A Phase 2 interventional study of Azacitidine and Ascorbic acid in Myelodysplastic Syndromes, Myeloproliferative Neoplasm and Acute Myeloid Leukemia, sponsored by Case Comprehensive Cancer Center. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-08-11.
Sponsored by Case Comprehensive Cancer Center · Phase 2, Interventional, and Treatment
Among 130 studies testing azacitidine in Myelodysplastic Syndromes — 13 open to participants now, 25 under way, 3 of them phase 3. Azacitidine · Myelodysplastic Syndromes.
Among the 130 studies testing azacitidine in Myelodysplastic Syndromes, this one is the 102nd largest by enrollment of the 126 interventional ones that give a count, is one of 5 that started in 2018, is one of 63 in phase 2, and was the 95th registered.
1,997 studies on the registry are indexed under Myelodysplastic Syndromes; 324 are open to participants now.
Browse Myelodysplastic Syndromes studies →13 of the 130 studies testing azacitidine in Myelodysplastic Syndromes are open now, in 12 countries. Recruiting first, the newest first:
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Sites in China
Ages 18–70
City of Hope Medical Center · Sites in United States
Ages 18–75
Medical College of Wisconsin · Sites in United States
Ages 18+
Institut de Recherches Internationales Servier · Sites in United States, Australia, Brazil and 7 more countries
Ages 18+
Beijing 302 Hospital · Sites in China
Ages 18–70
By country, each count listing its studies:
Azacitidine: first FDA approval 2004 (VIDAZA), first EMA approval 2008. What it is approved for, and how studies give it, is on its page →
Of the 262 Myelodysplastic Syndromes studies recruiting now, 165 (63%) set no upper age limit and 70 (27%) stop at 75 or younger; 57 take children under 18; 17 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
130 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 50 · Phase 2 86 · Phase 3 12 · Phase 4 3
First in 2000; this study’s year is marked. 5 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 12 is below the median of 40 across 1,611 interventional studies indexed under Myelodysplastic Syndromes.
Median enrollment 38 across the 126 interventional studies. Of the 88 that completed or were terminated, 42 (48%) have results posted.
This tab as a report to print or save as PDF → With an account.
32 of 130 have an industry lead sponsor. The 10 most frequent:
Case Comprehensive Cancer Center is the lead sponsor of 494 studies on the registry; 59 are open to participants now.
Of its 75 completed or terminated interventional studies of FDA-regulated products, 46 (61%) have results posted.
22 studies in Myelodysplastic Syndromes by Case Comprehensive Cancer Center: 1 open now, 2 under way, none of them phase 3. Of 20 finished, 5 (25%) have results posted.
Among the 22 studies in Myelodysplastic Syndromes by Case Comprehensive Cancer Center, this one is the 16th largest by enrollment of the 21 interventional ones that give a count, is the only one that started in 2018, is one of 13 in phase 2, and was the 19th registered.
Case Comprehensive Cancer Center’s Myelodysplastic Syndromes studies by the year they started, since 1997.
9 of the studies testing azacitidine in Myelodysplastic Syndromes that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06465953Phase 3Institut de Recherches Internationales ServierIvosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation | 2027-11-01 |
| NCT05788679Karolinska University HospitalA Study Using Subject-specific MRD to Adopt Treatment After HSCT for Subjects With MDS | 2026-12 |
| NCT00392353National Cancer Institute (NCI)Vorinostat and Azacitidine in Treating Patients With Myelodysplastic Syndromes or Acute Myeloid Leukemia | 2026-12-31 |
| NCT06502145Medical College of WisconsinIadademstat With Hypomethylating Agent in Patients With Myelodysplastic Syndrome | 2026-12-31 |
| NCT03862157M.D. Anderson Cancer CenterAzacitidine, Venetoclax, and Pevonedistat in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia | 2027-01-31 |
Add all 9 to your calendar (.ics) → With an account.
Among the 130 studies testing azacitidine in Myelodysplastic Syndromes, from their registry updates:
Among Case Comprehensive Cancer Center’s 22 in Myelodysplastic Syndromes:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
The purpose of this study is to evaluate the efficacy of treatment with azacitidine (an FDA approved drug for the treatment of MDS) and high dose ascorbic acid in patients with TET2 mutations. This approach is intended to enhance the enzymatic activity of TET2 protein, which in term may help to improve counts and symptoms, related to Myelodysplastic Syndromes and Acute Myeloid Leukemia. This combination is specific to individuals who carry this mutation.
Primary Endpoint To estimate the overall response rate (ORR) of the combination of standard dose azacitidine and oral dose of ascorbic acid in patients with MDS, AML, and MDS / Myeloproliferative Neoplasm (MPN) overlap with heterozygous TET2 mutations
Secondary Endpoints
Study Design This is an open-label, phase II study that will be conducted at Cleveland Clinic, Taussig Cancer Institute.
Azacitidine will be administered intravenously or subcutaneously at a fixed dose of 75mg/m2/day for 7 consecutive days, (allowing for weekends, and holidays) of each 28-day cycle. Ascorbic acid will be administered orally daily at 1 g/day three days prior to start azacitidine and then continues daily for a total of 28 days of each 28 day cycle.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patient with AML according to 2016 WHO criteria.
Relapse/Refractory is defined as at least 1 course of treatment for AML excluding any patients treated with azacitidine or decitabine.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Azacitidine will be administered intravenously or subcutaneously at a fixed dose of 75mg/m2/day for 7 consecutive days, (allowing for weekends, and holidays) of each 28-day cycle. Ascorbic acid will be administered orally daily at 1 g/day three days prior to start azacitidine and then continues daily for a total of 28 days of each 28 day cycle.
Drug: Azacitidine · Drug: Ascorbic acid
Azacitidine will be administered intravenously or subcutaneously at a fixed dose of 75mg/m2/day for 7 consecutive days, allowing interruptions for weekends and holidays within each 28-day cycle. No dose modifications will be permitted during the treatment period.
Ascorbic acid will be administered orally daily at 1 g/day three days prior to start azacitidine and then continues daily for a total of 28 days of each 28 day cycle. No dose modifications will be permitted during the treatment period.
Number of patients with response per MDS International Working Group 2006 Criteria
A binary indicator of overall response, defined as achieving CR, PR, or HI) per 2006 International Working Group (IWG) criteria Responses for MDS patients: Complete Response (CR): Bone marrow: ≤ 5% myeloblasts with normal maturation. Peripheral blood: Hgb ≥ 11 g/dL Platelets ≥ 100 × 109/L Neutrophils ≥ 1.0 × 109/L, Blasts 0% Partial response (PR): All CR criteria if abnormal before treatment except: bone marrow blasts decreased by ≥ 50% over pretreatment but still \> 5% Hematologic Improvement (HI): (responses must last at least 8 weeks) Erythroid response \[HI-E; (if pretreatment Hgb \< 11 g/dL)\]; Hgb increase by ≥ 1.5 g/dL; Relevant reduction of units of red blood cell (RBC) transfusions by an absolute number of at least 4 RBC transfusions/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for an Hgb of ≤ 9.0 g/dL pretreatment will count in the RBC transfusion response evaluation.
Time frame: 171 Days (6 cycles of 28 days plus 3 day loading period)
Number of AML patients with response
Complete Response (CR): Bone marrow: ≤ 5% myeloblasts with normal maturation of all cell lines, Absolute neutrophil count (ANC) \>/= 1.0 X 109/L, platelet count \>/= 100 X 109/L, no detectable Auer rods and no extramedulary leukemia. Complete Response (CR) with incomplete hematologic recovery (CRi): Responses as in CR but ANC \< 1.0 X 109/L. Complete Response (CR) with incomplete platelets recovery (CRp): Responses as in CR but platelets \< 100 X 109/L. Partial response (PR): All CR criteria if abnormal before treatment except: bone marrow blasts decreased by ≥ 50% over pretreatment but still \> 5%.
Time frame: 171 Days (6 cycles of 28 days plus 3 day loading period)
Incidence of adverse events
Incidence of adverse events of the combination defined by CTCAE 4.1 criteria. Toxicity will be any drug related Grade 3 or 4 toxicity.
Time frame: 171 Days (6 cycles of 28 days plus 3 day loading period)
Response duration
Response duration to the combination recorded from start of treatment to progression
Time frame: 171 Days (6 cycles of 28 days plus 3 day loading period)
Overall survival
Overall survival measured from start of treatment to death or last follow up
Time frame: Up to 1 year from end of treatment
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Mar 2018 to primary completion in Jan 2021, 33 months.
Of 227 non-industry phase 2 interventional studies under Myelodysplastic Syndromes, half reached primary completion within 76 months of starting (the median), counting those still running; a quarter within 44 and three quarters within 116. This study's 33 months is below that.
Counting every sponsor, half of 279 studies reached it within 66 months; a quarter within 40 and three quarters within 104.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Azacitidine · Ascorbic Acid
Case Comprehensive Cancer Center →