150 Participants Needed

DISC-0974 for Myelofibrosis

Recruiting at 13 trial locations
DM
Overseen ByDisc Medicine Clinical Trials
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Disc Medicine, Inc
Must be taking: JAK inhibitors
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

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.12345

Who Is on the Research Team?

WS

Will Savage, MD PhD

Principal Investigator

Disc Medicine

Are You a Good Fit for This Trial?

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.
See 4 more

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.
See 4 more

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

  • DISC-0974
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.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Phase 2: ExpansionExperimental Treatment1 Intervention
Group II: Phase 1b: Dose EscalationExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Disc Medicine, Inc

Lead Sponsor

Trials
6
Recruited
580+

Published Research Related to This Trial

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.
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.
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.
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]

Citations

Clinicopathologic features and prognostic indicators in Chinese patients with myelofibrosis. [2022]
Impact of ruxolitinib on the natural history of primary myelofibrosis: a comparison of the DIPSS and the COMFORT-2 cohorts. [2021]
Disease and Clinical Characteristics of Patients With a Clinical Diagnosis of Myelofibrosis Enrolled in the MOST Study. [2022]
Improving survival trends in primary myelofibrosis: an international study. [2012]
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]
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