Sickle Cell AnemiaBABY HUGNCT01783990

Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol

An observational study of Sickle Cell Anemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 14 sites in United States.

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

The BABY HUG Treatment Study was designed to see if treatment with the drug hydroxyurea (also called HU) in children with sickle cell disease could prevent organ damage, especially in the spleen and kidneys. There was also a chance that treatment could prevent painful crises, lung disease, stroke, and blood infection. Read more about this study

Who is it for?: Ages 24 months to 18 years
Conditions the study is for: Sickle Cell Anemia
What is being tested?: Hydroxyurea
How many people: 150 participants
Where: 14 sitesUnited States
Study dates: Oct 2012to 31 Dec 2016
Phase: Observational
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 24 months to 18 years
All sexes
No healthy volunteers
Child, Adult

Study population: All subjects enrolled in the BABY HUG Follow-Up I Study who participated for at least 24 months are eligible for the Follow-Up Study II.

Who may be able to join

All subjects enrolled in the BABY HUG Follow-Up I Study who participated for at least 24 months are eligible for the Follow-Up Study II

Who cannot join

Subjects that have received a Stem Cell Transplant are not eligible for enrollment

Trial locations and contacts

14 sites in 1 country.

City, postcode or hospital

14 sites in 1 country

United States14 sites

Emory University School of Medicine
Atlanta, Georgia, 30342
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205
Sinai Hospital of Baltimore Alfred I Coplan Pediatric Hematology Oncology Outpatient Center
Baltimore, Maryland, 21215
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Downstate Medical Center
Brooklyn, New York, 11203
Medical University of South Carolina
Charleston, South Carolina, 29425
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75390
Children's Hospital of Michigan/Wayne State University
Detroit, Michigan, 48201
Duke University Medical Center
Durham, North Carolina, 27710
University of Mississippi Medical Center
Jackson, Mississippi, 39216
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
University of Miami School of Medicine
Miami, Florida, 33136
Children's National Medical Center Center for Cancer and Blood Disorders
Washington D.C., District of Columbia, 20010
Howard University College of Medicine
Washington D.C., District of Columbia, 20060

Other trials in United States

About this study

Read the detailed description

The current observational trial, Follow-Up Study ((FUS) II includes enhanced neuropsychological, brain, cardiac, and pulmonary evaluations for this very well characterized cohort of subjects. Measures of spleen and renal function and markers of DNA damage will continue to be collected. Assessment of other target organs in sickle cell disease including pulmonary and cardiac function will be performed in addition to evaluation of developmental aspects of sickle cell disease (SCD) and potential HU toxicity.

Conditions: Sickle Cell Anemia
Being tested: Hydroxyurea
Medical subject headings: Anemia, Sickle Cell

What participants receive

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

HydroxyureaDrug
Parents and child's doctor may plan to use or not to use hydroxyurea.

Study groups in NCT01783990
Study group	Role	What they receive
Active		Complete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), and urine microalbumin:creatinine ratio were collected at study entry, annually, and exit to Follow-Up Study II. Variable-diversity-joining (VDJ) and a stored blood sample were collected at study entry and study exit. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, magnetic resonance imaging (MRI) / magnetic resonance angiography (MRA), cardiac echocardiogram, or neuropsychology testing were collected once during the study when the child was 10 years old. Receives: Drug: Hydroxyurea
Passive		Complete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), variable-diversity-joining (VDJ), urine microalbumin:creatinine ratio and a stored blood sample were collected at study entry and exit to Follow-Up Study II. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, MRI/MRA, cardiac echocardiogram, or neuropsychology testing were collected as part of clinical care. Receives: Drug: Hydroxyurea

What is being measured

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

Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo
The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between the randomized treatment groups (hydroxyurea vs placebo). The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) was defined as worse if it changed from normal to decreased or absent, or decreased to absent; and not worse if it changed from decreased to decreased, normal to normal, decreased to normal, absent to absent, or absent to decreased.
Measured: baseline and when child turned 10 years old
Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit
The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) was defined as worse if it changed from normal to decreased or absent, or decreased to absent; and not worse if it changed from decreased to decreased, normal to normal, decreased to normal, absent to absent, or absent to decreased.
Measured: baseline and when child turned 10 years old
Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo
The change in the percentage of pitted cell from randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between the randomized treatment groups (hydroxyurea vs placebo).
Measured: Baseline and End of follow-up II study (up to 13 years from randomization date)
Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit
The change in the percentage of pitted cell from the randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit.
Measured: Baseline and End of follow-up II study (up to 13 years from randomization date)
Change in Howell Jolly Body (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo
The change in Howell Jolly Body from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell Jolly Body was compared between the randomized treatment groups (hydroxyurea vs placebo).
Measured: Baseline and End of follow-up II study (up to 13 years from randomization date)
Change in Howell Jolly Body (HJB) Count From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea
The change in Howell Jolly Body count from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit.
Measured: Baseline and End of follow-up II study (up to 13 years from randomization date)

Timeline

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

Oct 201231 Dec 2016

First posted5 Feb 2013 (estimated)
Study startOct 2012
Primary completion31 Dec 2016
Study completion31 Dec 2016

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.
Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 150 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01783990
Sponsor's protocol ID: HHSN268201200023C
Acronym: BABY HUG
Status last verified: Sep 2016
First submitted: 1 Feb 2013
Last updated: 20 Aug 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

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 20 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol
ClinicalTrials.gov ID: NCT01783990
Acronym / sponsor ID: BABY HUG / HHSN268201200023C
Conditions: Sickle Cell Anemia
Study type: Observational
Recruitment status: Completed
Enrolment: 150 participants (actual)
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
Collaborators: National Institutes of Health (NIH)
Study start: Oct 2012
Primary completion: 31 Dec 2016 (actual)
Study completion: 31 Dec 2016 (actual)
First posted: 5 Feb 2013 (estimated)
Results: Posted 20 Aug 2020

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

Observational model: Cohort
Time perspective: Prospective
Number of arms: 2

Arm	Type	Receives
Active	–	Drug: HydroxyureaComplete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), and urine microalbumin:creatinine ratio were collected at study entry, annually, and exit to Follow-Up Study II. Variable-diversity-joining (VDJ) and a stored blood sample were collected at study entry and study exit. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, magnetic resonance imaging (MRI) / magnetic resonance angiography (MRA), cardiac echocardiogram, or neuropsychology testing were collected once during the study when the child was 10 years old.
Passive	–	Drug: HydroxyureaComplete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), variable-diversity-joining (VDJ), urine microalbumin:creatinine ratio and a stored blood sample were collected at study entry and exit to Follow-Up Study II. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, MRI/MRA, cardiac echocardiogram, or neuropsychology testing were collected as part of clinical care.

Who may qualify

Ages: 24 months to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult
Study population: All subjects enrolled in the BABY HUG Follow-Up I Study who participated for at least 24 months are eligible for the Follow-Up Study II.

Key inclusion criteria (1 listed)

All subjects enrolled in the BABY HUG Follow-Up I Study who participated for at least 24 months are eligible for the Follow-Up Study II

Key exclusion criteria (1 listed)

Subjects that have received a Stem Cell Transplant are not eligible for enrollment

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
HydroxyureaParents and child's doctor may plan to use or not to use hydroxyurea.	Drug	Active, Passive

Primary outcome

Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo
Time frame: baseline and when child turned 10 years old
The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between the randomized treatment groups (hydroxyurea vs placebo). The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) was defined as worse if it changed from normal to decreased or absent, or decreased to absent; and not worse if it changed from decreased to decreased, normal to normal, decreased to normal, absent to absent, or absent to decreased.
Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit
Time frame: baseline and when child turned 10 years old
The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) was defined as worse if it changed from normal to decreased or absent, or decreased to absent; and not worse if it changed from decreased to decreased, normal to normal, decreased to normal, absent to absent, or absent to decreased.
Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo
Time frame: Baseline and End of follow-up II study (up to 13 years from randomization date)
The change in the percentage of pitted cell from randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between the randomized treatment groups (hydroxyurea vs placebo).
Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit
Time frame: Baseline and End of follow-up II study (up to 13 years from randomization date)
The change in the percentage of pitted cell from the randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit.
Change in Howell Jolly Body (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo
Time frame: Baseline and End of follow-up II study (up to 13 years from randomization date)
The change in Howell Jolly Body from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell Jolly Body was compared between the randomized treatment groups (hydroxyurea vs placebo).
Change in Howell Jolly Body (HJB) Count From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea
Time frame: Baseline and End of follow-up II study (up to 13 years from randomization date)
The change in Howell Jolly Body count from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit.

Results and safety

Results posted to the registry on 20 Aug 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Active	130	125
Passive	20	18

Primary outcome: Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo

Time frame: baseline and when child turned 10 years old. Count of participants. Unit: Participants

Measure	Randomized to Hydroxyurea n = 58	Randomized to Placebo n = 50
Splenic function - worse from baseline	26	28
Splenic function - not worse from baseline	32	22

Randomized to Hydroxyurea vs Randomized to Placebo: p = 0.33, Fisher Exact

Population analysed: All subjects who had age 10 years and baseline spleen function measurement.

Primary outcome: Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit

Time frame: baseline and when child turned 10 years old. Count of participants. Unit: Participants

Measure	On Hydroxyurea at the Time of Visit n = 88	Off Hydroxyurea at the Time of Visit n = 17
Splenic function - worse from baseline	43	9
Splenic function - not worse from baseline	45	8

On Hydroxyurea at the Time of Visit vs Off Hydroxyurea at the Time of Visit: p = 0.80, Fisher Exact

Population analysed: All subjects who were known to be on hydroxyurea vs. off hydroxyurea at age 10 years, and had age 10 years and baseline spleen function measurement.

Primary outcome: Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo

Time frame: Baseline and End of follow-up II study (up to 13 years from randomization date). Median (Inter-Quartile Range). Unit: Percentage of pitted cell

Measure	Randomized to Hydroxyurea n = 70	Randomized to Placebo n = 68
Value	-1.60 (-5.00 to -0.60)	-1.75 (-6.25 to -0.40)

Randomized to Hydroxyurea vs Randomized to Placebo: p = 0.87, Kruskal-Wallis

Population analysed: All subjects who had end of study and baseline pitted cell measurement.

Primary outcome: Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit

Time frame: Baseline and End of follow-up II study (up to 13 years from randomization date). Median (Inter-Quartile Range). Unit: Percentage of pitted cell

Measure	On Hydroxyurea at the Time of Visit n = 116	Off Hydroxyurea at the Time of Visit n = 22
Value	-1.80 (-6.40 to -0.40)	-0.80 (-5.00 to -0.30)

On Hydroxyurea at the Time of Visit vs Off Hydroxyurea at the Time of Visit: p = 0.28, Kruskal-Wallis

Population analysed: All subjects who were known to be on hydroxyurea vs. off hydroxyurea at the end of study visit, and had end of study and baseline pitted cell count measurement.

Adverse events

Time frame: The date of consent to follow-up study II (FUS-II) through end of FUS-II, up to 4 years. Other adverse events reported at 2% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Randomized to Hydroxyurea	24 of 77 (31.2%)	0 of 77 (0%)	0 of 77
Randomized to Placebo	31 of 73 (42.5%)	0 of 73 (0%)	0 of 73

Limitations and caveats (from the sponsor): Most children used hydroxyurea (HU) for at least part of the FUS-II observation period. This may dilute any effect of randomized group. Most children were on HU at the specified time points, which reduces power to compare on/off HU.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Sickle Cell Anemia

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
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RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
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Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
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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.

Susan Assmann, PhD principal investigator

New England Research Institutes, Watertown, MA

14 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Sickle Cell Anemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 14 sites in United States.

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 1 inclusion and 1 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 20 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Aug 2020). 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?

Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo (time frame: baseline and when child turned 10 years old). The study lists 6 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 NCT01783990.

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 20 Aug 2020.