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NCT03634488

Management of Severe Acute Malnutrition in SCD, in Northern Nigeria

Completed Phase 2 Results posted Last updated 27 March 2024
What this trial tests

Phase 2 trial testing hydroxyurea (20mg/kg/day) in Sickle Cell Anemia in 132 participants. Completed in 9 November 2022.

Timeline
18 August 2021
Primary endpoint
5 October 2022
9 November 2022

Quick facts

Lead sponsorVanderbilt University Medical Center
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingsingle
Primary purposetreatment
Enrollment132
Start date18 August 2021
Primary completion5 October 2022
Estimated completion9 November 2022
Sites3 locations across United States, Nigeria

Drugs / interventions tested

Conditions studied

Sponsor

Vanderbilt University Medical Center

Who can join

Adults 5 to 12, any sex, with Sickle Cell Anemia or Severe Acute Malnutrition. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Enrollment Rate at the End of the 6-month Recruitment Period Primary · 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.

Eligible
GroupValue95% CI
Eligible Children Aged 5-12 Years With SCA111
Eligible Children Aged 5-12 Years Old Without SCD22
Randomized
GroupValue95% CI
Eligible Children Aged 5-12 Years With SCA110
Eligible Children Aged 5-12 Years Old Without SCD22
Unable to randomize
GroupValue95% CI
Eligible Children Aged 5-12 Years With SCA1
Eligible Children Aged 5-12 Years Old Without SCD0
Retention Over 12-week Period Primary · 12 weeks

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

Voluntarily withdrew from the trial
GroupValue95% CI
SCD - Ready-to-use Therapeutic Food and Hydroxyurea0
SCD - Ready-to-use Therapeutic Food Alone0
Siblings Without SCD0
Involuntary withdrawal from the trial
GroupValue95% CI
SCD - Ready-to-use Therapeutic Food and Hydroxyurea1
SCD - Ready-to-use Therapeutic Food Alone0
Siblings Without SCD0
Percentage of Ready-to-use Therapeutic Food Sachets Returned as Empty. Primary · 12 weeks

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

GroupValue95% CI
SCD - Ready-to-use Therapeutic Food and Hydroxyurea99.0299.00 – 99.04
SCD - Ready-to-use Therapeutic Food Alone99.0399.0 – 99.05
Siblings Without SCD99.0299.01 – 99.04
Number of Missed Visits Primary · 12 weeks

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

GroupValue95% CI
SCD - Ready-to-use Therapeutic Food and Hydroxyurea0
SCD - Ready-to-use Therapeutic Food Alone0
Siblings Without SCD0
Percentage of Hydroxyurea Pills Returned Primary · 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.

GroupValue95% CI
SCD - Ready-to-use Therapeutic Food and Hydroxyurea4.40.00 – 8.27
Change in Mean Corpuscular Volume Primary · 12 weeks

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

GroupValue95% CI
Ready-to-use Therapeutic Food and Hydroxyurea5.4± 12.7
Ready-to-use Therapeutic Food Alone-1.0± 10.9
Change in Fetal Hemoglobin Level Percentage Primary · 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.

GroupValue95% CI
SCD - Ready-to-use Therapeutic Food and Hydroxyurea3.91.7 – 6.8
Ready-to-use Therapeutic Food Alone2.20.5 – 4.9
Mean Corpuscular Volume Values at Exit Primary · 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).

GroupValue95% CI
Ready-to-use Therapeutic Food and Hydroxyurea88.5± 14.7
Ready-to-use Therapeutic Food Alone85.4± 11.4
Fetal Hemoglobin Levels at Exit Primary · 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).

GroupValue95% CI
Ready-to-use Therapeutic Food and Hydroxyurea12.27.5 – 16.9
Ready-to-use Therapeutic Food Alone9.45.6 – 13.8
Total Hemoglobin Levels at Exit Primary · 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).

GroupValue95% CI
Ready-to-use Therapeutic Food and Hydroxyurea8.3± 1.1
Ready-to-use Therapeutic Food Alone7.7± 1.3
Percentage of Participants Maintaining a BMI Z-score Less Than -3.0 Secondary · 12 weeks

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.

GroupValue95% CI
Ready-to-use Therapeutic Food and Hydroxyurea66.7
Ready-to-use Therapeutic Food Alone55.6
Siblings Without SCD30.1

Adverse events — posted to ClinicalTrials.gov

Time frame: The adverse event data was collected during the participant's 12-week study period, about 7 months after enrollment.. Reporting threshold: 2%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ready-to-use Therapeutic Food and Hydroxyurea
Serious: 1/56 (2%)
Deaths: 1/56
Ready-to-use Therapeutic Food Alone
Serious: 0/54 (0%)
Deaths: 0/54
Siblings Without SCD
Serious: 0/22 (0%)
Deaths: 0/22

Serious adverse events (1 terms)

ReactionSystemReady-to-use Therapeutic F…Ready-to-use Therapeutic F…Siblings Without SCD
Prolonged hospitalizationBlood and lymphatic system disorders
Other adverse events (6 terms — click to expand)

ReactionSystemReady-to-use Therapeutic F…Ready-to-use Therapeutic F…Siblings Without SCD
FeverGeneral disorders
MalariaInfections and infestations
PainBlood and lymphatic system disorders
Acute Chest SyndromeBlood and lymphatic system disorders
Elective SurgeryEar and labyrinth disorders
AnemiaBlood and lymphatic system disorders

Most-reported serious reactions: Prolonged hospitalization.

Data from ClinicalTrials.gov NCT03634488 adverse events section.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Hydroxyurea (hydroxycarbamide) for sickle cell disease.
    Rankine-Mullings AE, Nevitt SJ. · · 2022 · cited 42× · PMID 36047926 · DOI 10.1002/14651858.cd002202.pub3
  2. Feasibility trial for the management of severe acute malnutrition in older children with sickle cell anemia in Nigeria.
    Abdullahi SU, Gambo S, Murtala HA, Kabir H, et al · · 2023 · cited 7× · PMID 37428866 · DOI 10.1182/bloodadvances.2023010789
  3. Including malnourished siblings in treatment improves nutritional outcomes for children with sickle cell anemia in Northern Nigeria: Results from a feasibility trial.
    Murtala HA, Abdullahi SU, Gambo S, Kabir H, et al · · 2025 · PMID 41253090 · DOI 10.1016/j.nutres.2025.10.006
  4. 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.
    Brechko A, Abdullahi SU, Gambo S, Murtala HA, et al · · 2025 · PMID 40780831 · DOI 10.1136/bmjgh-2025-019096
  5. Impact of Food Insecurity on Malnutrition Treatment Response in Nigerian Children With Sickle Cell Anemia and Severe Acute Malnutrition.
    Ramirez-Cuebas G, Abdullahi SU, Gambo S, Murtala HA, et al · · 2025 · PMID 40062628 · DOI 10.1002/pbc.31637
  6. Impact of maternal depression on malnutrition treatment outcomes in older children with sickle cell anemia.
    Ritter C, Abdullahi SU, Gambo S, Murtala HA, et al · · 2024 · PMID 38268013 · DOI 10.1186/s40795-024-00826-0

Verify or expand the search:

Other recruiting trials for Sickle Cell Anemia

Currently open trials in the same condition.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03634488.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing