Trial Outcomes & Findings for Preemie Tots: A Pilot Study to Understand the Effects of Prematurity in Toddlerhood (NCT NCT01683565)

Last Updated: 2019-01-03

Results Overview

The Pervasive Developmental Disorders Screening Test-II stage 2 (PDDST-II) is a 14-item measure designed to discriminate Autism Spectrum Disorders from a related non-autistic developmental disorder in children ages 12 to 48 months old. The min score is 0 and the max score is 14. Scores equal to or greater than five yield a positive screen for autism. PDDST-II scores were measured at baseline and then again at study completion (90 days post randomization). The scores were calculated as the PDDST-II value at 90 days minus the PDDST-II value at baseline.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

31 participants

Primary outcome timeframe

Baseline to 90 days post randomization

Results posted on

2019-01-03

Participant Flow

Participant milestones

Participant milestones
Measure	Long Chain Polyunsaturated Fatty Acid (LCPUFA) Oil Supplement Eicosapentaenoic acid (EPA) + Docosahexaenoic acid (DHA) \+ Gamma-Linolenic acid (GLA) + Oleic Acid (OA) LCPUFA oil supplement: 2.5mL per day for 90 days Daily Dose: EPA (338mg) + DHA (225mg) + GLA (83mg) + OA (200mg	Canola Oil Placebo Canola Oil Placebo: 2.5mL per day for 90 days
Overall Study STARTED	15	16
Overall Study COMPLETED	12	15
Overall Study NOT COMPLETED	3	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Long Chain Polyunsaturated Fatty Acid (LCPUFA) Oil Supplement Eicosapentaenoic acid (EPA) + Docosahexaenoic acid (DHA) \+ Gamma-Linolenic acid (GLA) + Oleic Acid (OA) LCPUFA oil supplement: 2.5mL per day for 90 days Daily Dose: EPA (338mg) + DHA (225mg) + GLA (83mg) + OA (200mg	Canola Oil Placebo Canola Oil Placebo: 2.5mL per day for 90 days
Overall Study Lost to Follow-up	2	0
Overall Study Discontinued Intervention	1	1

Baseline Characteristics

Preemie Tots: A Pilot Study to Understand the Effects of Prematurity in Toddlerhood

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Long Chain Polyunsaturated Fatty Acid (LCPUFA) Oil Supplement n=15 Participants Eicosapentaenoic acid (EPA) + Docosahexaenoic acid (DHA) \+ Gamma-Linolenic acid (GLA) + Oleic Acid (OA) LCPUFA oil supplement: 2.5mL per day for 90 days Daily Dose: EPA (338mg) + DHA (225mg) + GLA (83mg) + OA (200mg	Canola Oil Placebo n=16 Participants Canola Oil Placebo: 2.5mL per day for 90 days	Total n=31 Participants Total of all reporting groups
Age, Categorical <=18 years	15 Participants n=5 Participants	16 Participants n=7 Participants	31 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	30 Months n=5 Participants	25 Months n=7 Participants	27 Months n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	3 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	13 Participants n=7 Participants	21 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants	16 participants n=7 Participants	31 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to 90 days post randomization

Outcome measures

Outcome measures
Measure	Long Chain Polyunsaturated Fatty Acid (LCPUFA) Oil Supplement n=15 Participants Eicosapentaenoic acid (EPA) + Docosahexaenoic acid (DHA) \+ Gamma-Linolenic acid (GLA) + Oleic Acid (OA) LCPUFA oil supplement: 2.5mL per day for 90 days Daily Dose: EPA (338mg) + DHA (225mg) + GLA (83mg) + OA (200mg	Canola Oil Placebo n=16 Participants Canola Oil Placebo: 2.5mL per day for 90 days
Child Behavior - Pervasive Developmental Disorders Screening Test-II Stage 2	-1.0 score on a scale Standard Deviation 2.6	-0.2 score on a scale Standard Deviation 1.9

PRIMARY outcome

Timeframe: Baseline to 90 days post randomization

Brief Infant-Toddler Social and Emotional Assessment (BITSEA) is a 42 item tool that is useful for identifying social-emotional problems and/or deficits in children. BITSEA includes the following subscales: Competence (11 Items, min score:0, max score:22), problem behaviors--dysregulation (8 items, min score:0, max score:16) , externalizing (6 items, min score:0, max score:12), internalizing (8 items, min score:0, max score:16), Autism Spectrum Disorder (17 Items, min score:0, max score:34), and Red Flags (14 items, min score:0, max score:28).The questions overlap and the problem subscale is a combination of dysregulation, externalizing, and internalizing. Higher problem scores indicate greater levels of social-emotional/behavioral problems. Lower Competence scores indicate possible delay/deficit. Scores were measured at baseline and then again at study completion (90 days post randomization). The change in BITSEA scores were calculated as the value at 90 days - the value at baseline.

Outcome measures

Outcome measures
Measure	Long Chain Polyunsaturated Fatty Acid (LCPUFA) Oil Supplement n=15 Participants Eicosapentaenoic acid (EPA) + Docosahexaenoic acid (DHA) \+ Gamma-Linolenic acid (GLA) + Oleic Acid (OA) LCPUFA oil supplement: 2.5mL per day for 90 days Daily Dose: EPA (338mg) + DHA (225mg) + GLA (83mg) + OA (200mg	Canola Oil Placebo n=16 Participants Canola Oil Placebo: 2.5mL per day for 90 days
Child Behavior-Brief Infant-Toddler Social and Emotional Assessment (BITSEA) Competence	1.9 score on a scale Standard Deviation 3.2	0.9 score on a scale Standard Deviation 3.6
Child Behavior-Brief Infant-Toddler Social and Emotional Assessment (BITSEA) Problems	3.2 score on a scale Standard Deviation 6.1	-3.2 score on a scale Standard Deviation 5.0
Child Behavior-Brief Infant-Toddler Social and Emotional Assessment (BITSEA) Dysregulation	-0.9 score on a scale Standard Deviation 2.3	-0.8 score on a scale Standard Deviation 2.2
Child Behavior-Brief Infant-Toddler Social and Emotional Assessment (BITSEA) Externalizing	0.3 score on a scale Standard Deviation 1.3	-0.7 score on a scale Standard Deviation 2.3
Child Behavior-Brief Infant-Toddler Social and Emotional Assessment (BITSEA) Internalizing	-0.9 score on a scale Standard Deviation 2.3	-0.5 score on a scale Standard Deviation 2.3
Child Behavior-Brief Infant-Toddler Social and Emotional Assessment (BITSEA) Austism Spectrum Disorder	-1.5 score on a scale Standard Deviation 2.6	0.8 score on a scale Standard Deviation 3.8
Child Behavior-Brief Infant-Toddler Social and Emotional Assessment (BITSEA) Red Flag	-2.5 score on a scale Standard Deviation 3.8	-1.0 score on a scale Standard Deviation 2.8

SECONDARY outcome

Timeframe: Baseline to 90 days post randomization

Population: Rows titles are fatty acids with different C:D ratio where, "C" stands for Carbohydrate; "D" stands for Double bond; "C:D" is the ratio of the total amount of Carbon atoms of the fatty acid in relation to the number of double (unsaturated) bonds in it.

The secondary outcome measures in this trial involve an examination of change in fatty acids from the first study visit to the final study visit.

Outcome measures

Outcome measures
Measure	Long Chain Polyunsaturated Fatty Acid (LCPUFA) Oil Supplement n=13 Participants Eicosapentaenoic acid (EPA) + Docosahexaenoic acid (DHA) \+ Gamma-Linolenic acid (GLA) + Oleic Acid (OA) LCPUFA oil supplement: 2.5mL per day for 90 days Daily Dose: EPA (338mg) + DHA (225mg) + GLA (83mg) + OA (200mg	Canola Oil Placebo n=15 Participants Canola Oil Placebo: 2.5mL per day for 90 days
Fatty Acid 16:1n-7	7.83 nmol/mL Standard Deviation 9.90	27.0 nmol/mL Standard Deviation 17.75
Fatty Acid 18:0	138 nmol/mL Standard Deviation 69.25	-40.3 nmol/mL Standard Deviation 99.18
Fatty Acid 18:1n-9	80.5 nmol/mL Standard Deviation 46.66	-21.6 nmol/mL Standard Deviation 50.19
Fatty Acid 10:0	2.15 nmol/mL Standard Deviation 3.23	-4.91 nmol/mL Standard Deviation 7.37
Fatty Acid 12:0	2.56 nmol/mL Standard Deviation 2.90	-2.43 nmol/mL Standard Deviation 6.68
Fatty Acid 14:0	-3.07 nmol/mL Standard Deviation 4.62	-8.42 nmol/mL Standard Deviation 6.84
Fatty Acid 16:0	177 nmol/mL Standard Deviation 99.60	-28.0 nmol/mL Standard Deviation 116.05
Fatty Acid 18:1n-7	11.0 nmol/mL Standard Deviation 4.98	-2.13 nmol/mL Standard Deviation 6.23
Fatty Acid 18:2n-6	18.7 nmol/mL Standard Deviation 69.83	-8.72 nmol/mL Standard Deviation 56.53
Fatty Acid 18:3n-6	1.69 nmol/mL Standard Deviation 2.54	-4.29 nmol/mL Standard Deviation 5.36
Fatty Acid 18:3n-3	2.44 nmol/mL Standard Deviation 2.19	-3.81 nmol/mL Standard Deviation 5.50
Fatty Acid 18:4n-3	-0.15 nmol/mL Standard Deviation 1.19	-3.44 nmol/mL Standard Deviation 3.75
Fatty Acid 20:3n-6	6.24 nmol/mL Standard Deviation 9.05	-17.1 nmol/mL Standard Deviation 15.30
Fatty Acid 20:4n-6	60.3 nmol/mL Standard Deviation 57.74	-7.72 nmol/mL Standard Deviation 70.23
Fatty Acid 20:5n-3	38.7 nmol/mL Standard Deviation 8.21	-2.73 nmol/mL Standard Deviation 12.90
Fatty Acid 22:5n-3	-3.27 nmol/mL Standard Deviation 4.88	-2.84 nmol/mL Standard Deviation 4.97
Fatty Acid 22:6n-3	109 nmol/mL Standard Deviation 26.50	-1.44 nmol/mL Standard Deviation 26.69
Fatty Acid total omega-6	86.9 nmol/mL Standard Deviation 122.71	-37.9 nmol/mL Standard Deviation 138.07
Fatty Acid total omega-3	146 nmol/mL Standard Deviation 34.13	-14.3 nmol/mL Standard Deviation 43.64

OTHER_PRE_SPECIFIED outcome

Timeframe: Pre-baseline to 90 days post randomization

Feasibility of a future full-scale multi-site study is assessed by examining the number of children screened, the proportion of these children who screen positive for ASD, the number of children who agree to participate in the trial, the number of children who return for the second and third study visits, baseline differences in individual fatty acids between the intervention and comparison groups, and adherence to the assigned treatment.

Outcome measures

Outcome measures
Measure	Long Chain Polyunsaturated Fatty Acid (LCPUFA) Oil Supplement n=15 Participants Eicosapentaenoic acid (EPA) + Docosahexaenoic acid (DHA) \+ Gamma-Linolenic acid (GLA) + Oleic Acid (OA) LCPUFA oil supplement: 2.5mL per day for 90 days Daily Dose: EPA (338mg) + DHA (225mg) + GLA (83mg) + OA (200mg	Canola Oil Placebo n=16 Participants Canola Oil Placebo: 2.5mL per day for 90 days
Feasibility: Future Full-scale Multi-site Study	15 Participants	16 Participants

Adverse Events

LCPUFA Oil Supplement

Serious events: 0 serious events

Other events: 15 other events

Deaths: 0 deaths

Canola Oil Placebo

Serious events: 0 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	LCPUFA Oil Supplement n=15 participants at risk EPA + DHA + GLA + OA oil supplement LCPUFA oil supplement: 2.5mL per day for 90 days Daily Dose: EPA (338mg) + DHA (225mg) + GLA (83mg) + OA (200mg)	Canola Oil Placebo n=16 participants at risk Canola Oil Placebo: 2.5mL per day for 90 days
Gastrointestinal disorders Gastrointestinal Symptoms	66.7% 10/15 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.	68.8% 11/16 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.
Ear and labyrinth disorders Ear Nose Throat Symptoms	40.0% 6/15 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.	56.2% 9/16 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.
Respiratory, thoracic and mediastinal disorders Respiratory symptoms	26.7% 4/15 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.	43.8% 7/16 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.
Skin and subcutaneous tissue disorders Skin and Limb Symptoms	26.7% 4/15 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.	6.2% 1/16 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.
Psychiatric disorders Behavioral Symptoms	20.0% 3/15 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.	18.8% 3/16 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.
Infections and infestations Cold/Fever	13.3% 2/15 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.	31.2% 5/16 • Baseline to 90 days post randomization Information to be collected includes event description, time of onset, Study Doctor's assessment of severity, relationship to study product,and time of resolution/stabilization of the event. All AEs occurring during study participation must be documented appropriately regardless of relationship.

Additional Information

Dr. Sarah Keim

Nationwide Children's Hospital

Phone: 614-722-3177

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place