Percentage
3.9 percentage of fetal hemoglobin
Interval 1.7 to 6.8
2.2 percentage of fetal hemoglobin
Interval 0.5 to 4.9

PRIMARY outcome

Timeframe: Feasibility over 12-week Period [Time Frame: 3 months]

Population: The final analysis included 54 participants from each arm with sickle cell anemia and severe acute malnutrition. The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in these main results.

The primary outcome is the proportion of eligible individuals who adhere to therapy (Ready-to-use therapeutic food and hydroxyurea). The adherence rate for hydroxyurea was determined based on mean corpuscular volume (MCV) values at exit (12 weeks).

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=54 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=54 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Mean Corpuscular Volume Values at Exit
88.5 fl
Standard Deviation 14.7
85.4 fl
Standard Deviation 11.4

PRIMARY outcome

Timeframe: Feasibility over 12-week Period [Time Frame: 3 months]

Population: The final analysis included 54 participants from each arm with sickle cell anemia and severe acute malnutrition. The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in these main results.

The primary outcome is the proportion of eligible individuals who adhere to therapy (Ready-to-use therapeutic food and hydroxyurea). The adherence rate for hydroxyurea was determined based on the fetal hemoglobin levels at exit (12 weeks).

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=54 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=54 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Fetal Hemoglobin Levels at Exit
12.2 percentage of fetal hemoglobin
Interval 7.5 to 16.9
9.4 percentage of fetal hemoglobin
Interval 5.6 to 13.8

PRIMARY outcome

Timeframe: Feasibility over 12-week Period [Time Frame: 3 months]

Population: The final analysis included 54 participants from each arm with sickle cell anemia and severe acute malnutrition. The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in these main results.

The primary outcome is the proportion of eligible individuals who adhere to therapy (Ready-to-use therapeutic food and hydroxyurea). The adherence rate for hydroxyurea was determined based on the total hemoglobin levels at exit (12 weeks).

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=54 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=54 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Total Hemoglobin Levels at Exit
8.3 g/dl
Standard Deviation 1.1
7.7 g/dl
Standard Deviation 1.3

SECONDARY outcome

Timeframe: 12 weeks

Population: The final analysis included 54 participants with sickle cell anemia from each arm with sickle cell anemia and severe acute malnutrition, along with 22 non-SCD siblings with severe acute malnutrition.

As a secondary outcome, we assessed the percentage of participants with and without SCA who continued to have a body mass index z-score of \<-3.0 at the end of the 12 weeks of treatment. Using the World Health Organization (WHO) growth reference, anthropometric measurements were converted to age and sex-specific z-scores. Anthropometric Indices (BMI-for-age (BMIZ), were calculated using WHO 2007 R Macro Package to assess growth and development of the children. Severe malnutrition/wasting (SAM) was defined as a body mass index (BMI) z-score \<-3.0.

Outcome measures

Outcome measures
Measure
Eligible Children Aged 5-12 Years With SCA
n=54 Participants
Children with SCA who met the criteria to participate in the study
Eligible Children Aged 5-12 Years Old Without SCD
n=54 Participants
Non-SCD siblings of participants with SCA who met the criteria to participate in the study
Siblings Without SCD
n=22 Participants
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Percentage of Participants Maintaining a BMI Z-score Less Than -3.0
66.7 percentage of participants
55.6 percentage of participants
30.1 percentage of participants

Adverse Events

Ready-to-use Therapeutic Food and Hydroxyurea

Serious events: 1 serious events
Other events: 2 other events
Deaths: 1 deaths

Ready-to-use Therapeutic Food Alone

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Siblings Without SCD

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

Serious adverse events

Serious adverse events
Measure
Ready-to-use Therapeutic Food and Hydroxyurea
n=56 participants at risk
50 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive Ready-to-use therapeutic food and hydroxyurea (20mg/kg/day) hydroxyurea (20mg/kg/day): Treatment of severe malnutrition in children with SCA in northern Nigeria Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Ready-to-use Therapeutic Food Alone
n=54 participants at risk
50 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive Ready-to-use therapeutic food alone Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Siblings Without SCD
n=22 participants at risk
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Blood and lymphatic system disorders
Prolonged hospitalization
1.8%
1/56 • Number of events 1 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/54 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/22 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.

Other adverse events

Other adverse events
Measure
Ready-to-use Therapeutic Food and Hydroxyurea
n=56 participants at risk
50 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive Ready-to-use therapeutic food and hydroxyurea (20mg/kg/day) hydroxyurea (20mg/kg/day): Treatment of severe malnutrition in children with SCA in northern Nigeria Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Ready-to-use Therapeutic Food Alone
n=54 participants at risk
50 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive Ready-to-use therapeutic food alone Ready-to-use therapeutic food: Treatment of severe malnutrition in children with SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food
Siblings Without SCD
n=22 participants at risk
Non-SCD siblings (5-12 years old) with severe malnutrition enrolled to receive Ready-to-use therapeutic food (500-1,000 daily calories) only.
Blood and lymphatic system disorders
Pain
1.8%
1/56 • Number of events 1 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
3.7%
2/54 • Number of events 2 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/22 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
Blood and lymphatic system disorders
Acute Chest Syndrome
1.8%
1/56 • Number of events 1 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/54 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/22 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
General disorders
Fever
3.6%
2/56 • Number of events 2 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
5.6%
3/54 • Number of events 3 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/22 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
Ear and labyrinth disorders
Elective Surgery
0.00%
0/56 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
1.9%
1/54 • Number of events 1 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/22 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
Blood and lymphatic system disorders
Anemia
1.8%
1/56 • Number of events 1 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
1.9%
1/54 • Number of events 1 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/22 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
Infections and infestations
Malaria
3.6%
2/56 • Number of events 2 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
5.6%
3/54 • Number of events 3 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.
0.00%
0/22 • The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.
The feasibility trial's primary objectives are to evaluate the intervention's feasibility and safety in children with sickle cell anemia. We, therefore, did not include the outcomes for the siblings in the main results.

Additional Information

Leshana Saint Jean

Vanderbilt UniversityVanderbilt-Meharry Center of Excellence in Sickle Cell Disease

Phone: 6158751992

Results disclosure agreements

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