Trial Outcomes & Findings for Safety and Efficacy Study of Oral Ferric Iron To Treat Iron Deficiency Anaemia in Quiescent Crohn's Disease (AEGIS-2) (NCT NCT01352221)

Results Overview

Primary efficacy endpoint, defined as the change in Hb concentration from Baseline to Week 12. Baseline was defined as the pre-dose Hb concentration measured at the Randomisation Visit (Week 0). Missing Randomisation Hb values were replaced by Screening Hb values, if the randomisation was within the protocol-specified window. Hb concentration (g/dL) was analysed by a central laboratory from blood samples collected at every clinic visit: Screening, Randomisation (Week 0), Weeks 4, 8, 12, 14, 16, 20, 24, 36, 48, 64, Weeks 14 to 64 were open-label. The baseline, absolute concentration and change from baseline in Hb at all post-randomisation visits were listed and summarised by week using descriptive statistics. An analysis of covariance (ANCOVA) was used to analyse the primary endpoint; this included treatment, gender and disease as factors and baseline Hb as a covariate.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

128 participants

Primary outcome timeframe

Baseline to Week 12 - double-blind phase

Results posted on

2020-10-30

Participant Flow

Potential subjects were selected from the general population attending each centre in UK, DE, AT or HU for routine care of their IBD and anaemia. Individuals interested in participating were invited for the Screening visit in order to assess eligibility. Written informed consent was obtained prior to conducting any study specific assessments

Subjects were male or female ≥18y with a confirmed diagnosis of Crohn's Disease (CD) in remission or mild-to-moderate CD (defined as CD Activity Index \[CDAI\] score \<220 at randomization); mild-to-moderate IDA (Hb concentration ≥9.5 g/dL and \<12.0 g/dL for females; ≥9.5 g/dL and \<13.0 g/dL for males; serum ferritin levels \<30 μg/L at Screening.

Participant milestones

Participant milestones
Measure	ST10 30mg ST10 capsules BD - double-blind phase	Placebo Matching placebo for ST10 capsules - double-blind phase	ST10 - Open-label Continuation From Active Arm in Double-blind Open-label extension of ST10 active treatment arm from double-blind phase	Placebo Switch to Open-label Extension ST10 Treatment Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Double-blind Phase STARTED	64	64	0	0
Double-blind Phase COMPLETED	55	53	0	0
Double-blind Phase NOT COMPLETED	9	11	0	0
Open-label Phase STARTED	0	0	50	47
Open-label Phase COMPLETED	0	0	37	36
Open-label Phase NOT COMPLETED	0	0	13	11

Reasons for withdrawal

Reasons for withdrawal
Measure	ST10 30mg ST10 capsules BD - double-blind phase	Placebo Matching placebo for ST10 capsules - double-blind phase	ST10 - Open-label Continuation From Active Arm in Double-blind Open-label extension of ST10 active treatment arm from double-blind phase	Placebo Switch to Open-label Extension ST10 Treatment Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Double-blind Phase Adverse Event	6	4	0	0
Double-blind Phase Withdrawal by Subject	3	5	0	0
Double-blind Phase Lost to Follow-up	0	1	0	0
Double-blind Phase Protocol Violation	0	1	0	0
Open-label Phase Physician Decision	0	0	2	1
Open-label Phase Pregnancy	0	0	1	0
Open-label Phase Adverse Event	0	0	8	4
Open-label Phase Withdrawal by Subject	0	0	1	6
Open-label Phase Worsening of IBD	0	0	1	0

Baseline Characteristics

Safety and Efficacy Study of Oral Ferric Iron To Treat Iron Deficiency Anaemia in Quiescent Crohn's Disease (AEGIS-2)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ST10 n=64 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=64 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Total n=128 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	62 Participants n=5 Participants	63 Participants n=7 Participants	125 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Age, Continuous	40.1 years STANDARD_DEVIATION 13.52 • n=5 Participants	38.5 years STANDARD_DEVIATION 12.3 • n=7 Participants	39.2 years STANDARD_DEVIATION 12.9 • n=5 Participants
Sex: Female, Male Female	40 Participants n=5 Participants	43 Participants n=7 Participants	83 Participants n=5 Participants
Sex: Female, Male Male	24 Participants n=5 Participants	21 Participants n=7 Participants	45 Participants n=5 Participants
Region of Enrollment United Kingdom	15 participants n=5 Participants	10 participants n=7 Participants	25 participants n=5 Participants
Region of Enrollment Hungary	11 participants n=5 Participants	15 participants n=7 Participants	26 participants n=5 Participants
Region of Enrollment Germany	35 participants n=5 Participants	32 participants n=7 Participants	67 participants n=5 Participants
Region of Enrollment Austria	4 participants n=5 Participants	6 participants n=7 Participants	10 participants n=5 Participants
Duration of Crohn's disease (years)	11.25 years STANDARD_DEVIATION 9.296 • n=5 Participants	11.01 years STANDARD_DEVIATION 8.09 • n=7 Participants	11.13 years STANDARD_DEVIATION 8.6 • n=5 Participants
Time since last IBD flare-up (months)	24.8 months STANDARD_DEVIATION 59.16 • n=5 Participants	21.51 months STANDARD_DEVIATION 38.46 • n=7 Participants	23.2 months STANDARD_DEVIATION 48.4 • n=5 Participants
Time since last OFP dose (months)	36.17 months STANDARD_DEVIATION 40.1 • n=5 Participants	33.34 months STANDARD_DEVIATION 44.04 • n=7 Participants	34.76 months STANDARD_DEVIATION 41.7 • n=5 Participants
Haemoglobin concentration at baseline	11 g/dL STANDARD_DEVIATION 1.03 • n=5 Participants	11.1 g/dL STANDARD_DEVIATION 0.85 • n=7 Participants	11.05 g/dL STANDARD_DEVIATION 0.93 • n=5 Participants
Serum Ferritin concentration at baseline	8.6 μg/L STANDARD_DEVIATION 6.8 • n=5 Participants	8.2 μg/L STANDARD_DEVIATION 6.5 • n=7 Participants	8.4 μg/L STANDARD_DEVIATION 6.6 • n=5 Participants
TSAT% at baseline	10.6 percentage STANDARD_DEVIATION 11.7 • n=5 Participants	9.5 percentage STANDARD_DEVIATION 7.5 • n=7 Participants	10.05 percentage STANDARD_DEVIATION 9.6 • n=5 Participants
Crohn's Disease Activity Index (CDAI) score at baseline	86.4 score on a scale n=5 Participants	105.3 score on a scale n=7 Participants	95.85 score on a scale n=5 Participants
Irritable Bowel Disease Questionnaire (IBDQ) score at baseline	175.0 score on a scale STANDARD_DEVIATION 30.92 • n=5 Participants	171.0 score on a scale STANDARD_DEVIATION 33.56 • n=7 Participants	173.02 score on a scale STANDARD_DEVIATION 32.23 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Week 12 - double-blind phase

Population: Full Analysis Set (FAS)

Primary efficacy endpoint, defined as the change in Hb concentration from Baseline to Week 12. Baseline was defined as the pre-dose Hb concentration measured at the Randomisation Visit (Week 0). Missing Randomisation Hb values were replaced by Screening Hb values, if the randomisation was within the protocol-specified window. Hb concentration (g/dL) was analysed by a central laboratory from blood samples collected at every clinic visit: Screening, Randomisation (Week 0), Weeks 4, 8, 12, 14, 16, 20, 24, 36, 48, 64, Weeks 14 to 64 were open-label. The baseline, absolute concentration and change from baseline in Hb at all post-randomisation visits were listed and summarised by week using descriptive statistics. An analysis of covariance (ANCOVA) was used to analyse the primary endpoint; this included treatment, gender and disease as factors and baseline Hb as a covariate.

Outcome measures

Outcome measures
Measure	ST10 n=58 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=53 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin (Hb) Concentration From Baseline to Week 12 (Full Analysis Set, FAS)	2.26 g/dL Standard Deviation 1.184	0.01 g/dL Standard Deviation 0.764

SECONDARY outcome

Timeframe: Subjects that achieved ≥1 g/dL change from baseline in Hb concentration at Week 12 - double-blind phase

Population: FAS

Logistic regression analysis of proportion of subjects that achieved ≥1 g/dL change from baseline in Hb concentration at Week 12 in the double-blind phase

Outcome measures

Outcome measures
Measure	ST10 n=64 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=64 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Proportion of Subjects That Achieved ≥1 g/dL Change From Baseline in Hb Concentration at Week 12 (Full Analysis Set, FAS) Yes	50 Participants	7 Participants
Proportion of Subjects That Achieved ≥1 g/dL Change From Baseline in Hb Concentration at Week 12 (Full Analysis Set, FAS) No	14 Participants	57 Participants

SECONDARY outcome

Timeframe: Baseline to Week 12 - double-blind phase

Population: FAS

Logistic regression analysis of proportion of subjects that achieved ≥2 g/dL change from baseline in Hb concentration at Week 12 in the double-blind phase

Outcome measures

Outcome measures
Measure	ST10 n=64 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=64 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Proportion of Subjects That Achieved ≥2 g/dL Change From Baseline in Hb Concentration at Week 12 (Full Analysis Set, FAS) Yes	36 Participants	0 Participants
Proportion of Subjects That Achieved ≥2 g/dL Change From Baseline in Hb Concentration at Week 12 (Full Analysis Set, FAS) No	28 Participants	64 Participants

SECONDARY outcome

Timeframe: Baseline to Week 12 - double-blind phase

Population: FAS

Logistic regression analysis of proportion of subjects that achieved Hb concentration within normal range at Week 12 end of double-blind phase

Outcome measures

Outcome measures
Measure	ST10 n=64 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=64 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Proportion of Subjects That Achieved Hb Concentration Within Normal Range at Week 12 (Full Analysis Set, FAS) Yes	42 Participants	8 Participants
Proportion of Subjects That Achieved Hb Concentration Within Normal Range at Week 12 (Full Analysis Set, FAS) No	22 Participants	56 Participants

SECONDARY outcome

Timeframe: Baseline to Week 4 - double-blind phase

ANCOVA analysis of the change in Hb concentration from Baseline to Week 4 of the double-blind phase - Full Analysis Set, multiple imputation

Outcome measures

Outcome measures
Measure	ST10 n=59 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=61 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Hb Concentration From Baseline to Week 4 (Full Analysis Set, FAS)	1.08 g/dL Standard Deviation 0.676	0 g/dL Standard Deviation 0.67

SECONDARY outcome

Timeframe: Baseline to Week 8 - double-blind phase

Population: FAS

ANCOVA analysis of Change in Hb concentration from Baseline to Week 8 of double-blind phase - FAS, multiple imputation

Outcome measures

Outcome measures
Measure	ST10 n=59 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=56 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Hb Concentration From Baseline to Week 8 (Full Analysis Set, FAS)	1.79 g/dL Standard Deviation 1.037	0.04 g/dL Standard Deviation 0.722

SECONDARY outcome

Timeframe: Baseline to Week 16 - open-label phase

Population: FAS

Change in Haemoglobin Concentration from Baseline to Week 16 (FAS), after 12-week double-blind phase and first 4 weeks of open-label ST10 treatment.

Outcome measures

Outcome measures
Measure	ST10 n=46 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=45 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 16 (Full Analysis Set, FAS)	2.34 g/dL Standard Deviation 1.281	1.04 g/dL Standard Deviation 1.023

SECONDARY outcome

Timeframe: Baseline to Week 20 - open-label phase

Population: FAS

Change in Haemoglobin Concentration from Baseline to Week 20 (FAS), after 12-week double-blind phase and then 8 weeks of open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=43 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=43 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 20 (Full Analysis Set, FAS)	2.45 g/dL Standard Deviation 1.213	1.46 g/dL Standard Deviation 1.056

SECONDARY outcome

Timeframe: Baseline to Week 24 - open-label phase

Population: FAS

Change in Haemoglobin Concentration from Baseline to Week 24 (FAS), after 12-week double-blind phase and then 12 weeks of open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=43 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=41 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 24 (Full Analysis Set, FAS)	2.68 g/dL Standard Deviation 1.127	1.87 g/dL Standard Deviation 1.195

SECONDARY outcome

Timeframe: Baseline to Week 36 - open-label phase

Population: FAS

Change in Haemoglobin Concentration from Baseline to Week 36 (FAS), after 12-week double-blind phase and then 24 weeks of open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=41 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=36 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 36 (Full Analysis Set, FAS)	2.85 g/dL Standard Deviation 1.227	2.17 g/dL Standard Deviation 1.048

SECONDARY outcome

Timeframe: Baseline to Week 48 - open-label phase

Population: FAS

Change in Haemoglobin Concentration from Baseline to Week 48 (FAS), after 12-week double-blind phase and then 36 weeks of open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=40 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=37 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 48 (Full Analysis Set, FAS)	3.09 g/dL Standard Deviation 1.339	2.0 g/dL Standard Deviation 1.191

SECONDARY outcome

Timeframe: Baseline to Week 64 - open-label phase

Population: FAS

Change in Haemoglobin Concentration from Baseline to Week 64 (FAS), after 12-week double-blind phase and then 52 weeks of open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=35 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=36 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 64 (Full Analysis Set, FAS)	3.07 g/dL Standard Deviation 1.457	2.19 g/dL Standard Deviation 1.605

SECONDARY outcome

Timeframe: Baseline to Week 64 EOS - open-label phase

Population: FAS

Change in Haemoglobin Concentration from Baseline to Week 64 EOS (FAS) - Week 64 was re-categorised as Week 64 EOS for those subjects who withdrew from the study early and the 'Week 64' visit was outside the visit window of 64 weeks ± 2 days

Outcome measures

Outcome measures
Measure	ST10 n=10 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=17 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 64 EOS (Full Analysis Set, FAS)	1.32 g/dL Standard Deviation 1.713	0.52 g/dL Standard Deviation 1.417

SECONDARY outcome

Timeframe: Baseline to Week 16 - open-label phase

Population: FAS

Proportion of subjects that achieved Haemoglobin Concentration within normal range at Week 16 (Full Analysis Set), after 12-week double-blind phase and first 4 weeks of open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=46 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=45 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 16 (Full Analysis Set, FAS) Yes	36 Participants	17 Participants
Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 16 (Full Analysis Set, FAS) No	10 Participants	28 Participants

SECONDARY outcome

Timeframe: Baseline to Week 36 - open-label phase

Population: FAS

Proportion of subjects that achieved Haemoglobin Concentration within normal range at Week 36 (Full Analysis Set), after 12-week double-blind phase and 24 weeks of open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=41 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=36 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 36 (Full Analysis Set, FAS) Yes	35 Participants	29 Participants
Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 36 (Full Analysis Set, FAS) No	6 Participants	7 Participants

SECONDARY outcome

Timeframe: Baseline to Week 64 - open-label phase

Population: FAS

Proportion of subjects that achieved Haemoglobin Concentration within normal range at Week 64 (Full Analysis Set), after 12-week double-blind phase and 52 weeks of open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=36 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=36 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 64 (Full Analysis Set, FAS) Yes	31 Participants	30 Participants
Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 64 (Full Analysis Set, FAS) No	5 Participants	6 Participants

SECONDARY outcome

Timeframe: Baseline to Week 12 - double-blind phase

Population: Full Analysis Set (FAS)

ANCOVA sensitivity analysis of the Primary efficacy endpoint analysis on the PPAS - Change in Haemoglobin Concentration from Baseline to Week 12

Outcome measures

Outcome measures
Measure	ST10 n=55 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=57 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 12 (Per Protocol Analysis Set, PPAS)	2.23 g/dL Standard Deviation 0.13	0.05 g/dL Standard Deviation 0.13

SECONDARY outcome

Timeframe: Baseline to Week 12 - double-blind phase

Population: Full Analysis Set (FAS)

ANCOVA sensitivity analysis of the Primary efficacy endpoint analysis on the FAS LOCF - Change in Haemoglobin Concentration from Baseline to Week 12

Outcome measures

Outcome measures
Measure	ST10 n=64 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=64 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Haemoglobin Concentration From Baseline to Week 12 (Full Analysis Set [FAS] LOCF)	2.11 g/dL Standard Deviation 0.12	-0.03 g/dL Standard Deviation 0.12

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to Week 12 - double-blind phase

Population: Full Analysis Set (FAS)

Change in serum Ferritin concentration from Baseline to Week 12 (Full Analysis Set), after 12-week double-blind phase

Outcome measures

Outcome measures
Measure	ST10 n=59 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=53 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Serum Ferritin Concentration From Baseline to Week 12 (Full Analysis Set, FAS)	17.3 μg/dL Standard Deviation 28.3	1.2 μg/dL Standard Deviation 7.85

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to Week 12 - double-blind phase

Population: Full Analysis Set (FAS)

Change in serum TSAT% from Baseline to Week 12 (FAS), after 12-week double-blind phase

Outcome measures

Outcome measures
Measure	ST10 n=59 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=52 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Serum TSAT% From Baseline to Week 12 (Full Analysis Set, FAS)	18.0 percent Standard Deviation 20.17	-0.4 percent Standard Deviation 7.82

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to Week 64 - open-label phase

Population: FAS

Change in serum Ferritin concentration from Baseline to Week 64 (FAS), after 12-week double-blind phase and 52 weeks open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=36 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=36 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Serum Ferritin Concentration From Baseline to Week 64 (Full Analysis Set, FAS)	60.4 μg/dL Standard Deviation 93.35	36.6 μg/dL Standard Deviation 46.8

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to Week 64 - open-label phase

Population: FAS

Change in serum TSAT% from Baseline to Week 64 (Full Analysis Set), after 12-week double-blind phase and 52 weeks open-label ST10 treatment

Outcome measures

Outcome measures
Measure	ST10 n=36 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=36 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change in Serum TSAT% From Baseline to Week 64 (Full Analysis Set, FAS)	18.8 percent Standard Deviation 12.46	17.7 percent Standard Deviation 16.2

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 12 - double-blind phase

Population: Full Analysis Set (FAS)

Irritable Bowel Disease Questionnaire (IBDQ) score at Week 12 (FAS), end of double-blind phase. The IBDQ was developed as an activity index for determining the effect of Crohn's disease symptoms on perceived quality of life. It is a 32-item questionnaire with four dimensions: bowel function, emotional status, systemic symptoms and social function. Total IBDQ score ranges from 32 to 224, with higher scores indicating better quality of life. The score of patients in remission usually is between 170 and 190.

Outcome measures

Outcome measures
Measure	ST10 n=60 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=60 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Irritable Bowel Disease Questionnaire (IBDQ) Score at Week 12 (Full Analysis Set, FAS)	178.3 score on a scale Standard Deviation 32.36	176.3 score on a scale Standard Deviation 31.5

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 64 - open-label phase

Population: FAS

Irritable Bowel Disease Questionnaire (IBDQ) score at Week 64 (FAS), after 12-week double-blind phase and 52 weeks of open-label ST10 treatment. The IBDQ was developed as an activity index for determining the effect of Crohn's disease symptoms on perceived quality of life. It is a 32-item questionnaire with four dimensions: bowel function, emotional status, systemic symptoms and social function. Total IBDQ score ranges from 32 to 224, with higher scores indicating better quality of life. The score of patients in remission usually is between 170 and 190.

Outcome measures

Outcome measures
Measure	ST10 n=37 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=36 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Irritable Bowel Disease Questionnaire (IBDQ) Score at Week 64 (Full Analysis Set, FAS)	180.7 score on a scale Standard Deviation 30.14	177.2 score on a scale Standard Deviation 36.97

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to Week 12 - double-blind phase

Population: Full Analysis Set (FAS)

Change from baseline (randomisation) in Crohn's Disease Activity Index (CDAI) score at Week 12 (FAS), end of double-blind phase (in subjects with CD). The CDAI is a research tool used to quantify the symptoms of patients with Crohn's disease. CDAI score can range from 0 to approximately 600. Traditionally, for clinical trials, clinical remission is defined as a CDAI score \<150, clinical response is a decrease in CDAI score of 70-100. Mildly active Crohn's disease is defined as a CDAI score 150-220, moderate-severe Crohn's is typically a CDAI 220-450, and severe disease is defined as a CDAI \>450.

Outcome measures

Outcome measures
Measure	ST10 n=31 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=32 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change From Baseline in Crohn's Disease Activity Index (CDAI) Score at Week 12 (Full Analysis Set, FAS)	-24 score on a scale Interval -151.0 to 49.0	12.5 score on a scale Interval -90.0 to 124.0

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to Week 64 - open-label phase

Population: FAS

Change from baseline in Crohn's Disease Activity Index (CDAI) score at Week 64 (FAS), after 12-week double blind phase and 52 weeks open-label ST10 treatment (in participants with CD only). The CDAI is a research tool used to quantify the symptoms of patients with Crohn's disease. CDAI score can range from 0 to approximately 600. Traditionally, for clinical trials, clinical remission is defined as a CDAI score \<150, clinical response is a decrease in CDAI score of 70-100. Mildly active Crohn's disease is defined as a CDAI score 150-220, moderate-severe Crohn's is typically a CDAI 220-450, and severe disease is defined as a CDAI \>450.

Outcome measures

Outcome measures
Measure	ST10 n=17 Participants ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo n=17 Participants Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Change From Baseline in Crohn's Disease Activity Index (CDAI) Score at Week 64 (Full Analysis Set, FAS)	-16.6 score on a scale Interval -112.0 to 54.0	-1.0 score on a scale Interval -99.0 to 58.0

Adverse Events

ST10 - Safety Set, Double-blind Phase

Serious events: 1 serious events

Other events: 39 other events

Deaths: 0 deaths

Placebo - Safety Set, Double-blind Phase

Serious events: 2 serious events

Other events: 46 other events

Deaths: 0 deaths

ST10 Continuation - Safety Set, Open-label Phase

Serious events: 8 serious events

Other events: 40 other events

Deaths: 0 deaths

Placebo Switch to ST10 Treatment-Safety Set, Open-label Phase

Serious events: 2 serious events

Other events: 35 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	ST10 - Safety Set, Double-blind Phase n=64 participants at risk Adverse events reported in the double-blind phase active treatment arm with ST10 (Ferric Maltol). ST10 30 mg capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.	Placebo - Safety Set, Double-blind Phase n=64 participants at risk Adverse events reported in the double-blind phase placebo treatment arm. Matching placebo capsules for ST10 taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase	ST10 Continuation - Safety Set, Open-label Phase n=50 participants at risk Adverse events reported in the open-label extension phase for continuation of active treatment with ST10 (Ferric Maltol) from the double-blind phase active treatment arm	Placebo Switch to ST10 Treatment-Safety Set, Open-label Phase n=47 participants at risk Adverse events reported in the open-label extension phase from those subjects continuing treatment from the double-blind placebo arm; subjects commenced ST10 open-label treatment after completion of the double-blind phase at the Week 12 visit.
Gastrointestinal disorders Abdominal Pain	1.6% 1/64 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	4.0% 2/50 • Number of events 2 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Gastrointestinal disorders Diarrhoea	1.6% 1/64 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/50 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Gastrointestinal disorders Crohn's disease	1.6% 1/64 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	1.6% 1/64 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/50 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Gastrointestinal disorders Rectal abscess	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	1.6% 1/64 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/50 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Gastrointestinal disorders Colitis ulcerative	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.0% 1/50 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Surgical and medical procedures Cholesteatoma removal	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.0% 1/50 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Cardiac disorders Angina pectoris	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/50 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.1% 1/47 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/50 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.1% 1/47 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
General disorders Hernia	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.0% 1/50 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Gastrointestinal disorders Rectal haemorrhage	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.0% 1/50 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Infections and infestations Herpes zoster	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.0% 1/50 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Infections and infestations Peritonitis	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.0% 1/50 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/47 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.
Infections and infestations Pneumonia	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/64 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	0.00% 0/50 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.	2.1% 1/47 • Number of events 1 • Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination). Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject's on-going condition or intercurrent illness.

Other adverse events

Additional Information

Jackie Mitchell MA DPhil

Shield Therapeutics

Phone: +44 (0) 191 511 8515

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place