What side effects are associated with this type of intervention?

Hydroxyurea, a common treatment for Sickle Cell Disease, can cause side effects such as bone marrow suppression, leading to neutropenia, thrombocytopenia, and anemia. Other potential side effects include gastrointestinal disturbances, skin ulcers, and an increased risk of infections. L-glutamine, another treatment, is generally well-tolerated but may cause constipation, nausea, headache, and abdominal pain. Investigational therapies like IMR-687, a PDE9 inhibitor, are still under study, but potential side effects could include gastrointestinal issues, headache, and dizziness, similar to other PDE inhibitors.

What prior FDA approvals exist?

Hydroxyurea is the most established FDA-approved treatment for Sickle Cell Disease, known for its efficacy in reducing vaso-occlusive pain and other complications. It has been shown to improve survival and decrease the need for blood transfusions. Other treatments targeting the pathogenesis of SCD-associated vaso-occlusion include L-glutamine, voxelotor, and crizanlizumab. These therapies work through different mechanisms to reduce pain and improve patient outcomes. While IMR-687, a Phosphodiesterase 9 (PDE9) Inhibitor, is currently under investigation, it represents a novel approach in the landscape of SCD treatment.

What other types of research exist?

Promising alternatives to IMR-687 for Sickle Cell Disease patients include PDE5 inhibitors like sildenafil, which have shown potential in reducing platelet activation and managing pulmonary hypertension associated with SCD. Additionally, NO-based therapeutics may also be beneficial due to their role in vasodilation and reducing complications related to hemolysis.

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Phosphodiesterase 9 (PDE9) Inhibitor

IMR-687 for Sickle Cell Disease

Phase 2

Waitlist Available

Research Sponsored by Imara, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male subjects must be unlikely to impregnate a partner

Be older than 18 years old

Must not have

Subjects with known active hepatitis B or hepatitis C, with active or acute event of malaria or who are known to be positive for human immunodeficiency virus (HIV)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to month 49

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing IMR-687, a medication, in adult patients with Sickle Cell Anemia who were in a previous study. It aims to see if the medication is safe and well-tolerated by monitoring side effects and body responses.

Who is the study for?

This trial is for adults with Sickle Cell Anemia who finished the IMR-SCD-102 study. Participants must be able to consent, not pregnant or likely to become so, and have normal kidney and liver function tests. They can't join if they have severe anemia or high hemoglobin levels, active hepatitis B/C, malaria events, or HIV.

What is being tested?

The trial is testing the long-term safety and effects of a drug called IMR-687 in adults with Sickle Cell Anemia who previously completed a related Phase 2a study.

What are the potential side effects?

While specific side effects are not listed here, this extension study aims to monitor how safe and tolerable the drug IMR-687 is over a longer period after initial trials.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am a male and unlikely to father a child.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I do not have active hepatitis B or C, malaria, or HIV.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to month 49

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to month 49

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to month 49 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Proportion of patients with adverse events and serious adverse events

Proportion of patients with changes in clinical laboratory tests

Proportion of patients with changes in safety cardiac parameters

+1 more

Side effects data

From 2020 Phase 2 trial • 100 Patients • NCT03401112

42%

Sickle cell anaemia with crisis

25%

Upper respiratory tract infection

17%

Headache

17%

Influenza like illness

17%

Pain

17%

Nasopharyngitis

17%

Nausea

13%

Uterine leiomyoma

Pain in extremity

Ocular icterus

Hepatic lesion

Abdominal pain upper

Vomiting

Musculoskeletal pain

Diarrhoea

Fatigue

Oropharyngeal pain

100%

80%

60%

40%

20%

Study treatment Arm

All Placebo

Placebo (With HU)

IMR-687 50 mg/100 mg (Without HU)

IMR-687 50 mg/100 mg (With HU)

All IMR-687

IMR-687 100 mg/200 mg (Without HU)

Placebo (Without HU)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Open LabelExperimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

IMR-687

2018

Completed Phase 2

~170

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, red blood cell transfusions, and investigational therapies like PDE9 inhibitors. Hydroxyurea works by increasing fetal hemoglobin (Hb F) production, which reduces the sickling of red blood cells and decreases vaso-occlusive events. Red blood cell transfusions provide normal red blood cells to improve oxygen delivery and reduce complications. PDE9 inhibitors, like IMR-687, aim to increase cyclic guanosine monophosphate (cGMP) levels, which can reduce inflammation and improve blood flow. These mechanisms are crucial for SCD patients as they help prevent painful crises, reduce organ damage, and improve overall quality of life.

Magnesium for treating sickle cell disease.CPC-111 (Cypros Pharmaceutical Corp).