DISC-0974 for Myelofibrosis

Recruiting at 13 trial locations

Overseen By Disc Medicine Clinical Trials

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Disc Medicine, Inc

Must be taking: JAK inhibitors

Must not be taking: Iron chelation

Disqualifiers: Hereditary hemochromatosis, Hemoglobinopathy, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

This phase 1b/2a open-label study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of DISC-0974 as well as categorize the effects on anemia response in subjects with myelofibrosis or myelodysplastic syndrome and anemia.

Will I have to stop taking my current medications?

Yes, you may need to stop taking certain medications before joining the trial. There is a required washout period (time without taking certain medications) of at least 28 days for specific treatments like androgens, erythropoietin, and others. If you are on JAK inhibitors or hydroxyurea, a 60-day washout period is needed if you discontinue them before screening.

What data supports the idea that DISC-0974 for Myelofibrosis is an effective drug?

The available research does not provide specific data on the effectiveness of DISC-0974 for Myelofibrosis. However, it does mention another drug, ruxolitinib, which has shown to improve survival in patients with primary myelofibrosis compared to placebo or other therapies. Patients taking ruxolitinib lived longer, with a survival time of 5 years compared to 3.5 years for those not taking it. This suggests that ruxolitinib is an effective treatment for Myelofibrosis, but there is no direct data on DISC-0974 in the provided information. ^{1 2 3 4 5}

Research Team

Will Savage, MD PhD

Principal Investigator

Disc Medicine

Eligibility Criteria

Adults over 18 with myelofibrosis and anemia can join this trial. They should have a certain level of disease severity, be able to perform daily activities, and have kidneys that work well enough. Participants need to stop some treatments before joining and meet specific blood count criteria. People who've had recent major surgery or other cancers (with exceptions), active infections, or severe heart problems cannot join.

Inclusion Criteria

I can take care of myself and perform daily activities.

My bone marrow biopsy shows I have a high-risk form of myelofibrosis.

I am not expected to have a stem cell transplant within the next 8 months.

Exclusion Criteria

I do not have severe allergies, heart issues, active Hepatitis B or C, or uncontrolled HIV.

Laboratory Exclusions like peripheral blood myeloblasts, positive direct antiglobulin test

I haven't had recent iron removal treatment or changed my blood thinner regimen.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1b: Dose Escalation

DISC-0974 is administered subcutaneously every 4 weeks to assess safety, tolerability, pharmacokinetics, and pharmacodynamics

8-12 weeks

3 visits (in-person)

Phase 2: Expansion

DISC-0974 is administered subcutaneously every 4 weeks to evaluate effects on anemia response

16 weeks

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• DISC-0974 (Unknown)

Trial Overview The study is testing DISC-0974's safety and how it affects the body in patients with myelofibrosis-related anemia. It's an early-stage trial where everyone gets the drug; there are no comparison groups.

Participant Groups

Group I: Phase 2: Expansion Experimental Treatment 1 Intervention

In the Phase 2 (expansion) portion of the study, DISC-0974 will be administered subcutaneously every 4 weeks.

Group II: Phase 1b: Dose Escalation Experimental Treatment 1 Intervention

In the Phase 1b (dose-escalation) portion of the study, DISC-0974 will be administered subcutaneously every 4 weeks.

Who Is Running the Clinical Trial?

Disc Medicine, Inc

Lead Sponsor

Trials

6

Recruited

580+

Findings from Research

In a study of 270 Chinese patients with myelofibrosis (MF), the median overall survival for primary MF was 66 months, with age over 65, low platelet count, and leukemic transformation identified as significant negative prognostic factors.

The study found that unfavorable karyotypes and circulating blasts greater than 1% were also associated with poorer leukemia-free survival, indicating that these factors could help predict outcomes in MF patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinicopathologic features and prognostic indicators in Chinese patients with myelofibrosis. Gill, H., Leung, AY., Chan, CC., et al.[2022]

In a study comparing 100 patients with primary myelofibrosis (PMF) treated with ruxolitinib to 350 patients from the dynamic IPSS (DIPSS) study, those receiving ruxolitinib had a significantly longer survival time of 5 years compared to 3.5 years for the DIPSS cohort.

The analysis indicated that ruxolitinib may alter the natural progression of PMF, with a hazard ratio of 0.61 suggesting a 39% reduction in the risk of death compared to the DIPSS patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Impact of ruxolitinib on the natural history of primary myelofibrosis: a comparison of the DIPSS and the COMFORT-2 cohorts. Passamonti, F., Maffioli, M., Cervantes, F., et al.[2021]

In a study of 205 patients with lower-risk myelofibrosis (MF), it was found that over half had low hemoglobin and hematocrit levels, indicating significant blood-related issues, and nearly all patients had comorbid conditions, with hypertension being the most common.

At the time of enrollment, 55.6% of patients were on MF-directed treatment, primarily hydroxyurea and ruxolitinib, highlighting the common treatment patterns for managing lower-risk MF.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Disease and Clinical Characteristics of Patients With a Clinical Diagnosis of Myelofibrosis Enrolled in the MOST Study. Gerds, AT., Lyons, RM., Colucci, P., et al.[2022]

A study of 802 patients with primary myelofibrosis (PMF) showed that those diagnosed between 1996 and 2007 had a median survival of 6.5 years, compared to 4.6 years for those diagnosed from 1980 to 1995, indicating a significant improvement in survival rates over time.

Patients diagnosed more recently also exhibited fewer severe symptoms, such as marked anemia and high leukocyte counts, suggesting that while overall survival has improved, those in high-risk categories still face challenges that need to be addressed with new therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Improving survival trends in primary myelofibrosis: an international study. Cervantes, F., Dupriez, B., Passamonti, F., et al.[2012]

In a study of 172 patients with primary myelofibrosis, several time-dependent factors were identified as significant predictors of death and blast phase (BP) progression, including the onset of anemia, leukocytosis, and thrombocytopenia.

Leukocytosis was particularly notable, as its occurrence during follow-up was linked to a higher risk of BP, suggesting that patients with this condition should be closely monitored to better assess disease severity and inform treatment decisions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Identification during the follow-up of time-dependent prognostic factors for the competing risks of death and blast phase in primary myelofibrosis: a study of 172 patients. Morel, P., Duhamel, A., Hivert, B., et al.[2021]

References

1. England pubmed.ncbi.nlm.nih.gov

Clinicopathologic features and prognostic indicators in Chinese patients with myelofibrosis. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Impact of ruxolitinib on the natural history of primary myelofibrosis: a comparison of the DIPSS and the COMFORT-2 cohorts. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Disease and Clinical Characteristics of Patients With a Clinical Diagnosis of Myelofibrosis Enrolled in the MOST Study. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Improving survival trends in primary myelofibrosis: an international study. [2012]

5. United States pubmed.ncbi.nlm.nih.gov

Identification during the follow-up of time-dependent prognostic factors for the competing risks of death and blast phase in primary myelofibrosis: a study of 172 patients. [2021]