Sickle Cell DiseasePhase 2NCT04474314
A Study of IMR-687 in Subjects With Sickle Cell Disease
A Phase 2 study testing IMR-687 in people with Sickle Cell Disease, run by Cardurion Pharmaceuticals, Inc. at 49 sites in 13 countries.
Trial summary
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease Read more about this study
- Who is it for?
- Ages 18 to 65 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
- 115 participants
- Where
- 49 sitesin 13 countries
- Study dates
- 13 Aug 2020to 4 May 2022
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of SCD (HbSS, HbSB0 thalassemia, or HbSB+ thalassemia)
- Hemoglobin of >5.5 and <10.5 g/dL; Hb values within 21 days post-transfusion will be excluded.
- Subjects must have had at least 2 and no more than 12 documented episodes of VOCs in the past 12 months at the time of informed consent signing and at randomization (Day 1).
- Subjects receiving HU must have received it continuously for at least 6 months prior to signing informed consent, and must have been on a stable dose for at least 3 months prior to signing the informed consent, with no anticipated need for dose adjustments during the study including the screening period, in the opinion of the investigator.
- Female subjects must not be pregnant or breastfeeding and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner.
- Must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff.
Who cannot join
- Hospital discharge for sickle cell crisis or other vaso-occlusive event within the 4 days prior to randomization (Day 1).
- Subjects participating in a chronic/prophylactic RBC transfusion program (i.e., regularly scheduled RBC transfusions); any transfusions within 21 days of screening or baseline Hb measurements
- Subjects with HbF >25% at screening.
- Significant kidney disease (eGFR <45mL/min) and liver dysfunction: alanine aminotransferase or aspartate aminotransferase >3x upper limit of normal.
- Body mass index (BMI) <17.0 kg/m2 and a total body weight <45 kg; or a BMI >35 kg/m2.
- Subjects with known active hepatitis A, hepatitis B, or hepatitis C, with active or acute event of malaria, or who are known to be positive for human immunodeficiency virus (HIV).
Show all 12Show fewer
- Stroke requiring medical intervention within 24 weeks prior to randomization (Day 1).
- Prior exposure to IMR-687.
- Subjects taking direct acting oral anti-coagulants (apixaban, dabigatran, rivaroxaban, edoxaban, or ticagrelor) or taking warfarin unless they stopped the treatment at least 28 days prior to randomization (Day 1).
- A history of use of crizanlizumab (Adakveo®) or voxelotor (Oxbryta®) within 6 months prior to signing the informed consent.
- Receipt of erythropoietin, luspatercept (Reblozyl®)or other hematopoietic growth factor treatment within 3 months of signing the ICF or anticipated need for such agents during the study.
- Prior gene therapy.
Trial locations and contacts
49 sites in 13 countries.
49 sites in 13 countries
United States14 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342
Johns Hopkins Hospital
Baltimore, Maryland, 21287
University of Alabama at Birmingham School of Medicine - 1917 Clinic
Birmingham, Alabama, 35233
The University of Illinois at Chicago College of Medicine
Chicago, Illinois, 60612-4333
Children's Hospital of Michigan
Detroit, Michigan, 48201
University of Connecticut Health Main Building
Farmington, Connecticut, 06030
University of California San Diego Moores Cancer Center
La Jolla, California, 92093
Arkansas Primary Care Clinic
Little Rock, Arkansas, 72204
Weill Cornell Medicine - Center for Blood Disorders
New York, New York, 10021
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
Virginia Commonwealth University Health - Ambulatory Care Center
Richmond, Virginia, 23219
Center For Inherited Blood Disorders
Santa Ana, California, 92705
Baylor Scott & White Medical Center-Temple
Temple, Texas, 76508
The Oncology Institute Long Beach
Whittier, California, 90603
United Kingdom6 sites
University Hospitals Bristol NHS Foundation Trust
Bristol, England, BS1 3NU
University College London Hospitals NHS
London, England, NW1 2PG
Guy's and Saint Thomas' NHS Foundation Trust
London, England, SE1 9RT
King's College Hospital NHS Foundation Trust
London, England, SE5 9RS
Manchester University NHS Foundation Trust
Manchester, England, M13 9WL
Oxford University Hospitals NHS Foundation Trust
Oxford, England, OX3 7LE
Italy5 sites
Ente Ospedaliero Ospedali Galliera
Genoa, 16128
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
Palermo, 90146
Fondazione Policlinico Universitario Agostino Gemelli
Roma, Rome, 00168
Azienda Ospedaliero - Universitaria San Luigi Gonzaga
Turin, Orbassano, 10043
Uganda5 sites
Jinja Regional Referral Hospital
Jinja, PO Box 43
Uganda Cancer Institute
Kampala, PO Box 3935
Makerere University College of Health Sciences
Kampala, PO Box 7072
Joint Clinical Research Center - Lubowa
Kampala, Wskiso District
Infectious Diseases Research Collaboration - Tororo
Tororo, 256
Tunisia4 sites
Hedi Chaker Hospital
Sfax, 3089
Centre Hôpital Universitaire Farhat Hached
Sousse, 4000
Centre National de Greffe de la Moelle Osseuse
Tunis, 1006
Hospital Aziza Othmana
Tunis, 1008
Greece3 sites
Laiko General Hospital of Athens
Athens, Attica, 11526
University General Hospital of Patras
Pátrai, 26504
Ippokrateio General Hospital of Thessaloniki
Thessaloniki, 54642
Kenya3 sites
Kenya Medical Research Institute - Kisumu
Kisumu, Nyanza, 40100
Gertrude's Children's Hospital
Nairobi, 00100
The Centre for Respiratory Diseases Research - Kenya Medical Research Institute
Nairobi, 00100
Lebanon3 sites
American University of Beirut Medical Center
Beirut, 01107 2020
Chronic Care Center
Hazmiyeh
Hopital Nini
Tripoli, North Governorate
Ghana2 sites
Korle Bu Teaching Hospital
Accra, PO Box 77
Kintampo Health Research Centre
Kintampo, Brong-Ahafo Region
Netherlands1 site
Hagaziekenhuis Van Den Haag - Leyweg
The Hague, South Holland, 2545 AA
Senegal1 site
Centre National De Transfusion Sanguine - Du Senegal
Dakar, 5002
About this study
Official title: A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects With Sickle Cell Disease
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
Read the detailed description
A phase 2b, randomized, double-blind, placebo-controlled, multicenter study of subjects with sickle cell disease (SCD; homozygous sickle hemoglobin [HbSS], sickle-β0 [HbSβ0] thalassemia, or sickle-β+ [HbSβ+] thalassemia) to evaluate the safety and efficacy of the phosphodiesterase type 9 (PDE9) inhibitor, IMR-687, administered once daily (qd) for 52 weeks.
- Conditions
- Sickle Cell Disease
- Being tested
- IMR-687, Placebo
- Keywords
- Sickle Cell Disease
- 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 once daily IMR-687
PlaceboDrug
Oral administration of once daily Placebo
| Study group | Role | What they receive |
|---|---|---|
| Higher dose IMR-687 | Experimental | Oral administration of once daily IMR-687 Receives: Drug: IMR-687 |
| Lower Dose IMR-687 | Experimental | Oral administration of once daily IMR-687 Receives: Drug: IMR-687 |
| Placebo | Placebo comparator | Oral administration of once daily Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect on the Incidence of Vaso-occlusive Crises (VOCs)
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.
Measured: Baseline to Week 52
Proportion of Patients With Adverse Events and Serious Adverse Events
Incidence of Adverse Events Incidence of Serious Adverse Events
Measured: Baseline to Week 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2020
- Study start13 Aug 2020
- Primary completion2 Mar 2022
- Study completion4 May 2022
Why it stopped: A recently conducted Interim analysis of IMR-SCD-301 demonstrated that while IMR-687 was generally well-tolerated, it failed to meet its primary efficacy endpoint. So, the sponsor has decided to discontinue this study.
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
- 115 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Imara, Inc.
- Responsible party
- Sponsor
- Investigators
- Kenneth Attie, MD, Study director, Imara, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04474314
- Sponsor's protocol ID
- IMR-SCD-301
- Other IDs
- 2019-004471-39, 2019
- Status last verified
- May 2025
- First submitted
- 26 Jun 2020
- Last updated
- 15 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
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 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT04474314
- Acronym / sponsor ID
- IMR-SCD-301
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: A recently conducted Interim analysis of IMR-SCD-301 demonstrated that while IMR-687 was generally well-tolerated, it failed to meet its primary efficacy endpoint. So, the sponsor has decided to discontinue this study.
- Enrolment
- 115 participants (actual)
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Imara, Inc.
- Study start
- 13 Aug 2020 (actual)
- Primary completion
- 2 Mar 2022 (actual)
- Study completion
- 4 May 2022 (actual)
- First posted
- 16 Jul 2020 (actual)
- Results
- Posted 18 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
Detailed description
A phase 2b, randomized, double-blind, placebo-controlled, multicenter study of subjects with sickle cell disease (SCD; homozygous sickle hemoglobin [HbSS], sickle-β0 [HbSβ0] thalassemia, or sickle-β+ [HbSβ+] thalassemia) to evaluate the safety and efficacy of the phosphodiesterase type 9 (PDE9) inhibitor, IMR-687, administered once daily (qd) for 52 weeks.
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 |
|---|---|---|
| Higher dose IMR-687 | Experimental | Drug: IMR-687Oral administration of once daily IMR-687 |
| Lower Dose IMR-687 | Experimental | Drug: IMR-687Oral administration of once daily IMR-687 |
| Placebo | Placebo comparator | Drug: PlaceboOral administration of once daily Placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Confirmed diagnosis of SCD (HbSS, HbSB0 thalassemia, or HbSB+ thalassemia)
- Hemoglobin of >5.5 and <10.5 g/dL; Hb values within 21 days post-transfusion will be excluded.
- Subjects must have had at least 2 and no more than 12 documented episodes of VOCs in the past 12 months at the time of informed consent signing and at randomization (Day 1).
- Subjects receiving HU must have received it continuously for at least 6 months prior to signing informed consent, and must have been on a stable dose for at least 3 months prior to signing the informed consent, with no anticipated need for dose adjustments during the study including the screening period, in the opinion of the investigator.
- Female subjects must not be pregnant or breastfeeding and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner.
Key exclusion criteria (12 listed)
- Hospital discharge for sickle cell crisis or other vaso-occlusive event within the 4 days prior to randomization (Day 1).
- Subjects participating in a chronic/prophylactic RBC transfusion program (i.e., regularly scheduled RBC transfusions); any transfusions within 21 days of screening or baseline Hb measurements
- Subjects with HbF >25% at screening.
- Significant kidney disease (eGFR <45mL/min) and liver dysfunction: alanine aminotransferase or aspartate aminotransferase >3x upper limit of normal.
- Body mass index (BMI) <17.0 kg/m2 and a total body weight <45 kg; or a BMI >35 kg/m2.
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 once daily IMR-687 | Drug | Higher dose IMR-687, Lower Dose IMR-687 |
| PlaceboOral administration of once daily Placebo | Drug | Placebo |
Primary outcome
Effect on the Incidence of Vaso-occlusive Crises (VOCs)
Time frame: Baseline to Week 52
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.
Proportion of Patients With Adverse Events and Serious Adverse Events
Time frame: Baseline to Week 56
Incidence of Adverse Events Incidence of Serious Adverse Events
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.
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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.
Kenneth Attie, MD study director
Imara, Inc.
49 sites in 13 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 49 sites in 13 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 6 inclusion and 12 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: stopped early, 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 49 sites in United States, United Kingdom, Italy, Uganda, Tunisia and 8 more countries. 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 18 Oct 2023). 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?
Effect on the Incidence of Vaso-occlusive Crises (VOCs) (time frame: Baseline to Week 52). The study lists 2 primary outcomes in all. 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.