All > Anemia > Myelodysplastic Syndrome

Participants with gene mutations other than SF3B1 for Anemia

Moffitt Cancer Center, Tampa, FL
Anemia+2 More ConditionsLuspatercept - Drug
Eligibility
18+
All Sexes

Study Summary

This trial will test if luspatercept can reduce the need for blood transfusions in participants with anemia caused by MDS or MDS/MPN. It will also assess the safety and tolerability of luspatercept.

Eligible Conditions
  • Myeloproliferative Neoplasms
  • Anemia
  • Myelodysplastic Syndrome

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 2

Similar Trials

1
AG-120
1
DFV890 low dose
1
NIS793

Study Objectives

1 Primary · 4 Secondary · Reporting Duration: End of treatment, up to 18 months

Month 18
ASC specks changes with response
Month 19
Incidence of treatment related adverse events
Month 18
Duration of Response
Hematological Improvement
RBC Transfusion Independence

Trial Safety

Phase-Based Safety

2 of 3
This is further along than 68% of similar trials

Similar Trials

1
AG-120
1
DFV890 low dose
1
NIS793

Side Effects for

Luspatercept + BSC
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%Vomiting
13%Dizziness
13%Nausea
13%Myalgia
13%Abdominal pain
12%Asthenia
11%Abdominal pain upper
11%Influenza
10%Hypertension
9%Musculoskeletal pain
9%Dyspepsia
9%Influenza like illness
9%Nasal congestion
8%Gastroenteritis
8%Nasopharyngitis
8%Urticaria
7%Toothache
7%Tonsillitis
7%Hyperuricaemia
6%Neck pain
6%Urinary tract infection
6%Osteoporosis
6%Pain
6%Viral upper respiratory tract infection
5%Lethargy
5%Alanine aminotransferase increased
5%Menstruation irregular
5%Constipation
5%Spinal pain
5%Injection site pain
5%Transfusion reaction
5%Liver iron concentration increased
4%Musculoskeletal chest pain
4%Fall
2%Anaemia
1%Transient ischaemic attack
1%Cholecystitis acute
1%Extramedullary haemopoiesis
1%Septic shock
1%Cholangitis
1%Cerebrovascular accident
1%Cellulitis
1%Pneumonia
1%Deep vein thrombosis
This histogram enumerates side effects from a completed 2021 Phase 3 trial (NCT02604433) in the Luspatercept + BSC ARM group. Side effects include: Upper respiratory tract infection with 42%, Headache with 35%, Back pain with 32%, Arthralgia with 23%, Bone pain with 22%.

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.

Trial Design

2 Treatment Groups

Participants with gene mutations other than SF3B1
1 of 2
Participants with SF3B1 mutation
1 of 2

Experimental Treatment

70 Total Participants · 2 Treatment Groups

Primary Treatment: Participants with gene mutations other than SF3B1 · No Placebo Group · Phase 2

Participants with gene mutations other than SF3B1
Drug
Experimental Group · 1 Intervention: Luspatercept · Intervention Types: Drug
Participants with SF3B1 mutation
Drug
Experimental Group · 1 Intervention: Luspatercept · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Luspatercept
2016
Completed Phase 3
~1020

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: end of treatment, up to 18 months

Who is running the clinical trial?

H. Lee Moffitt Cancer Center and Research InstituteLead Sponsor
514 Previous Clinical Trials
128,206 Total Patients Enrolled
1 Trials studying Anemia
9 Patients Enrolled for Anemia
Bristol-Myers SquibbIndustry Sponsor
2,542 Previous Clinical Trials
4,019,586 Total Patients Enrolled
2 Trials studying Anemia
237 Patients Enrolled for Anemia
Rami Komrokji, MDPrincipal InvestigatorMoffitt Cancer Center
5 Previous Clinical Trials
225 Total Patients Enrolled
1 Trials studying Anemia
9 Patients Enrolled for Anemia

Eligibility Criteria

Age 18+ · All Participants · 1 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You must either agree to not have sex or use highly effective birth control for a certain period of time before and after the study treatment.
References

Frequently Asked Questions

Could you please provide information about the enrollment eligibility for this project?

"Clinicaltrials.gov asserts that this trial, which was initially made public on February 8th 2023, is currently searching for participants with the most recent edit occuring on February 16th 2023." - Anonymous Online Contributor

Unverified Answer

Does the FDA recognize any additional genetic mutations for this treatment?

"Since there is some data confirming the safety of participants with gene mutations other than SF3B1, but none confirming efficacy, our team assigned it a rating of 2." - Anonymous Online Contributor

Unverified Answer

To what extent has participation been demonstrated in this clinical experiment?

"Affirmative. According to the information on clinicaltrials.gov, this investigation is actively searching for suitable participants; it was originally posted in February 8th 2023 and last updated a week later. The trial needs 70 recruits from 1 medical centre." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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