Sickle Cell AnemiaPhase 2NCT03634488

Management of Severe Acute Malnutrition in SCD, in Northern Nigeria

A Phase 2 study testing hydroxyurea (20mg/kg/day) and Ready-to-use therapeutic food in people with Sickle Cell Anemia and Severe Acute Malnutrition, run by Vanderbilt University Medical Center at 3 sites in 2 countries.

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

Who is it for?: Ages 5 to 12 yearsHealthy volunteers can take part
Conditions the study is for: Sickle Cell Anemia, Severe Acute Malnutrition
What is being tested?: hydroxyurea (20mg/kg/day) and Ready-to-use therapeutic food
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 132 participants
Where: 3 sitesin 2 countries
Study dates: 18 Aug 2021to 9 Nov 2022
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 5 to 12 years
All sexes
Healthy volunteers accepted
Child

Who may be able to join

confirmed diagnoses of SCA, comparison children without SCD
severe malnutrition defined as a BMI z-score < -3
age between 5 and 12 years (assessment can take place up until the 13th birthday)
pass the appetite test
uncomplicated malnutrition (good appetite, alert, no signs of infection of respiratory distress)

Who cannot join

children with complicated severe acute malnutrition
children with electrolyte disturbances (serum Na, K, PO4) at baseline
children on disease-modifying therapy (hydroxyurea or regular blood transfusion therapy)
children enrolled in other studies
children with diabetes and other chronic illnesses
children with known HIV infection
children with a known allergy to dairy or peanuts.

Trial locations and contacts

3 sites in 2 countries.

Country

City, postcode or hospital

3 sites in 2 countries

Nigeria2 sites

Aminu Kano Teaching Hospital
Kano
Murtala Mohammad Specialist Hospital
Kano

Other trials in Nigeria

United States1 site

Vanderbilt University Medical Center
Nashville, Tennessee, 37232-9000

Other trials in United States

About this study

Official title: Management of Severe Acute Malnutrition in Children With Sickle Cell Disease Greater Than 5 Years of Age Living in Northern Nigeria

Except for children with HIV, all recommendations for treatment of childhood malnutrition are for children < 5 years of age. The overall goal of this randomized controlled nutrition feasibility trial is to identify whether families of children with sickle cell disease (SCD) 5 years and older agree to participate over a 12-week period. The investigators will also establish a safety protocol for monitoring potential complications associated with treating severe malnutrition in children 5 years and older with and without SCD, in a low-resource setting.

Read the detailed description

The overall goal of this feasibility trial is to determine the acceptability of a randomized controlled trial to ascertain the optimal strategy for the treatment of severe malnutrition in children with sickle cell disease (SCD) 5 years and older. No international standard or evidence-based guidelines exist for the treatment of severe malnutrition (defined as BMI Z-score below -3) in children with SCD. With an expanding pediatric population of more than 75 million in Nigeria, coupled with decreasing childhood infectious disease-related mortality, the next emerging threats to preventable childhood deaths are non-communicable diseases. Data from our NIH-funded randomized controlled primary stroke prevention trial in Nigeria (NCT02560935), in which the investigators evaluated children with SCD between 5 and 12 years of age, demonstrated that 29% (230/803) of the cohort met criteria for severe malnutrition. Approximately 92% of the cohort in northern Nigeria identified as having severe malnutrition was below the 5th percentile for weight of children with SCD living in the US, Canada, or Europe. These data indicate older children with SCD living in northern Nigeria are undernourished when compared to children living with SCD in high-resource settings. A potentially unique attribute to treating malnutrition in children with SCD is the use of FDA approved anti-metabolite, hydroxyurea, to prevent vaso-occlusive pain events in children. The beneficial effects of hydroxyurea include, but are not limited to, decreased inflammation and increased hemoglobin levels. Preliminary evidence in this cohort of older children with sickle cell anemia (SCA) in northern Nigeria reveals that moderate fixed-dose hydroxyurea (20 mg/kg/day) significantly increases BMI in children with severe malnutrition. The investigators propose a randomized controlled feasibility trial in older children (5 to 12 years of age) with SCA living in northern Nigeria. In preparation for a definitive phase III trial to determine if ready-to-use therapeutic food and moderate fixed-dose hydroxyurea therapy is superior to ready-to-use therapeutic food alone, the investigators will randomly allocate up to 150 children between 5 and 12 years of age with SCA and severe uncomplicated malnutrition to each of the two arms. In aim 1, the investigators will assess the feasibility (rate of recruitment, retention, and adherence) of a randomized controlled trial (RCT) in children with SCD and severe malnutrition to a 12-week intervention period. For aim 2, the investigators will establish the safety protocol to monitor for unknown rates of complications associated with treating malnutrition in children with SCD. To decrease the likelihood of sharing limited food resources in a poor family and to determine the specificity of malnutrition for children with SCD in northern Nigeria, the investigators will screen and treat up to 100 malnourished non-SCD siblings of the trial participants. After completion of this feasibility trial, the investigators will use the acquired knowledge to design a phase III trial to definitively determine the optimal treatment strategy for severe malnutrition in older children with SCD living in Africa, potentially affecting thousands of children in this region.

Conditions: Sickle Cell Anemia, Severe Acute Malnutrition
Being tested: hydroxyurea (20mg/kg/day), Ready-to-use therapeutic food
Keywords: sickle cell disease, sub-Saharan Africa, malnutrition, low-income settings, severe acute malnutrition, sickle cell anemia, Nigeria
Medical subject headings: Anemia, Sickle Cell, Severe Acute Malnutrition, Malnutrition

What participants receive

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

hydroxyurea (20mg/kg/day)Drug
Also called hydrea
Treatment of severe malnutrition in children with SCA in northern Nigeria
Ready-to-use therapeutic foodDietary supplement
Also called RUTF
Treatment of severe malnutrition in children with and without SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food

Study groups in NCT03634488
Study group	Role	What they receive
SCD - Ready-to-use therapeutic food and Hydroxyurea	Experimental	50-75 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) Receives: Drug: hydroxyurea (20mg/kg/day); Dietary Supplement: Ready-to-use therapeutic food
SCD - Ready-to-use therapeutic food alone	Placebo comparator	50-75 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive ready-to-use therapeutic food alone Receives: Dietary Supplement: Ready-to-use therapeutic food
Non-SCD siblings with severe malnutrition	Placebo comparator	To decrease the likelihood of sharing limited food resources, we will enroll up to 100 malnourished non-SCD siblings. Receives: Dietary Supplement: Ready-to-use therapeutic food

What is being measured

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

Enrollment Rate at the End of the 6-month Recruitment Period
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.
Measured: 6 months
Retention Over 12-week Period
The primary outcome is the proportion of participants who completed the 12-week trial, known as the retention rate for the trial.
Measured: 12 weeks
Percentage of Ready-to-use Therapeutic Food Sachets Returned as Empty.
Adherence to the ready-to-use therapeutic food was evaluated based on the percentage of empty food sachets returned at each visit.
Measured: 12 weeks
Number of Missed Visits
Adherence to monthly visits was assessed based on the number of missed visits
Measured: 12 weeks
Percentage of Hydroxyurea Pills Returned
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.
Measured: 12 weeks
Change in Mean Corpuscular Volume
Adherence to hydroxyurea was evaluated based on change in mean corpuscular volume
Measured: 12 weeks
Change in Fetal Hemoglobin Level Percentage
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.
Measured: Baseline to 12 weeks
Mean Corpuscular Volume Values at Exit
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).
Measured: Feasibility over 12-week Period [Time Frame: 3 months]
Fetal Hemoglobin Levels at Exit
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).
Measured: Feasibility over 12-week Period [Time Frame: 3 months]
Total Hemoglobin Levels at Exit
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).
Measured: Feasibility over 12-week Period [Time Frame: 3 months]

Secondary outcomes (1)

Percentage of Participants Maintaining a BMI Z-score Less Than -3.0
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.
Measured: 12 weeks

Timeline

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

18 Aug 20219 Nov 2022

First posted16 Aug 2018
Study start18 Aug 2021
Primary completion5 Oct 2022
Study completion9 Nov 2022

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.
Publications (5)Papers the sponsor has linked to this study.
Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
More trials run by Vanderbilt University Medical CenterThe 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
Model description: A 12-week, open label, randomized controlled feasibility trial in children with sickle cell anemia between 5 and 12 years of age to treat uncomplicated severe malnutrition.
Masking: Single blind
Who is masked: Investigator
Enrolment: 132 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03634488
Sponsor's protocol ID: 170577
Status last verified: Feb 2024
First submitted: 30 Jan 2018
Last updated: 27 Mar 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Murtala HA, Abdullahi SU, Gambo S, Kabir H, Shamsu KA, Gwarzo G, Acra SA, Stallings VA, Rodeghier M, DeBaun MR, Klein LJ. Including malnourished siblings in treatment improves nutritional outcomes for children with sickle cell anemia in Northern Nigeria: Results from a feasibility trial. Nutr Res. 2025 Dec;144:32-37. doi: 10.1016/j.nutres.2025.10.006. Epub 2025 Oct 18.
PubMed 41253090
Brechko A, Abdullahi SU, Gambo S, Murtala HA, Kabir H, Shamsu KA, Stallings VA, Rodeghier M, Ramesh A, Sellers A, Schlundt DG, DeBaun MR, Klein LJ. Adherence to ready-to-use therapeutic food in older children with sickle cell anaemia and severe acute malnutrition: a mixed-methods study in a low-income setting. BMJ Glob Health. 2025 Aug 7;10(8):e019096. doi: 10.1136/bmjgh-2025-019096.
PubMed 40780831
Ramirez-Cuebas G, Abdullahi SU, Gambo S, Murtala HA, Kabir H, Shamsu KA, Gwarzo G, Acra SA, Stallings VA, Rodeghier M, DeBaun MR, Klein LJ. Impact of Food Insecurity on Malnutrition Treatment Response in Nigerian Children With Sickle Cell Anemia and Severe Acute Malnutrition. Pediatr Blood Cancer. 2025 Jun;72(6):e31637. doi: 10.1002/pbc.31637. Epub 2025 Mar 10.
PubMed 40062628
Ritter C, Abdullahi SU, Gambo S, Murtala HA, Kabir H, Shamsu KA, Gwarzo G, Banaei Y, Acra SA, Stallings VA, Rodeghier M, DeBaun MR, Klein LJ. Impact of maternal depression on malnutrition treatment outcomes in older children with sickle cell anemia. BMC Nutr. 2024 Jan 24;10(1):18. doi: 10.1186/s40795-024-00826-0.
PubMed 38268013
Abdullahi SU, Gambo S, Murtala HA, Kabir H, Shamsu KA, Gwarzo G, Acra S, Stallings VA, Rodeghier M, DeBaun MR, Klein LJ. Feasibility trial for the management of severe acute malnutrition in older children with sickle cell anemia in Nigeria. Blood Adv. 2023 Oct 24;7(20):6024-6034. doi: 10.1182/bloodadvances.2023010789.
PubMed 37428866

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis

All Sickle Cell Anemia trials

Study at a glance

From the registry record, last updated by the sponsor on 27 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Management of Severe Acute Malnutrition in Children With Sickle Cell Disease Greater Than 5 Years of Age Living in Northern Nigeria
ClinicalTrials.gov ID: NCT03634488
Acronym / sponsor ID: 170577
Conditions: Sickle Cell Anemia, Severe Acute Malnutrition
Keywords: sickle cell disease, sub-Saharan Africa, malnutrition, low-income settings, severe acute malnutrition, sickle cell anemia, Nigeria
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 132 participants (actual)
Lead sponsor: Vanderbilt University Medical CenterUniversity, hospital or other
Collaborators: Aminu Kano Teaching Hospital, Murtala Muhammad Specialist Hospital
Study start: 18 Aug 2021 (actual)
Primary completion: 5 Oct 2022 (actual)
Study completion: 9 Nov 2022 (actual)
First posted: 16 Aug 2018 (actual)
Results: Posted 27 Mar 2024

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 (investigator)
Number of arms: 3
Model description: A 12-week, open label, randomized controlled feasibility trial in children with sickle cell anemia between 5 and 12 years of age to treat uncomplicated severe malnutrition.

Arm	Type	Receives
SCD - Ready-to-use therapeutic food and Hydroxyurea	Experimental	Drug: hydroxyurea (20mg/kg/day), Dietary Supplement: Ready-to-use therapeutic food50-75 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)
SCD - Ready-to-use therapeutic food alone	Placebo comparator	Dietary Supplement: Ready-to-use therapeutic food50-75 children (5-12 years old) with SCA and severe malnutrition will be randomly allocated to receive ready-to-use therapeutic food alone
Non-SCD siblings with severe malnutrition	Placebo comparator	Dietary Supplement: Ready-to-use therapeutic foodTo decrease the likelihood of sharing limited food resources, we will enroll up to 100 malnourished non-SCD siblings.

Who may qualify

Ages: 5 to 12 years
Sex: All
Healthy volunteers: Accepted
Age groups: Child

Key inclusion criteria (5 listed)

confirmed diagnoses of SCA, comparison children without SCD
severe malnutrition defined as a BMI z-score < -3
age between 5 and 12 years (assessment can take place up until the 13th birthday)
pass the appetite test
uncomplicated malnutrition (good appetite, alert, no signs of infection of respiratory distress)

Key exclusion criteria (7 listed)

children with complicated severe acute malnutrition
children with electrolyte disturbances (serum Na, K, PO4) at baseline
children on disease-modifying therapy (hydroxyurea or regular blood transfusion therapy)
children enrolled in other studies
children with diabetes and other chronic illnesses

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
hydroxyurea (20mg/kg/day)Also: hydreaTreatment of severe malnutrition in children with SCA in northern Nigeria	Drug	SCD - Ready-to-use therapeutic food and Hydroxyurea
Ready-to-use therapeutic foodAlso: RUTFTreatment of severe malnutrition in children with and without SCA in northern Nigeria with an additional 500-1000 calories from ready-to-use-therapeutic food	Dietary supplement	Non-SCD siblings with severe malnutrition, SCD - Ready-to-use therapeutic food alone, SCD - Ready-to-use therapeutic food and Hydroxyurea

Primary outcome

Enrollment Rate at the End of the 6-month Recruitment Period
Time frame: 6 months
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.
Retention Over 12-week Period
Time frame: 12 weeks
The primary outcome is the proportion of participants who completed the 12-week trial, known as the retention rate for the trial.
Percentage of Ready-to-use Therapeutic Food Sachets Returned as Empty.
Time frame: 12 weeks
Adherence to the ready-to-use therapeutic food was evaluated based on the percentage of empty food sachets returned at each visit.
Number of Missed Visits
Time frame: 12 weeks
Adherence to monthly visits was assessed based on the number of missed visits
Percentage of Hydroxyurea Pills Returned
Time frame: 12 weeks
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.
Change in Mean Corpuscular Volume
Time frame: 12 weeks
Adherence to hydroxyurea was evaluated based on change in mean corpuscular volume
Change in Fetal Hemoglobin Level Percentage
Time frame: Baseline to 12 weeks
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.
Mean Corpuscular Volume Values at Exit
Time frame: Feasibility over 12-week Period [Time Frame: 3 months]
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).
Fetal Hemoglobin Levels at Exit
Time frame: Feasibility over 12-week Period [Time Frame: 3 months]
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).
Total Hemoglobin Levels at Exit
Time frame: Feasibility over 12-week Period [Time Frame: 3 months]
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).

1 secondary outcome

Percentage of Participants Maintaining a BMI Z-score Less Than -3.0
Time frame: 12 weeks

Results and safety

Results posted to the registry on 27 Mar 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
SCD: Ready-to-use Therapeutic Food and Hydroxyurea	56	54
SCD: Ready-to-use Therapeutic Food Alone	54	54
Siblings Without SCD	22	22

Primary outcome: Enrollment Rate at the End of the 6-month Recruitment Period

Time frame: 6 months. Number. Unit: participants

Measure	Eligible Children Aged 5-12 Years With SCA n = 111	Eligible Children Aged 5-12 Years Old Without SCD n = 22
Eligible	111	22
Randomized	110	22
Unable to randomize	1	0

Population analysed: 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.

Primary outcome: Retention Over 12-week Period

Time frame: 12 weeks. Number. Unit: participants

Measure	SCD - Ready-to-use Therapeutic Food and Hydroxyurea n = 56	SCD - Ready-to-use Therapeutic Food Alone n = 54	Siblings Without SCD n = 22
Voluntarily withdrew from the trial	0	0	0
Involuntary withdrawal from the trial	1	0	0

Population analysed: 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.

Primary outcome: Percentage of Ready-to-use Therapeutic Food Sachets Returned as Empty.

Time frame: 12 weeks. Mean (95% Confidence Interval). Unit: percentage of empty sachets

Measure	SCD - Ready-to-use Therapeutic Food and Hydroxyurea n = 54	SCD - Ready-to-use Therapeutic Food Alone n = 54	Siblings Without SCD n = 22
Value	99.02 (99.00 to 99.04)	99.03 (99.0 to 99.05)	99.02 (99.01 to 99.04)

Population analysed: 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.

Primary outcome: Number of Missed Visits

Time frame: 12 weeks. Number. Unit: missed visits

Measure	SCD - Ready-to-use Therapeutic Food and Hydroxyurea n = 54	SCD - Ready-to-use Therapeutic Food Alone n = 54	Siblings Without SCD n = 22
Value	0	0	0

Population analysed: Per-protocol analysis of participants.

1 secondary outcome is reported in the full results.

Adverse events

Time frame: The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.. Other adverse events reported at 2% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Ready-to-use Therapeutic Food and Hydroxyurea	1 of 56 (1.8%)	2 of 56 (3.6%)	1 of 56
Ready-to-use Therapeutic Food Alone	0 of 54 (0%)	4 of 54 (7.4%)	0 of 54
Siblings Without SCD	0 of 22 (0%)	0 of 22 (0%)	0 of 22

Limitations and caveats (from the sponsor): The sample size for this feasibility study was not determined with the goal of assessing differences in efficacy between the treatment arms. As a feasibility study, the sample size was not based on determining the difference in efficacy of the 2 treatment arms.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 41253090Murtala HA, Abdullahi SU, Gambo S, Kabir H, Shamsu KA, Gwarzo G, Acra SA, Stallings VA, Rodeghier M, DeBaun MR, Klein LJ. Including malnourished siblings in treatment improves nutritional outcomes for children with sickle cell anemia in Northern Nigeria: Results from a feasibility trial. Nutr Res. 2025 Dec;144:32-37. doi: 10.1016/j.nutres.2025.10.006. Epub 2025 Oct 18.
Derived from this study: PubMed 40780831Brechko A, Abdullahi SU, Gambo S, Murtala HA, Kabir H, Shamsu KA, Stallings VA, Rodeghier M, Ramesh A, Sellers A, Schlundt DG, DeBaun MR, Klein LJ. Adherence to ready-to-use therapeutic food in older children with sickle cell anaemia and severe acute malnutrition: a mixed-methods study in a low-income setting. BMJ Glob Health. 2025 Aug 7;10(8):e019096. doi: 10.1136/bmjgh-2025-019096.
Derived from this study: PubMed 40062628Ramirez-Cuebas G, Abdullahi SU, Gambo S, Murtala HA, Kabir H, Shamsu KA, Gwarzo G, Acra SA, Stallings VA, Rodeghier M, DeBaun MR, Klein LJ. Impact of Food Insecurity on Malnutrition Treatment Response in Nigerian Children With Sickle Cell Anemia and Severe Acute Malnutrition. Pediatr Blood Cancer. 2025 Jun;72(6):e31637. doi: 10.1002/pbc.31637. Epub 2025 Mar 10.
Derived from this study: PubMed 38268013Ritter C, Abdullahi SU, Gambo S, Murtala HA, Kabir H, Shamsu KA, Gwarzo G, Banaei Y, Acra SA, Stallings VA, Rodeghier M, DeBaun MR, Klein LJ. Impact of maternal depression on malnutrition treatment outcomes in older children with sickle cell anemia. BMC Nutr. 2024 Jan 24;10(1):18. doi: 10.1186/s40795-024-00826-0.
Derived from this study: PubMed 37428866Abdullahi SU, Gambo S, Murtala HA, Kabir H, Shamsu KA, Gwarzo G, Acra S, Stallings VA, Rodeghier M, DeBaun MR, Klein LJ. Feasibility trial for the management of severe acute malnutrition in older children with sickle cell anemia in Nigeria. Blood Adv. 2023 Oct 24;7(20):6024-6034. doi: 10.1182/bloodadvances.2023010789.
Individual participant dataSharing planned: No

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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.

Michael DeBaun principal investigator

Vanderbilt University Medical Center

3 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 7 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 27 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Nigeria, 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. Some participants in this study may receive a placebo. 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 27 Mar 2024). 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?

Enrollment Rate at the End of the 6-month Recruitment Period (time frame: 6 months). The study lists 10 primary outcomes in all. 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 NCT03634488, which also lists 5 linked publications.

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 27 Mar 2024.