Trial Outcomes & Findings for A Study of IMR-687 in Subjects With Sickle Cell Disease (NCT NCT04474314)
NCT ID: NCT04474314
Last Updated: 2025-05-15
Results Overview
Annualized rate of VOCs. For each subject, the total number of VOCs on treatment were divided by the time on treatment divided by 52 weeks. The median was then summarized.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
115 participants
Primary outcome timeframe
Baseline to Week 52
Results posted on
2025-05-15
Participant Flow
Participant milestones
| Measure |
Higher Dose IMR-687
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Lower Dose IMR-687
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Placebo
Oral administration of once daily Placebo
Placebo: Oral administration of once daily Placebo
|
|---|---|---|---|
|
Overall Study
STARTED
|
49
|
34
|
32
|
|
Overall Study
COMPLETED
|
2
|
6
|
8
|
|
Overall Study
NOT COMPLETED
|
47
|
28
|
24
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
1 participant in the high dose group was missing Baseline HbF
Baseline characteristics by cohort
| Measure |
Higher Dose IMR-687
n=49 Participants
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Lower Dose IMR-687
n=34 Participants
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Placebo
n=32 Participants
Oral administration of once daily Placebo
Placebo: Oral administration of once daily Placebo
|
Total
n=115 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
25.0 years
STANDARD_DEVIATION 7.91 • n=49 Participants
|
27.9 years
STANDARD_DEVIATION 8.44 • n=34 Participants
|
26.2 years
STANDARD_DEVIATION 9.27 • n=32 Participants
|
26.2 years
STANDARD_DEVIATION 8.47 • n=115 Participants
|
|
Sex: Female, Male
Female
|
36 Participants
n=49 Participants
|
14 Participants
n=34 Participants
|
14 Participants
n=32 Participants
|
64 Participants
n=115 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=49 Participants
|
20 Participants
n=34 Participants
|
18 Participants
n=32 Participants
|
51 Participants
n=115 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=49 Participants
|
0 Participants
n=34 Participants
|
0 Participants
n=32 Participants
|
0 Participants
n=115 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=49 Participants
|
0 Participants
n=34 Participants
|
0 Participants
n=32 Participants
|
0 Participants
n=115 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=49 Participants
|
0 Participants
n=34 Participants
|
0 Participants
n=32 Participants
|
0 Participants
n=115 Participants
|
|
Race (NIH/OMB)
Black or African American
|
34 Participants
n=49 Participants
|
15 Participants
n=34 Participants
|
20 Participants
n=32 Participants
|
69 Participants
n=115 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=49 Participants
|
10 Participants
n=34 Participants
|
10 Participants
n=32 Participants
|
26 Participants
n=115 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=49 Participants
|
0 Participants
n=34 Participants
|
0 Participants
n=32 Participants
|
0 Participants
n=115 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
9 Participants
n=49 Participants
|
9 Participants
n=34 Participants
|
2 Participants
n=32 Participants
|
20 Participants
n=115 Participants
|
|
Region of Enrollment
United Kingdom
|
3 Participants
n=49 Participants
|
1 Participants
n=34 Participants
|
1 Participants
n=32 Participants
|
5 Participants
n=115 Participants
|
|
Region of Enrollment
Ghana
|
11 Participants
n=49 Participants
|
2 Participants
n=34 Participants
|
8 Participants
n=32 Participants
|
21 Participants
n=115 Participants
|
|
Region of Enrollment
Kenya
|
5 Participants
n=49 Participants
|
4 Participants
n=34 Participants
|
4 Participants
n=32 Participants
|
13 Participants
n=115 Participants
|
|
Region of Enrollment
Oman
|
1 Participants
n=49 Participants
|
4 Participants
n=34 Participants
|
0 Participants
n=32 Participants
|
5 Participants
n=115 Participants
|
|
Region of Enrollment
Greece
|
0 Participants
n=49 Participants
|
2 Participants
n=34 Participants
|
1 Participants
n=32 Participants
|
3 Participants
n=115 Participants
|
|
Region of Enrollment
Lebanon
|
0 Participants
n=49 Participants
|
4 Participants
n=34 Participants
|
3 Participants
n=32 Participants
|
7 Participants
n=115 Participants
|
|
Region of Enrollment
Morocco
|
5 Participants
n=49 Participants
|
2 Participants
n=34 Participants
|
3 Participants
n=32 Participants
|
10 Participants
n=115 Participants
|
|
Region of Enrollment
Senegal
|
4 Participants
n=49 Participants
|
2 Participants
n=34 Participants
|
1 Participants
n=32 Participants
|
7 Participants
n=115 Participants
|
|
Region of Enrollment
Uganda
|
16 Participants
n=49 Participants
|
8 Participants
n=34 Participants
|
5 Participants
n=32 Participants
|
29 Participants
n=115 Participants
|
|
Region of Enrollment
Tunisia
|
1 Participants
n=49 Participants
|
3 Participants
n=34 Participants
|
4 Participants
n=32 Participants
|
8 Participants
n=115 Participants
|
|
Region of Enrollment
United States
|
3 Participants
n=49 Participants
|
2 Participants
n=34 Participants
|
2 Participants
n=32 Participants
|
7 Participants
n=115 Participants
|
|
BMI
|
21.04 kg/m^2
STANDARD_DEVIATION 3.571 • n=49 Participants
|
21.03 kg/m^2
STANDARD_DEVIATION 3.451 • n=34 Participants
|
21.10 kg/m^2
STANDARD_DEVIATION 2.794 • n=32 Participants
|
21.06 kg/m^2
STANDARD_DEVIATION 3.308 • n=115 Participants
|
|
HbF
|
11.51 % of HbF
STANDARD_DEVIATION 7.775 • n=48 Participants • 1 participant in the high dose group was missing Baseline HbF
|
10.16 % of HbF
STANDARD_DEVIATION 5.453 • n=34 Participants • 1 participant in the high dose group was missing Baseline HbF
|
11.16 % of HbF
STANDARD_DEVIATION 4.903 • n=32 Participants • 1 participant in the high dose group was missing Baseline HbF
|
11.01 % of HbF
STANDARD_DEVIATION 6.384 • n=114 Participants • 1 participant in the high dose group was missing Baseline HbF
|
PRIMARY outcome
Timeframe: Baseline to Week 52Population: ITT Analysis set
Annualized rate of VOCs. For each subject, the total number of VOCs on treatment were divided by the time on treatment divided by 52 weeks. The median was then summarized.
Outcome measures
| Measure |
Higher Dose IMR-687
n=47 Participants
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Lower Dose IMR-687
n=33 Participants
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Placebo
n=32 Participants
Oral administration of once daily Placebo
Placebo: Oral administration of once daily Placebo
|
|---|---|---|---|
|
Effect on the Incidence of Vaso-occlusive Crises (VOCs)
|
2.32 VOCs/weeks/52
Interval 1.02 to 5.51
|
1.20 VOCs/weeks/52
Interval 0.0 to 3.9
|
2.48 VOCs/weeks/52
Interval 0.51 to 5.03
|
PRIMARY outcome
Timeframe: Baseline to Week 56Population: Safety Analysis Set
Incidence of Adverse Events Incidence of Serious Adverse Events
Outcome measures
| Measure |
Higher Dose IMR-687
n=47 Participants
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Lower Dose IMR-687
n=33 Participants
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Placebo
n=32 Participants
Oral administration of once daily Placebo
Placebo: Oral administration of once daily Placebo
|
|---|---|---|---|
|
Proportion of Patients With Adverse Events and Serious Adverse Events
Number of participants with Treatment-Emergent Adverse Events
|
40 Participants
|
29 Participants
|
28 Participants
|
|
Proportion of Patients With Adverse Events and Serious Adverse Events
Number of participants with Treatment Emergent Serious Adverse Events
|
17 Participants
|
12 Participants
|
12 Participants
|
Adverse Events
Higher Dose IMR-687
Serious events: 17 serious events
Other events: 38 other events
Deaths: 0 deaths
Lower Dose IMR-687
Serious events: 12 serious events
Other events: 29 other events
Deaths: 0 deaths
Placebo
Serious events: 12 serious events
Other events: 24 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Higher Dose IMR-687
n=47 participants at risk
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Lower Dose IMR-687
n=33 participants at risk
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Placebo
n=32 participants at risk
Oral administration of once daily Placebo
Placebo: Oral administration of once daily Placebo
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Sickle Cell Anemia with Crisis
|
25.5%
12/47 • 56 Weeks
|
27.3%
9/33 • 56 Weeks
|
31.2%
10/32 • 56 Weeks
|
|
Blood and lymphatic system disorders
Anaemia
|
6.4%
3/47 • 56 Weeks
|
12.1%
4/33 • 56 Weeks
|
0.00%
0/32 • 56 Weeks
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/47 • 56 Weeks
|
3.0%
1/33 • 56 Weeks
|
0.00%
0/32 • 56 Weeks
|
|
Infections and infestations
Sepsis
|
4.3%
2/47 • 56 Weeks
|
3.0%
1/33 • 56 Weeks
|
6.2%
2/32 • 56 Weeks
|
|
Infections and infestations
Malaria
|
2.1%
1/47 • 56 Weeks
|
3.0%
1/33 • 56 Weeks
|
9.4%
3/32 • 56 Weeks
|
Other adverse events
| Measure |
Higher Dose IMR-687
n=47 participants at risk
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Lower Dose IMR-687
n=33 participants at risk
Oral administration of once daily IMR-687
IMR-687: Oral administration of once daily IMR-687
|
Placebo
n=32 participants at risk
Oral administration of once daily Placebo
Placebo: Oral administration of once daily Placebo
|
|---|---|---|---|
|
Nervous system disorders
Headache
|
25.5%
12/47 • 56 Weeks
|
27.3%
9/33 • 56 Weeks
|
21.9%
7/32 • 56 Weeks
|
|
Blood and lymphatic system disorders
Sickle Cell Anaemia with Crisis
|
25.5%
12/47 • 56 Weeks
|
27.3%
9/33 • 56 Weeks
|
31.2%
10/32 • 56 Weeks
|
|
Gastrointestinal disorders
Nausea
|
23.4%
11/47 • 56 Weeks
|
21.2%
7/33 • 56 Weeks
|
3.1%
1/32 • 56 Weeks
|
|
Nervous system disorders
Dizziness
|
21.3%
10/47 • 56 Weeks
|
9.1%
3/33 • 56 Weeks
|
6.2%
2/32 • 56 Weeks
|
|
Gastrointestinal disorders
Vomiting
|
19.1%
9/47 • 56 Weeks
|
18.2%
6/33 • 56 Weeks
|
0.00%
0/32 • 56 Weeks
|
|
Infections and infestations
Malaria
|
12.8%
6/47 • 56 Weeks
|
6.1%
2/33 • 56 Weeks
|
12.5%
4/32 • 56 Weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place