Study Summary
This trial is testing the combination of venetoclax and azacitidine to see if it is effective and has manageable side effects in treating patients with high-risk myelodysplastic syndrome.
Eligible Conditions
- Myelodysplastic Syndrome
- Chronic Myelomonocytic Leukemia
- Therapy-Related Myelodysplastic Syndrome
- Refractory Myelodysplastic Syndromes
Treatment Effectiveness
Effectiveness Progress
Study Objectives
2 Primary · 14 Secondary · Reporting Duration: Up to 5 years
Year 5
Duration of response
Year 5
Event-free survival
Year 5
Overall survival
Year 5
Progression-free survival
Up to 5 years
Biological markers of response
Effects of therapy on myelodysplastic syndrome
Incidence of adverse events (Phase I)
Overall response rate (Phase 2)
Rate of CR
Rate of bone marrow blast response
Rate of cytogenetic response
Rate of hematologic improvement
Rate of marrow/morphologic CR
Rate of platelet transfusion independence
Rate of red blood cell transfusion independence
Therapeutic procedure
Time to transformation to acute myeloid leukemia
Up to 8 weeks
Maximum tolerated dose (MTD) of the combination regimen of venetoclax and azacitidine (Phase I)
Trial Safety
Safety Progress
Side Effects for
Venetoclax + Rituximab
61%Neutropenia
39%Diarrhoea
21%Upper respiratory tract infection
21%Nausea
18%Fatigue
18%Cough
14%Constipation
14%Anaemia
14%Pyrexia
12%Thrombocytopenia
11%Headache
11%Nasopharyngitis
11%Insomnia
10%Bronchitis
9%Sinusitis
8%Infusion related reaction
8%Back pain
8%Vomiting
8%Pneumonia
7%Pharyngitis
7%Rash
7%Abdominal pain
6%Oedema peripheral
6%Hyperkalaemia
6%Productive cough
6%Hypokalaemia
6%Neutrophil count decreased
6%Dizziness
6%Lower respiratory tract infection
6%Dyspnoea
6%Urinary tract infection
6%Hypertension
6%Arthralgia
5%Pruritus
5%Alanine aminotransferase increased
5%Conjunctivitis
5%Oropharyngeal pain
4%Chills
4%Febrile neutropenia
4%Oral herpes
4%Decreased appetite
2%Influenza
2%Tumour lysis syndrome
2%Muscle spasms
2%Autoimmune haemolytic anaemia
2%Lung infection
2%Squamous cell carcinoma
1%Haemophilus infection
1%Meningitis
1%Pneumonia influenzal
1%Pneumonia streptococcal
1%Respiratory tract infection
1%Sepsis
1%Tooth abscess
1%Deep vein thrombosis
1%Oesophageal obstruction
1%Hyperpyrexia
1%Dehydration
1%Ascites
1%Angina pectoris
1%Gastrointestinal haemorrhage
1%Erysipelas
1%Viral upper respiratory tract infection
1%Diabetes mellitus
1%Colorectal cancer
1%Cervical dysplasia
1%Eye haemorrhage
1%Sudden cardiac death
1%Urinary tract infection pseudomonal
1%Lacunar infarction
1%Disseminated intravascular coagulation
1%Uterine haemorrhage
1%Peritoneal tuberculosis
1%Humerus fracture
1%Dyspepsia
1%Nephrolithiasis
1%Myelodysplastic syndrome
1%Immune thrombocytopenic purpura
1%Ventricular tachycardia
1%Deafness
1%Small intestinal obstruction
1%Diverticulitis
1%Herpes zoster
1%Status epilepticus
1%Pancytopenia
1%Cardiac failure
1%Myocardial infarction
1%Vertigo
1%Herpes simplex otitis externa
1%Moraxella infection
1%Skin cancer
1%Rhinovirus infection
1%Adenocarcinoma gastric
1%Campylobacter gastroenteritis
1%Cystitis
1%Gastroenteritis rotavirus
1%Viral infection
1%Respiratory tract infection fungal
1%Colon cancer
1%Hyperphosphataemia
1%Malignant melanoma
1%Metastatic malignant melanoma
1%Pancreatic carcinoma
1%Prostatic adenoma
1%Acute kidney injury
1%Bronchiectasis
1%Lung disorder
1%Pulmonary embolism
1%Appendicitis
1%Crohn's disease
1%Bile duct obstruction
1%Respiratory tract infection viral
1%Acute respiratory failure
1%Fluid overload
1%Basal cell carcinoma
Trial Design
1 Treatment Group
Treatment (venetoclax, azacitidine)
1 of 1
Experimental Treatment
116 Total Participants · 1 Treatment Group
Primary Treatment: Venetoclax · No Placebo Group · Phase 1 & 2
Treatment (venetoclax, azacitidine)Experimental Group · 2 Interventions: Venetoclax, Azacitidine · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Venetoclax
FDA approved
Azacitidine
FDA approved
Trial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 5 years
Who is running the clinical trial?
National Cancer Institute (NCI)NIH
13,153 Previous Clinical Trials
41,162,217 Total Patients Enrolled
M.D. Anderson Cancer CenterLead Sponsor
2,827 Previous Clinical Trials
1,792,731 Total Patients Enrolled
Guillermo Garcia-ManeroPrincipal InvestigatorM.D. Anderson Cancer Center
9 Previous Clinical Trials
1,539 Total Patients Enrolled
Eligibility Criteria
Age 18+ · All Participants · 8 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:References
- Bazinet, Alexandre, Faezeh Darbaniyan, Elias Jabbour, Guillermo Montalban-Bravo, Maro Ohanian, Kelly Chien, Tapan Kadia, et al.. 2022. “Azacitidine Plus Venetoclax in Patients with High-risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukaemia: Phase 1 Results of a Single-centre, Dose-escalation, Dose-expansion, Phase 1–2 Study”. The Lancet Haematology. Elsevier BV. doi:10.1016/s2352-3026(22)00216-2.
- Bazinet A, Darbaniyan F, Jabbour E, Montalban-Bravo G, Ohanian M, Chien K, Kadia T, Takahashi K, Masarova L, Short N, Alvarado Y, Yilmaz M, Ravandi F, Andreeff M, Kanagal-Shamanna R, Ganan-Gomez I, Colla S, Qiao W, Huang X, McCue D, Mirabella B, Kantarjian H, Garcia-Manero G. Azacitidine plus venetoclax in patients with high-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: phase 1 results of a single-centre, dose-escalation, dose-expansion, phase 1-2 study. Lancet Haematol. 2022 Oct;9(10):e756-e765. doi: 10.1016/S2352-3026(22)00216-2. Epub 2022 Sep 2.
- 2019. "Venetoclax and Azacitidine for the Treatment of High-Risk Recurrent or Refractory Myelodysplastic Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04160052.
Frequently Asked Questions
Is this research currently enrolling participants?
"As per clinicaltrials.gov, this research project is presently recruiting participants. It was first posted on October 1st 2019 and last modified on June 8th 2022." - Anonymous Online Contributor
Unverified Answer
In what other capacities has Venetoclax been examined?
"At present, 340 Venetoclax trials are active with 54 in Phase 3. Toronto is one of the prime locations for these studies; however, there exist 11,189 clinical trial sites that conduct research on this medication." - Anonymous Online Contributor
Unverified Answer
How many individuals are currently participating in this trial's proceedings?
"Absolutely. The clinicaltrials.gov website attests to the fact that this trial is currently recruiting individuals, as it was initially posted on October 1st 2019 and recently updated on June 8th 2022. 116 patients are needed from one particular medical site for successful completion of the experiment." - Anonymous Online Contributor
Unverified Answer
To what maladies is Venetoclax typically prescribed as a remedy?
"Venetoclax is regularly used for induction chemotherapy, yet can also be beneficial in alleviating the symptoms associated with refractory anemias, leukemia, myelocytic and acute multilineage dysplasia." - Anonymous Online Contributor
Unverified Answer