β ThalassemiaPhase 2NCT04411082
A Study of IMR-687 in Subjects With Beta Thalassemia
A Phase 2 study testing IMR-687 in people with β Thalassemia, run by Cardurion Pharmaceuticals, Inc. at 36 sites in 13 countries.
Trial summary
A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- β Thalassemia
- 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
- 122 participants
- Where
- 36 sitesin 13 countries
- Study dates
- 16 Oct 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
- Documented diagnosis of β-thalassemia or HbE/ β-thalassemia in their medical history. Concomitant alpha gene deletion, duplication, or triplication is allowed.
- Documentation of the dates of transfusion events and the number of all pRBC units per event within the 12 weeks prior to the Baseline (Day 1) visit. .
- Must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff.
- TDT Subjects: subjects must be regularly transfused, defined as >3 to 10 pRBC units in the12 weeks prior to Baseline (Day 1) visit and no transfusion-free period for >35 days during that period.
- NTDT subjects: Subjects must be transfusion independent, defined as 0 to ≤3 units of pRBCs received during the 12-week period prior to the Baseline (Day 1) visit, must not be on a regular transfusion program, must be RBC transfusion-free for at least ≥ 4 weeks prior to randomization, and must not be scheduled to start a regular
- hematopoietic stem cell transplantation within 9 months.
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- NTDT subjects: Subjects must have Hb ≤10.0 g/dL at Screening; the screening Hb sample must be collected 7 to 28 days prior to randomization. Hb values within 21 days post-transfusion will be excluded.
- ECOG performance score of 0 to 1
- Female subjects must not be pregnant, or breastfeeding and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner.
Who cannot join
- Diagnosis of α-thalassemia (e.g., hemoglobin H [HbH]) or hemoglobin S (HbS)/ β thalassemia.
- Body mass index (BMI) <17.0 kg/m2 or a total body weight <45 kg; or 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).
- Stroke requiring medical intervention ≤24 weeks prior to randomization.
- Platelet count >1000 × 109/L.
- Participated in another clinical study of an investigational agent (or device) within 30 days or 5-half-lives of date of informed consent, whichever is longer, or is currently participating in another study.
Show all 9Show fewer
- For Subjects on iron chelation therapy (ICT) at the time of ICF signing, initiation of ICT less than 24 weeks before the predicted randomization date.
- Prior exposure to sotatercept or luspatercept, IMR-687, or gene therapy within 6 months prior to randomization (Day 1).
- Subjects who have major organ damage
Trial locations and contacts
36 sites in 13 countries.
36 sites in 13 countries
Malaysia6 sites
Hospital Sultanah Bahiyah
Alor Star, Kedah, 05460
Hospital Pulau Pinang
George Town, Pulau Pinang, 10450
Hospital Raja Permaisuri Bainun
Ipoh, Perak, 30450
Hospital Sultanah Aminah Johor Bharu
Johor Bahru, Johor, 80100
Hospital Queen Elizabeth - Kota Kinabalu
Kota Kinabalu, Sabah, 88586
Hospital Umum Sarawak
Kuching, Sarawak, 93586
Greece4 sites
Aghia Sofia General Children's Hospital
Athens, Attica, 11527
Laiko General Hospital of Athens
Athens, Attica, 11527
University General Hospital of Patras
Patra, Peloponnese, 26504
Ippokrateio General Hospital of Thessaloniki
Thessaloniki, Central Macedonia, 54642
Israel4 sites
Emek Medical Center
Afula, 18101
Rambam Health Care Campus
Haifa, Haifa District, 3109601
Hadassah University Hospital Ein Kerem
Jerusalem, Jerusalem, 9112001
The Galilee Medical Center
Nahariya, Northern District, 2210001
Turkey (Türkiye)4 sites
Hacettepe Üniversitesi
Ankara, 06230
Ege Universitesi Tip Fakultesi
Izmir, 35100
Akdeniz Üniversitesi
Mersin, Mersin, 33110
Mersin Üniversitesi Tıp Fakültesi
Mersin, Mersin, 33110
France3 sites
Hôpital Edouard Herriot
Lyon, Rhone, 69437
Hôpital Necker-Enfants Malades
Paris, 75015
Institut Universitaire du Cancer de Toulouse Oncopole
Toulouse, Haute-Garonn, 31059
Georgia3 sites
M. Zodelava Hematology Centre
Tbilisi, Borjomi, 0112
National Center of Surgery
Tbilisi, 0159
Medinvest - Institute of Hematology and Transfusiology
Tbilisi, 0186
Tunisia3 sites
Centre Hôpital Universitaire Farhat Hached
Sousse, 4000
Centre National de Greffe de la Moelle Osseuse
Tunis, 1006
Hospital Aziza Othmana
Tunis, 1008
United Kingdom3 sites
Whittington Health NHS Trust
London, England, N19 5NF
University College London Hospitals NHS Foundation Trust
London, England, NW1 2PG
Manchester University NHS Foundation Trust
Manchester, England, M13 9WL
Italy2 sites
Azienda Ospedaliera Giuseppe Brotzu
Orbassano, Turin, 10043
Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli
Orbassano, Turin, 10043
Netherlands1 site
Amsterdam Universitair Medische Centra - Academisch Medisch Centrum
Amsterdam, North Holland, 1105 AZ
About this study
Official title: A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects With Beta Thalassemia
A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
Read the detailed description
A phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of IMR-687 (phosphodiesterase (PDE) 9 inhibitor) administered once daily (qd) orally for 36 weeks in 2 populations of adult subjects with β-thalassemia: Population 1 (Transfusion Dependent Thalassemia (TDT) subjects) and Population 2 (Non-Transfusion Dependent Thalassemia (NTDT) subjects).
- Conditions
- β Thalassemia
- Being tested
- IMR-687, Placebo
- Keywords
- Transfusion, TDT, NTDT
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 |
|---|---|---|
| Lower Dose IMR-687 | Experimental | Oral administration of once daily IMR-687 Receives: Drug: IMR-687 |
| Higher 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.
IMR-687 Safety and Tolerability
Incidence and severity of Adverse Events Incidence and severity of Serious Adverse Events
Measured: Baseline to Week 40
Secondary outcomes (8)
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 12 to Week 24
Proportion of patients with ≥33% hematological improvement (as measured by reduced transfusion burden) from Week 12 to Week 24 compared to the 12 weeks prior to Baseline (Day 1)
Measured: Baseline to Week 24
NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 12 to Week 24 in the Absence of Transfusions.
Proportion of subjects with an increase from baseline of ≥1.0 g/dL in mean Hb values at Week 12 to Week 24 in the absence of transfusions.
Measured: Baseline to Week 24
NTDT Patients: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 12 to Week 24 in Absence of Transfusions
Proportion of subjects with an increase from baseline of ≥3% in mean HbF values at Week 12 to Week 24 in absence of transfusions
Measured: Baseline to Week 24
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 24 to Week 36
Proportion of patients with ≥33% hematological improvement from Week 24 to Week 36 compared to the 12 weeks prior to Baseline (Day 1)
Measured: Baseline to Week 36
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50 % Hematological Improvement From Week 12 to Week 24
Proportion of patients with ≥50% hematological improvement from Week 12 to Week 24 compared to the 12 weeks prior to Baseline (Day 1)
Measured: Baseline to Week 24
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50% Hematological Improvement From Week 24 to Week 36
Proportion of patients with ≥50% hematological improvement from Week 24 to Week 36 compared to the 12 weeks prior to Baseline (Day 1)
Measured: Baseline to Week 36
NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 24 to Week 36 in the Absence of Transfusions
Proportion of subjects with an increase from baseline of ≥1.0 g/dL in mean Hb values at Week 24 to Week 36 in the absence of transfusions.
Measured: Baseline to Week 36
NTDT: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 24 to Week 36 in Absence of Transfusions
Proportion of subjects with an increase from baseline of ≥3% in mean HbF values at Week 24 to Week 36 in absence of transfusions
Measured: Baseline to Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2020
- Study start16 Oct 2020
- Primary completion11 Mar 2022
- Study completion4 May 2022
Why it stopped: IMR-BTL-201demonstrated that while IMR-687 was generally well-tolerated, it failed to show any meaningful benefit in transfusion burden or improvement in most disease-related biomarkers. 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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for β ThalassemiaStudies 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
- None (open label)
- Enrolment
- 122 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Imara, Inc.
- Responsible party
- Sponsor
- Investigators
- Steve Luperchio, Study director, Cardurion Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04411082
- Sponsor's protocol ID
- IMR-BTL-201
- Other IDs
- 2019-002989-12, 2019
- Status last verified
- May 2025
- First submitted
- 15 Apr 2020
- Last updated
- 15 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Foong WC, Loh CK, Ho JJ, Lau DS. Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias. Cochrane Database Syst Rev. 2023 Jan 13;1(1):CD013767. doi: 10.1002/14651858.CD013767.pub2.
PubMed 36637054
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 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects With Beta Thalassemia
- ClinicalTrials.gov ID
- NCT04411082
- Acronym / sponsor ID
- IMR-BTL-201
- Conditions
- β Thalassemia
- Keywords
- Transfusion, TDT, NTDT
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: IMR-BTL-201demonstrated that while IMR-687 was generally well-tolerated, it failed to show any meaningful benefit in transfusion burden or improvement in most disease-related biomarkers. So, the sponsor has decided to discontinue this study
- Enrolment
- 122 participants (actual)
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Imara, Inc.
- Study start
- 16 Oct 2020 (actual)
- Primary completion
- 11 Mar 2022 (actual)
- Study completion
- 4 May 2022 (actual)
- First posted
- 2 Jun 2020 (actual)
- Results
- Posted 30 Jun 2022
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 Tolerability of IMR-687 in Subjects with Beta Thalassemia
Detailed description
A phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of IMR-687 (phosphodiesterase (PDE) 9 inhibitor) administered once daily (qd) orally for 36 weeks in 2 populations of adult subjects with β-thalassemia: Population 1 (Transfusion Dependent Thalassemia (TDT) subjects) and Population 2 (Non-Transfusion Dependent Thalassemia (NTDT) subjects).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Lower Dose IMR-687 | Experimental | Drug: IMR-687Oral administration of once daily IMR-687 |
| Higher 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 (9 listed)
- Documented diagnosis of β-thalassemia or HbE/ β-thalassemia in their medical history. Concomitant alpha gene deletion, duplication, or triplication is allowed.
- Documentation of the dates of transfusion events and the number of all pRBC units per event within the 12 weeks prior to the Baseline (Day 1) visit. .
- Must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff.
- TDT Subjects: subjects must be regularly transfused, defined as >3 to 10 pRBC units in the12 weeks prior to Baseline (Day 1) visit and no transfusion-free period for >35 days during that period.
- NTDT subjects: Subjects must be transfusion independent, defined as 0 to ≤3 units of pRBCs received during the 12-week period prior to the Baseline (Day 1) visit, must not be on a regular transfusion program, must be RBC transfusion-free for at least ≥ 4 weeks prior to randomization, and must not be scheduled to start a regular
Key exclusion criteria (9 listed)
- Diagnosis of α-thalassemia (e.g., hemoglobin H [HbH]) or hemoglobin S (HbS)/ β thalassemia.
- Body mass index (BMI) <17.0 kg/m2 or a total body weight <45 kg; or 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).
- Stroke requiring medical intervention ≤24 weeks prior to randomization.
- Platelet count >1000 × 109/L.
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
IMR-687 Safety and Tolerability
Time frame: Baseline to Week 40
Incidence and severity of Adverse Events Incidence and severity of Serious Adverse Events
8 secondary outcomes
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 12 to Week 24
Time frame: Baseline to Week 24
NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 12 to Week 24 in the Absence of Transfusions.
Time frame: Baseline to Week 24
NTDT Patients: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 12 to Week 24 in Absence of Transfusions
Time frame: Baseline to Week 24
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 24 to Week 36
Time frame: Baseline to Week 36
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50 % Hematological Improvement From Week 12 to Week 24
Time frame: Baseline to Week 24
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50% Hematological Improvement From Week 24 to Week 36
Time frame: Baseline to Week 36
NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 24 to Week 36 in the Absence of Transfusions
Time frame: Baseline to Week 36
NTDT: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 24 to Week 36 in Absence of Transfusions
Time frame: Baseline to Week 36
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.
- Derived from this study: PubMed 36637054Foong WC, Loh CK, Ho JJ, Lau DS. Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias. Cochrane Database Syst Rev. 2023 Jan 13;1(1):CD013767. doi: 10.1002/14651858.CD013767.pub2.
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A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
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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.
Steve Luperchio study director
Cardurion Pharmaceuticals
36 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 β Thalassemia, run by Cardurion Pharmaceuticals, Inc. at 36 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 9 inclusion and 9 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 36 sites in Malaysia, Greece, Israel, Turkey (Türkiye), France 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 30 Jun 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?
IMR-687 Safety and Tolerability (time frame: Baseline to Week 40). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04411082, which also lists 1 linked publication.
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.