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Luspatercept +/− Hydroxyurea for Myeloproliferative Disorders

Phase 2
Waitlist Available
Led By Abhishek Mangaonkar, M.D.
Research Sponsored by Mayo Clinic
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Refractory or unlikely to respond (erythropoietin [EPO] >= 200 U/L) or documented intolerance to erythropoiesis stimulating agent (ESA). Patients should have either a documented intolerance or contraindication to ESA or a lack of response after ESA therapy trial of at least four weeks; and ESA therapy should be stopped four weeks prior to trial enrollment
Age >= 18 years
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 6 months
Awards & highlights

Study Summary

This trial is testing the effects of luspatercept with or without hydroxyurea on patients with myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis or unclassifiable with ring sideroblasts.

Who is the study for?
Adults diagnosed with certain myelodysplastic/myeloproliferative neoplasms who have not been treated with luspatercept or sotatercept, but may have had other treatments. They should need blood transfusions or have symptomatic anemia and be in a condition to participate as judged by performance scores. Women of childbearing age must test negative for pregnancy and agree to contraception; men must also agree to use contraception.Check my eligibility
What is being tested?
The trial is testing the effectiveness of luspatercept alone or combined with hydroxyurea in treating specific blood disorders. Luspatercept aims to stimulate the immune system and stop cancer growth, while hydroxyurea blocks enzymes needed for cell growth. The study will determine safe doses and how well patients respond.See study design
What are the potential side effects?
Luspatercept might cause side effects like tiredness, muscle aches, headaches, joint pain, nausea, dizziness, increased risk of blood clots and possible allergic reactions. Hydroxyurea can lead to low blood counts increasing infection risk, mouth sores, skin changes and gastrointestinal upset.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can't use or didn't respond to drugs for anemia related to my condition.
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I am 18 years old or older.
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I have been diagnosed with a specific type of blood disorder that includes ringed sideroblasts.
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I haven't taken luspatercept-aamt or sotatercept, but may have had other treatments.
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I can take care of myself and perform daily activities.
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I've needed blood transfusions or have had symptoms of anemia recently.
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My kidney function, measured by creatinine clearance, is adequate.
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I am 18 years old or older.
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I have been diagnosed with a specific type of blood disorder that includes ringed sideroblasts.
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I can take care of myself and am up and about more than half of my waking hours.
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I've needed blood transfusions or have had symptoms of anemia recently.
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I have not had surgery to remove my uterus or both ovaries.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 6 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Erythroid response rate
Secondary outcome measures
Duration of response
Incidence of adverse events (AEs)
Leukemia-free survival
+2 more

Side effects data

From 2021 Phase 3 trial • 336 Patients • NCT02604433
42%
Upper respiratory tract infection
35%
Headache
32%
Back pain
23%
Arthralgia
22%
Bone pain
22%
Cough
21%
Pyrexia
18%
Diarrhoea
18%
Oropharyngeal pain
17%
Fatigue
16%
Pharyngitis
15%
Pain in extremity
13%
Nausea
13%
Vomiting
13%
Myalgia
13%
Dizziness
13%
Abdominal pain
12%
Asthenia
11%
Abdominal pain upper
11%
Influenza
10%
Hypertension
9%
Dyspepsia
9%
Influenza like illness
9%
Musculoskeletal pain
9%
Nasal congestion
8%
Urticaria
8%
Gastroenteritis
8%
Nasopharyngitis
7%
Toothache
7%
Tonsillitis
7%
Hyperuricaemia
6%
Urinary tract infection
6%
Pain
6%
Viral upper respiratory tract infection
6%
Neck pain
6%
Osteoporosis
5%
Constipation
5%
Injection site pain
5%
Transfusion reaction
5%
Alanine aminotransferase increased
5%
Liver iron concentration increased
5%
Spinal pain
5%
Lethargy
5%
Menstruation irregular
4%
Musculoskeletal chest pain
4%
Fall
2%
Anaemia
1%
Transient ischaemic attack
1%
Cerebrovascular accident
1%
Cholangitis
1%
Cholecystitis acute
1%
Extramedullary haemopoiesis
1%
Septic shock
1%
Cellulitis
1%
Pneumonia
1%
Deep vein thrombosis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Luspatercept + BSC
Placebo + BSC

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort A (luspatercept, hydroxyurea)Experimental Treatment6 Interventions
Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity. Patients undergo blood sample collection, bone marrow biopsy and aspirate at baseline and on study.
Group II: Cohort A (luspatercept)Experimental Treatment5 Interventions
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity. Patients undergo blood sample collection, bone marrow biopsy and aspirate at baseline and on study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Hydroxyurea
2006
Completed Phase 4
~3620
Biospecimen Collection
2004
Completed Phase 2
~1920
Bone Marrow Aspiration
2011
Completed Phase 2
~1740
Luspatercept
2018
Completed Phase 3
~1050

Find a Location

Who is running the clinical trial?

Mayo ClinicLead Sponsor
3,176 Previous Clinical Trials
3,758,038 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,608 Previous Clinical Trials
40,915,345 Total Patients Enrolled
Abhishek Mangaonkar, M.D.Principal InvestigatorMayo Clinic in Rochester

Media Library

Luspatercept (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05005182 — Phase 2
Myelodysplastic/Myeloproliferative Neoplasm Research Study Groups: Cohort A (luspatercept), Cohort A (luspatercept, hydroxyurea)

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are new participants still being welcomed into this research project?

"Yes, the information on clinicaltrials.gov suggests that this trial is actively searching for participants. The clinical trial was originally posted on 1/17/2022 and was last updated on 1/19/2022. The study is admitting 54 patients across 1 locations."

Answered by AI

How many individuals have signed up for this research project?

"That is correct. The listing on clinicaltrials.gov reveals that this study is looking for 54 individuals at 1 location. This information was first posted on January 17th, 2022 and was most recently updated on January 19th, 2022."

Answered by AI

When might we see Luspatercept hit the market?

"Luspatercept was given a safety score of 2 because, while there is data supporting its safety, none of it points to the efficacy of the medication."

Answered by AI

What are the standard applications for Luspatercept?

"Luspatercept is not only effective in treating anemia, but also chemoradiation, thrombocythemia, essential, and meningiomas."

Answered by AI
~1 spots leftby Mar 2025