Sickle Cell DiseasePhase 2NCT03401112
A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)
A Phase 2 study testing IMR-687 in people with Sickle Cell Disease, run by Cardurion Pharmaceuticals, Inc. at 13 sites in 2 countries.
Trial summary
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia). Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- IMR-687
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 13 sitesin 2 countries
- Study dates
- 26 Jan 2018to 28 Aug 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male or female participants with confirmed SCA
- Age 18 to 55 years, inclusive
- For participants on HU, must have been on a stable dose for at least 60 days prior to screening
Who cannot join
- Total hemoglobin >12.5 or <6 grams/deciliter
- Red blood cell transfusion within 60 days of baseline
- >7 hospitalizations for vaso-occlusive crises (VOCs) within the last year
- Estimated glomerular filtration rate <50 milliliter/minute
- Aspartate aminotransferase/alanine aminotransferase >3x the upper limit of normal
Trial locations and contacts
13 sites in 2 countries.
13 sites in 2 countries
United States7 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
University of Illinois
Chicago, Illinois, 60612
Loretto Hospital
Chicago, Illinois, 60644
University of Connecticut Health Center
Farmington, Connecticut, 06030
Foundation for Sickle Cell Disease Research
Hollywood, Florida, 33021
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Baylor Scott & White Health
Temple, Texas, 76508
United Kingdom6 sites
Sandwell & West Birmingham Hospital
Birmingham, B18 7QH
Bristol Haematology and Oncology Centre
Bristol, BS2 8ED
Royal London Hospital
London, E1 1BB
University College London Hospital
London, NW1 2PG
Guy's Hospital
London, SE1 9RT
Oxford Cancer & Haematology Centre, The Churchill Hospital
Oxford, OX3 7LE
About this study
Official title: A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).
Read the detailed description
This is a proof-of-concept study in adult SCA participants, ages 18 to 55 years old, to examine the safety, tolerability, and pharmacokinetic (PK), as well as the potential pharmacodynamic (PD) effects and clinical efficacy, of IMR-687 across a range of doses.
IMR-687 was administered in 2 populations of participants with SCA: those who were not receiving hydroxyurea (HU) and those who were receiving a stable dose of HU according to standard of care.
- Conditions
- Sickle Cell Disease
- Being tested
- IMR-687, Placebo
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IMR-687Drug
Oral administration of IMR-687 once daily with or without HU.
PlaceboDrug
Oral administration of placebo once daily with or without HU.
| Study group | Role | What they receive |
|---|---|---|
| IMR-687 50 mg/100 mg | Experimental | A starting dose of IMR-687 50 mg with dose escalation after 4 or 12 weeks, up to 100 mg was administered to participants. Duration of administration was 16 (Week 17) or 24 weeks (Week 25). Receives: Drug: IMR-687 |
| IMR-687 100 mg/200 mg | Experimental | A starting dose of IMR-687 100 mg with dose escalation after 4 or 12 weeks, up to 200 mg was administered to participants. Duration of administration was 24 weeks (Week 25). Receives: Drug: IMR-687 |
| Placebo | Placebo comparator | Matching placebo was administered for 16 (Week 17) or 24 weeks (Week 25). Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number Of Participants With Treatment-emergent Adverse Events (TEAEs) And Serious Adverse Events (SAEs)
An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. An SAE was defined as any AE that resulted in 1 or more of the following outcomes: death, required or prolonged hospitalization, life-threatening, persistent or significant disability/incapacity, congenital anomaly or birth defect, or other medically important event. A summary of serious and all other non-serious AEs, regardless of causality, is located in the Reported Adverse Events module.
Measured: Day 1 (after dosing) through up to Week 24
Secondary outcomes (6)
Pharmacokinetic (PK) Of Participants Who Did Not Concomitantly Receive HU: Maximum Plasma Concentration (Cmax) Of IMR-687
For PK assessments of participants who did not concomitantly receive HU, serial blood samples for IMR-687 plasma concentrations were drawn predose at 0.5, 1, 1.5, 2, 4, 6, and 8 hours after administration of study drug; and at 24 hours after administration of study drug. Day 1 (single-dose) and steady-state (Week 25) assessments are presented.
Measured: Day 1 and Week 25
PK Of Participants Who Did Not Concomitantly Receive HU: Area Under The Concentration-time Curve (AUC) From Time 0 To 24 Hours Postdose (AUC0-24h) Of IMR-687
For PK assessments of participants who did not concomitantly receive HU, serial blood samples for IMR-687 plasma concentrations were drawn predose at 0.5, 1, 1.5, 2, 4, 6, and 8 hours after administration of study drug; and at 24 hours after administration of study drug. Day 1 (single-dose) and steady-state (Week 25) assessments are presented.
Measured: Day 1 and Week 25
PK Of Participants Who Concomitantly Received HU: Cmax Of IMR-687
For PK assessments of participants who concomitantly received HU, serial blood samples for IMR-687 PK were drawn predose at 0.5, 1, 1.5, 2, 4, 6, and 8 hours after administration of study drug; and at 24 hours after administration of study drug. Day 1 (single-dose) and steady-state (Week 17) assessments are presented.
Measured: Day 1 and Week 17
PK Of Participants Who Concomitantly Received HU: AUC0-24h Of IMR-687
For PK assessments of participants who concomitantly received HU, serial blood samples for IMR-687 plasma concentrations were drawn predose at 0.5, 1, 1.5, 2, 4, 6, and 8 hours after administration of study drug; and at 24 hours after administration of study drug. Day 1 (single-dose) and steady-state (Week 17) assessments are presented.
Measured: Day 1 and Week 17
PK Of Participants Who Concomitantly Received HU: Cmax Of HU
For PK assessments of participants who concomitantly received HU, serial blood samples for HU PK were drawn predose and at 0.5, 1, 1.5, 3, 6, 8, and 10 hours after self-administration of the prescribed dose of HU. HU in the presence (end of treatment [EOT]: Week 17) or absence of IMR-687 (Baselines 1 and 2) are presented. HU concentration data were not sorted with respect to HU dose.
Measured: Baseline (1 and 2) and Week 17
PK Of Participants Who Concomitantly Received HU: AUC0-24h Of HU
For PK assessments of participants who concomitantly received HU, serial blood samples for HU PK were drawn predose and at 0.5, 1, 1.5, 3, 6, 8, and 10 hours after self-administration of the prescribed dose of HU. HU in the presence (EOT: Week 17) or absence of IMR-687 (Baselines 1 and 2) are presented. HU concentration data were not sorted with respect to HU dose.
Measured: Baseline (1 and 2) and Week 17
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2018
- Study start26 Jan 2018
- Primary completion28 Aug 2020
- Study completion28 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Imara, Inc.
- Responsible party
- Sponsor
- Investigators
- Regulatory Operations, Study director, Cardurion Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03401112
- Sponsor's protocol ID
- IMR-SCD-102
- Other IDs
- 2017-000653-39, 2017
- Status last verified
- May 2025
- First submitted
- 2 Jan 2018
- Last updated
- 15 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)
- ClinicalTrials.gov ID
- NCT03401112
- Acronym / sponsor ID
- IMR-SCD-102
- Conditions
- Sickle Cell Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Imara, Inc.
- Study start
- 26 Jan 2018 (actual)
- Primary completion
- 28 Aug 2020 (actual)
- Study completion
- 28 Aug 2020 (actual)
- First posted
- 17 Jan 2018 (actual)
- Results
- Posted 14 Apr 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).
Detailed description
This is a proof-of-concept study in adult SCA participants, ages 18 to 55 years old, to examine the safety, tolerability, and pharmacokinetic (PK), as well as the potential pharmacodynamic (PD) effects and clinical efficacy, of IMR-687 across a range of doses.
IMR-687 was administered in 2 populations of participants with SCA: those who were not receiving hydroxyurea (HU) and those who were receiving a stable dose of HU according to standard of care.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| IMR-687 50 mg/100 mg | Experimental | Drug: IMR-687A starting dose of IMR-687 50 mg with dose escalation after 4 or 12 weeks, up to 100 mg was administered to participants. Duration of administration was 16 (Week 17) or 24 weeks (Week 25). |
| IMR-687 100 mg/200 mg | Experimental | Drug: IMR-687A starting dose of IMR-687 100 mg with dose escalation after 4 or 12 weeks, up to 200 mg was administered to participants. Duration of administration was 24 weeks (Week 25). |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo was administered for 16 (Week 17) or 24 weeks (Week 25). |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Male or female participants with confirmed SCA
- Age 18 to 55 years, inclusive
- For participants on HU, must have been on a stable dose for at least 60 days prior to screening
Key exclusion criteria (5 listed)
- Total hemoglobin >12.5 or <6 grams/deciliter
- Red blood cell transfusion within 60 days of baseline
- >7 hospitalizations for vaso-occlusive crises (VOCs) within the last year
- Estimated glomerular filtration rate <50 milliliter/minute
- Aspartate aminotransferase/alanine aminotransferase >3x the upper limit of normal
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IMR-687Oral administration of IMR-687 once daily with or without HU. | Drug | IMR-687 100 mg/200 mg, IMR-687 50 mg/100 mg |
| PlaceboOral administration of placebo once daily with or without HU. | Drug | Placebo |
Primary outcome
Number Of Participants With Treatment-emergent Adverse Events (TEAEs) And Serious Adverse Events (SAEs)
Time frame: Day 1 (after dosing) through up to Week 24
An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. An SAE was defined as any AE that resulted in 1 or more of the following outcomes: death, required or prolonged hospitalization, life-threatening, persistent or significant disability/incapacity, congenital anomaly or birth defect, or other medically important event. A summary of serious and all other non-serious AEs, regardless of causality, is located in the Reported Adverse Events module.
6 secondary outcomes
Pharmacokinetic (PK) Of Participants Who Did Not Concomitantly Receive HU: Maximum Plasma Concentration (Cmax) Of IMR-687
Time frame: Day 1 and Week 25
PK Of Participants Who Did Not Concomitantly Receive HU: Area Under The Concentration-time Curve (AUC) From Time 0 To 24 Hours Postdose (AUC0-24h) Of IMR-687
Time frame: Day 1 and Week 25
PK Of Participants Who Concomitantly Received HU: Cmax Of IMR-687
Time frame: Day 1 and Week 17
PK Of Participants Who Concomitantly Received HU: AUC0-24h Of IMR-687
Time frame: Day 1 and Week 17
PK Of Participants Who Concomitantly Received HU: Cmax Of HU
Time frame: Baseline (1 and 2) and Week 17
PK Of Participants Who Concomitantly Received HU: AUC0-24h Of HU
Time frame: Baseline (1 and 2) and Week 17
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Regulatory Operations study director
Cardurion Pharmaceuticals
13 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IMR-687 in people with Sickle Cell Disease, run by Cardurion Pharmaceuticals, Inc. at 13 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Apr 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number Of Participants With Treatment-emergent Adverse Events (TEAEs) And Serious Adverse Events (SAEs) (time frame: Day 1 (after dosing) through up to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 May 2025.