Trial Outcomes & Findings for Oral Supplementation of 2'-Fucosyllactose in Allogeneic Bone Marrow Transplant Recipients (NCT NCT04263597)
NCT ID: NCT04263597
Last Updated: 2026-09-02
Results Overview
Change in fecal butyrate levels from baseline at day+ 7
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
70 participants
Primary outcome timeframe
Day+ 7 after transplant
Results posted on
2026-09-02
Participant Flow
Participant milestones
| Measure |
Phase II: 2'-Fucosyllactose for Ages >10 Years, 10g
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase I: 2'-Fucosyllactose for Ages 0-5 Years, 2.5g
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase I: 2'-Fucosyllactose for Ages 5.1-10 Years, 5g
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase I: 2'-Fucosyllactose for Ages >10 Years, 10g
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase II: 2'-Fucosyllactose for Ages 0-5 Years, 2.5g
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase II: 2'-Fucosyllactose for Ages 5.1-10 Years, 5g
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
18
|
8
|
7
|
11
|
14
|
12
|
|
Overall Study
COMPLETED
|
15
|
8
|
7
|
10
|
14
|
11
|
|
Overall Study
NOT COMPLETED
|
3
|
0
|
0
|
1
|
0
|
1
|
Reasons for withdrawal
| Measure |
Phase II: 2'-Fucosyllactose for Ages >10 Years, 10g
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase I: 2'-Fucosyllactose for Ages 0-5 Years, 2.5g
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase I: 2'-Fucosyllactose for Ages 5.1-10 Years, 5g
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase I: 2'-Fucosyllactose for Ages >10 Years, 10g
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase II: 2'-Fucosyllactose for Ages 0-5 Years, 2.5g
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Phase II: 2'-Fucosyllactose for Ages 5.1-10 Years, 5g
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
0
|
1
|
0
|
1
|
|
Overall Study
Physician Decision
|
1
|
0
|
0
|
0
|
0
|
0
|
|
Overall Study
Screen Failure
|
2
|
0
|
0
|
0
|
0
|
0
|
Baseline Characteristics
Oral Supplementation of 2'-Fucosyllactose in Allogeneic Bone Marrow Transplant Recipients
Baseline characteristics by cohort
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=23 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
n=19 Participants
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
n=28 Participants
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
Total
n=70 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
23 Participants
n=136 Participants
|
19 Participants
n=136 Participants
|
20 Participants
n=272 Participants
|
62 Participants
n=60 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
8 Participants
n=272 Participants
|
8 Participants
n=60 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
0 Participants
n=60 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=136 Participants
|
7 Participants
n=136 Participants
|
13 Participants
n=272 Participants
|
28 Participants
n=60 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=136 Participants
|
12 Participants
n=136 Participants
|
15 Participants
n=272 Participants
|
42 Participants
n=60 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
0 Participants
n=60 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
3 Participants
n=272 Participants
|
3 Participants
n=60 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
0 Participants
n=60 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=136 Participants
|
2 Participants
n=136 Participants
|
2 Participants
n=272 Participants
|
5 Participants
n=60 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=136 Participants
|
17 Participants
n=136 Participants
|
19 Participants
n=272 Participants
|
58 Participants
n=60 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
1 Participants
n=272 Participants
|
1 Participants
n=60 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
3 Participants
n=272 Participants
|
3 Participants
n=60 Participants
|
|
Region of Enrollment
United States
|
23 participants
n=136 Participants
|
19 participants
n=136 Participants
|
28 participants
n=272 Participants
|
70 participants
n=60 Participants
|
PRIMARY outcome
Timeframe: Day+100 after transplantPopulation: per protocol only patients with \>/= 60% adherence to 2FL were evaluable.
Patients with a bloodstream infection occurring anytime between first day of 2-FL dosing until day+100 after transplant were collected. Typically 2FL was started with start of chemotherapy which was usually up to 22 days before transplant
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=16 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
n=12 Participants
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
n=15 Participants
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Number of Bloodstream Infections
|
1 Participants
|
0 Participants
|
4 Participants
|
PRIMARY outcome
Timeframe: 1 week prior to start of chemotherapy until day+30 after transplant (transplant occurred up to 22 days from start of chemotherapy)Population: Details of adherence are provided broken down by Phase I and Phase II and further by Arms.
Daily drug doses were documented by patients or nursing staff as applicable. Patients who took at least 60% of their total planned doses were deemed to meet protocol defined adherence to 2-FL
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=23 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
n=19 Participants
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
n=28 Participants
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Number of Patients Able to Take 2FL
|
15 Participants
|
13 Participants
|
15 Participants
|
PRIMARY outcome
Timeframe: Day+ 7 after transplantPopulation: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens and small subgroup sizes precluded meaningful statistical comparisons. Participants meeting protocol-defined adherence threshold (\>60%) were included.
Change in fecal acetate levels from baseline at day+ 7
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=16 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Change in Fecal Acetate Levels
|
-8.25 u mol/gram of stool sample
Interval -18.88 to 14.86
|
—
|
—
|
PRIMARY outcome
Timeframe: Day+ 7 after transplantPopulation: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens and small subgroup sizes precluded meaningful statistical comparisons. Participants meeting protocol-defined adherence threshold (\>60%) were included
Change in fecal propionate levels from baseline at day+ 7
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=16 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Change in Fecal Propionate Levels
|
-1.45 u mol/gram of stool sample
Interval -6.17 to 0.65
|
—
|
—
|
PRIMARY outcome
Timeframe: Day+ 7 after transplantPopulation: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens and small subgroup sizes precluded meaningful statistical comparisons. Participants meeting protocol-defined adherence threshold (\>60%) were included
Change in fecal butyrate levels from baseline at day+ 7
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=16 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Change in Fecal Butyrate Levels
|
-1.55 u mol/gram of stool sample
Interval -3.2 to 1.1
|
—
|
—
|
SECONDARY outcome
Timeframe: Day+30 after transplantPopulation: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens (n=12) precluded meaningful statistical comparisons. Only participants meeting the protocol-defined adherence threshold (\>60%) were included
Stool underwent shotgun metagenomic sequencing and Kraken2 taxonomic classification; read counts were rarefied to a common depth. For each participant, the relative abundance (percentage of total classified reads) of the genus Bifidobacterium was calculated at baseline and at Day +30; phylum values were obtained by summing all constituent genera. The change from baseline (Day +30 minus baseline) was computed for participants with paired samples. Values are summarized as median \[interquartile range\].
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=12 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Relative Abundance of Fecal Bifidobacteria at Day+30 Compared to Baseline
|
-1.66 percentage of total classified reads (%)
Interval -13.7 to -0.02
|
—
|
—
|
SECONDARY outcome
Timeframe: Day+30 after transplantPopulation: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited small subgroup sizes precluded meaningful statistical comparisons. Only participants meeting the protocol-defined adherence included.
Stool underwent shotgun metagenomic sequencing and Kraken2 taxonomic classification; read counts were rarefied to a common depth. For each participant, the relative abundance (percentage of total classified reads) of the phyla Firmicutes was calculated at baseline and at Day +30; phylum values were obtained by summing all constituent genera. The change from baseline (Day +30 minus baseline) was computed for participants with paired samples. Values are summarized as median \[interquartile range\].
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=12 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Relative Abundance of Fecal Firmicutes at Day+30 Compared to Baseline for Patients on 2FL
|
0.19 Percent of total classified reads
Interval -19.03 to 22.72
|
—
|
—
|
SECONDARY outcome
Timeframe: Day+100 after transplantPopulation: Grades I-IV acute GVHD
Incidence of acute GVHD in patients on 2FL
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=16 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
n=12 Participants
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
n=15 Participants
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Incidence of Acute GVHD
|
1 Participants
|
3 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Day+ 100 after transplantIncidence of mucosal barrier injury laboratory confirmed bloodstream infections were collected from first dose of 2-FL until day+100 after transplant in evaluable patients
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=16 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
n=12 Participants
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
n=15 Participants
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Incidence of Mucosal Barrier Injury Laboratory-confirmed Bloodstream Infection (MBI-LCBI)
|
1 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: BaselinePopulation: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens and small subgroup sizes precluded meaningful statistical comparisons. Participants meeting protocol-defined adherence threshold (\>60%) were included.
Unit of measure is urine 3-indole sulfate levels in mM normalized to urine creatinine
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=35 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Urine 3-indoxyl Sulfate (3-IS) Levels
|
0.03 mM/mM urine creatinine
Interval 0.02 to 0.06
|
—
|
—
|
SECONDARY outcome
Timeframe: day+7Population: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens and small subgroup sizes precluded meaningful statistical comparisons. Participants meeting protocol-defined adherence threshold (\>60%) were included
Unit of measure is urine 3-indole sulfate levels in mM normalized to urine creatinine
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=29 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Urine 3-indoxyl Sulfate (3-IS) Levels
|
0.05 mM/mM urine creatinine
Interval 0.03 to 0.09
|
—
|
—
|
SECONDARY outcome
Timeframe: day+14Population: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens and small subgroup sizes precluded meaningful statistical comparisons. Participants meeting protocol-defined adherence threshold (\>60%) were included.
Unit of measure is urine 3-indole sulfate levels in mM normalized to urine creatinine
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=34 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Urine 3-indoxyl Sulfate (3-IS) Levels
|
0.04 mM/mM urine creatinine
Interval 0.03 to 0.08
|
—
|
—
|
SECONDARY outcome
Timeframe: Day+30Population: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens and small subgroup sizes precluded meaningful statistical comparisons. Participants meeting protocol-defined adherence threshold (\>60%) were included
Unit of measure is urine 3-indole sulfate levels in mM normalized to urine creatinine
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=25 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Urine 3-indoxyl Sulfate (3-IS) Levels
|
0.03 mM/mM urine creatinine
Interval 0.02 to 0.03
|
—
|
—
|
SECONDARY outcome
Timeframe: Day+30 after transplantPopulation: Correlative analyses combined Phase I and II participants because this pilot study was designed to detect biological signals rather than compare age groups or study phases as prespecified. Age-based dosing was used to achieve similar 2'-FL exposure across ages and was not intended to create biologically distinct groups. Limited paired biospecimens and small subgroup sizes precluded meaningful statistical comparisons. Participants meeting protocol-defined adherence threshold (\>60%) were included.
Stool underwent shotgun metagenomic sequencing and Kraken2 taxonomic classification; read counts were rarefied to a common depth. For each participant, the relative abundance (percentage of total classified reads) of the phyla Proteobacteria was calculated at baseline and at Day +30); phylum values were obtained by summing all constituent genera. The change from baseline (Day +30 minus baseline) was computed for participants with paired samples. Values are summarized as median \[interquartile range\].
Outcome measures
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=12 Participants
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Relative Abundance of Fecal Proteobacteria at Day+30 Compared to Baseline for Patients on 2FL
|
4.33 percentage of total classified reads (%)
Interval -3.08 to 28.1
|
—
|
—
|
Adverse Events
2'-Fucosyllactose for Ages 0-5 Years
Serious events: 7 serious events
Other events: 16 other events
Deaths: 0 deaths
2'-Fucosyllactose for Ages 5.1-10 Years
Serious events: 3 serious events
Other events: 12 other events
Deaths: 0 deaths
2'-Fucosyllactose for Ages >10 Years
Serious events: 8 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=23 participants at risk
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
n=18 participants at risk
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
n=26 participants at risk
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia, Grade 3
|
13.0%
3/23 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
11.5%
3/26 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia, Grade 4
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Skin and subcutaneous tissue disorders
Other - Rash, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Vascular disorders
Hypotension, Grade 3
|
8.7%
2/23 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Vascular disorders
Hypotension, Grade 4
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
7.7%
2/26 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Sepsis, Grade 4
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Renal and urinary disorders
Acute Kidney Injury, Grade 4
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Gastrointestinal disorders
GI Hemorrhage, Grade 4
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
General disorders
Fever, Grade 1
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Renal and urinary disorders
Acute Kidney Injury, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Skin and subcutaneous tissue disorders
Pruritus, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Hepatobiliary disorders
Sinusoidal Obstruction Syndrome, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Musculoskeletal and connective tissue disorders
Extremity Pain - Bilateral Leg, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Gastrointestinal disorders
Diarrhea, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Respiratory, thoracic and mediastinal disorders
Diffuse Alveolar Hemorrhage, Grade 4
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
Other adverse events
| Measure |
2'-Fucosyllactose for Ages 0-5 Years
n=23 participants at risk
Dose for ages 0-5 years: 2.5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages 5.1-10 Years
n=18 participants at risk
Dose for ages 5.1-10 years: 5 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
2'-Fucosyllactose for Ages >10 Years
n=26 participants at risk
Dose for ages \>10 years: 10 g/day;
2'-fucosyllactose: 2FL powder will be provided to participants randomized to receive 2FL in packets. They will be instructed to drink this daily by adding the required amount to food or drink. It may also be mixed in standard feeds or mixed with water and administered by enteral tube, whenever applicable.
|
|---|---|---|---|
|
Infections and infestations
Enterococcus Faecalis Bacteremia, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
11.5%
3/26 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Metabolism and nutrition disorders
Hypokalemia, Grade 4
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Rothis Mucilaginosa Bactermia, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Staphylococcal Epidermis, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Staphylococcus Bacteremia, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
COVID-19, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
16.7%
3/18 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Investigations
Blood Bilirubin Increased, Grade 3
|
17.4%
4/23 • Number of events 5 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
16.7%
3/18 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
7.7%
2/26 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia, Grade 3
|
21.7%
5/23 • Number of events 5 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
22.2%
4/18 • Number of events 5 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
30.8%
8/26 • Number of events 9 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Respiratory, thoracic and mediastinal disorders
Idiopathic Pulmonary Syndrome, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Vascular disorders
Hematoma - Mediastinal, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Staphylococcus Aureus Bacteremia, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Vascular disorders
TA-TMA, Grade 3
|
13.0%
3/23 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Metabolism and nutrition disorders
Hyponatremia, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Cytomegalovirus infection reactivation, Grade 3
|
8.7%
2/23 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
7.7%
2/26 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Renal and urinary disorders
Acute Kidney Injury, Grade 4
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Gastrointestinal disorders
Mucositis, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Investigations
ALT increased, Grade 3
|
13.0%
3/23 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
16.7%
3/18 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
11.5%
3/26 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Investigations
AST increased, Grade 3
|
8.7%
2/23 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
27.8%
5/18 • Number of events 5 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
7.7%
2/26 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Vascular disorders
Hypotension, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
11.5%
3/26 • Number of events 3 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Eye disorders
Other - Worsening Kertopathy and corneal epithelial defect, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Nervous system disorders
Seizure, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Bacteroides fragilis, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Enterobacter cloacae, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Streptococcus, Grade 4
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Renal and urinary disorders
BK Cystitis, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
11.1%
2/18 • Number of events 2 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Investigations
Thrombocytopenia, Grade 4
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
General disorders
Fever, Grade 4
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Enterococcus Gallinarum, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Metabolism and nutrition disorders
Hypokalemia, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
5.6%
1/18 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Achromobacter, Grade 3
|
4.3%
1/23 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/26 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
|
Infections and infestations
Coagulase-negative staphylococcal, Grade 3
|
0.00%
0/23 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
0.00%
0/18 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
3.8%
1/26 • Number of events 1 • 30 days after last dose of 2FL, up to 60 days from first dose of 2FL
|
Additional Information
Pooja Khandelwal, MD
Cincinnati Children's Hospital Medical Center
Phone: (513) 803-4762
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place