Myelodysplastic SyndromePhase 1NCT01290302
Blinded Cross-Over Bioequivalence (BE) Trial of Luitpold Azacitidine vs Vidaza
A Phase 1 study testing Luitpold Azacitidine and Vidaza® in people with Myelodysplastic Syndrome and Myelofibrosis, run by American Regent, Inc. at 1 site in United States.
Trial summary
The purpose of this study is to assess the bioequivalence of subcutaneous Vidaza® and subcutaneous Luitpold Azacitidine pharmacokinetics and to assess the comparative safety of subcutaneous Vidaza® versus subcutaneous Luitpold Azacitidine. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Myelofibrosis, Chronic Myeloid Leukemia
- What is being tested?
- Luitpold Azacitidine and Vidaza®
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Nov 2010to 21 Dec 2012
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent obtained prior to initiation of any study-specific procedures.
- Patients with one of the following - myelodysplastic syndrome of the following French-American- British (FAB) subtypes: refractory anemia (RA), RA with ringed sideroblasts (if accompanied by neutropenia, or thrombocytopenia, or requiring transfusion), RA with excess of blasts (RAEB), RAEB in transformation (RAEB-T), or chronic myelomonocytic leukemia (CMMoL); myelofibrosis; chronic myeloid leukemia; or chronic lymphocytic leukemia who's physician feels should receive azacitidine.
- Male or female patients aged at least 18 years.
- Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2.
- Life expectancy > or = to 3 months.
- Adequate organ function, including the following: Hepatic - Total bilirubin < or = to 1.5 x the upper limit of normal (ULN), aspartate transaminases (AST) and alanine transaminases (ALT) < or = to 2 x ULN and Renal - Serum creatinine < or = to 1.5 x ULN.
- Female patients of child-bearing potential must have a negative pregnancy test and must be using at least one form of contraception as approved by the Investigator for 4 weeks prior to the study and 4 months after the last dose of azacitidine.
- Male patients must use a form of barrier contraception approved by the investigator during the study and for 4 months after the last dose of azacitidine.
Who cannot join
- Hypersensitivity to azacitidine or mannitol.
- Anticipated need for red blood cells (RBC) or platelet transfusion 2 days prior to or up to 2 days after treatment initiation.
- Chemotherapy (excluding previous azacitidine treatment) or radiotherapy within 4 weeks of randomization (6 weeks for nitrosoureas or mitomycin C).
- Significant electrophysical abnormalities in pre-trial EKG.
- Present history of locally advanced or metastatic malignant disease or leukemia.
- Use of recreational drugs or history of drug addiction, within the prior 6 months.
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- Known history of a positive hepatitis screen, including hepatitis B surface antigens or hepatitis C virus (HCV) antibodies.
- Known history of HIV or syphilis.
- History of clinically significant adverse events due to chemotherapy, radiotherapy or investigational agents.
- Presence of an advanced malignant hepatic tumor.
- Presence of an ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, symptomatic or poorly controlled cardiac arrhythmia, uncontrolled thrombotic or hemorrhagic disorder, or any other serious uncontrolled medical disorders.
- Presence of any significant central nervous system or psychiatric disorder(s) that would hamper the patients compliance.
- Treatment with any other investigational agent, or participation in another clinical trial within 28 days prior to study entry.
- Pregnant or breast-feeding patients or any patient with childbearing potential not using adequate contraception.
Trial locations and contacts
1 site in 1 country.
United States1 site
Luitpold Pharmaceuticals, Inc.
Norristown, Pennsylvania, 19403
About this study
Official title: Blinded Cross-over Bioequivalence Trial of Luitpold Azacitidine Versus Vidaza® in Patients With Myelodysplastic Syndrome, Myelofibrosis, Chronic Myeloid Leukemia or Chronic Lymphocytic Leukemia
The purpose of this study is to assess the bioequivalence of subcutaneous Vidaza® and subcutaneous Luitpold Azacitidine pharmacokinetics and to assess the comparative safety of subcutaneous Vidaza® versus subcutaneous Luitpold Azacitidine.
Read the detailed description
To assess the bioequivalence of Vidaza® and Luitpold Azacitidine pharmacokinetics, in terms of Maximal Concentration (Cmax), Area Under the Curve From Time Zero to the Time of Last Quantifiable Concentration (AUC 0-t), and Area Under the Curve From Time Zero Extrapolated to Infinity (AUC 0-∞), following SC administration.
To assess the comparative safety of Vidaza® versus Luitpold Azacitidine during the 2 day study period.
- Conditions
- Myelodysplastic Syndrome, Myelofibrosis, Chronic Myeloid Leukemia, Chronic Lymphocytic Leukemia
- Being tested
- Luitpold Azacitidine, Vidaza®
- Medical subject headings
- Myelodysplastic Syndromes, Primary Myelofibrosis, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Lymphocytic, Chronic, B-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Luitpold AzacitidineDrug
Also called Single use vials containing 100 mg azacitidine/vials,.The formulation contained mannitol as an excipient. Lot 090035 and Lot 120011
Subcutaneous (SC) at a dose of 75 mg/m^2 per day on days 1 and 2 of a treatment cycle
Vidaza®Drug
Also called Vidaza (azacitidine for injection), Celegene Corporation, commercially available formulation. The formulation contained mannitol as a excipient. Lot 91096A
Subcutaneous (SC) at a dose of 75 mg/m^2 per day on days 1 and 2 of a treatment cycle
| Study group | Role | What they receive |
|---|---|---|
| Luitpold Azacitidine first, then Vidaza® | Experimental | Participants first received Luitpold Azacitidine subcutaneously (SC) on Day 1. After a washout period of 24 hours, they then received Vidaza on Day 2. Receives: Drug: Luitpold Azacitidine |
| Vidaza® first, then Luitpold Azacitidine | Active comparator | Participants first received Vidaza subcutaneously (SC) on Day 1. After a washout period of 24 hours, they then received Luitpold Azacitidine on Day 2. Receives: Drug: Vidaza® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area Under the Curve (AUC) From Time Zero to the Time of Last Quantifiable Concentration (AUC 0-t)
The AUC is a measure of systemic drug exposure, which is obtained by collecting a series of blood samples and measuring the concentrations of drug in each sample; AUC 0-t is defined as AUC from time 0 to the last data point
Measured: 0.125, 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, and 10 hours after dosing on days 1 and 2.
Area Under the Curve From Time Zero Extrapolated to Infinity (AUC 0-∞)
The AUC is a measure of systemic drug exposure, which is obtained by collecting a series of blood samples and measuring the concentrations of drug in each sample. AUC 0-∞ is defined as area under the concentration vs. time curve from zero to infinity.
Measured: 0.125, 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, and 10 hours after dosing on days 1 and 2
Observed Maximal Concentration (Cmax)
Cmax refers to the highest measured drug concentration which is obtained by collecting a series of blood samples and measuring the concentrations of drug in each sample.
Measured: 0.125, 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, and 10 hours after dosing on days 1 and 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2011 (estimated)
- Study start22 Nov 2010
- Primary completionDec 2012
- Study completion21 Dec 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- American Regent, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Joseph Perno, MD,PhD, Study director, Medical Director
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01290302
- Sponsor's protocol ID
- 1AZA10001
- Status last verified
- Nov 2024
- First submitted
- 10 Jan 2011
- Last updated
- 12 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Blinded Cross-over Bioequivalence Trial of Luitpold Azacitidine Versus Vidaza® in Patients With Myelodysplastic Syndrome, Myelofibrosis, Chronic Myeloid Leukemia or Chronic Lymphocytic Leukemia
- ClinicalTrials.gov ID
- NCT01290302
- Acronym / sponsor ID
- 1AZA10001
- Conditions
- Myelodysplastic Syndrome, Myelofibrosis, Chronic Myeloid Leukemia, Chronic Lymphocytic Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- American Regent, Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Nov 2010 (actual)
- Primary completion
- Dec 2012 (actual)
- Study completion
- 21 Dec 2012 (actual)
- First posted
- 7 Feb 2011 (estimated)
- Results
- Posted 25 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the bioequivalence of subcutaneous Vidaza® and subcutaneous Luitpold Azacitidine pharmacokinetics and to assess the comparative safety of subcutaneous Vidaza® versus subcutaneous Luitpold Azacitidine.
Detailed description
To assess the bioequivalence of Vidaza® and Luitpold Azacitidine pharmacokinetics, in terms of Maximal Concentration (Cmax), Area Under the Curve From Time Zero to the Time of Last Quantifiable Concentration (AUC 0-t), and Area Under the Curve From Time Zero Extrapolated to Infinity (AUC 0-∞), following SC administration.
To assess the comparative safety of Vidaza® versus Luitpold Azacitidine during the 2 day study period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Luitpold Azacitidine first, then Vidaza® | Experimental | Drug: Luitpold AzacitidineParticipants first received Luitpold Azacitidine subcutaneously (SC) on Day 1. After a washout period of 24 hours, they then received Vidaza on Day 2. |
| Vidaza® first, then Luitpold Azacitidine | Active comparator | Drug: Vidaza®Participants first received Vidaza subcutaneously (SC) on Day 1. After a washout period of 24 hours, they then received Luitpold Azacitidine on Day 2. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Signed informed consent obtained prior to initiation of any study-specific procedures.
- Patients with one of the following - myelodysplastic syndrome of the following French-American- British (FAB) subtypes: refractory anemia (RA), RA with ringed sideroblasts (if accompanied by neutropenia, or thrombocytopenia, or requiring transfusion), RA with excess of blasts (RAEB), RAEB in transformation (RAEB-T), or chronic myelomonocytic leukemia (CMMoL); myelofibrosis; chronic myeloid leukemia; or chronic lymphocytic leukemia who's physician feels should receive azacitidine.
- Male or female patients aged at least 18 years.
- Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2.
- Life expectancy > or = to 3 months.
Key exclusion criteria (14 listed)
- Hypersensitivity to azacitidine or mannitol.
- Anticipated need for red blood cells (RBC) or platelet transfusion 2 days prior to or up to 2 days after treatment initiation.
- Chemotherapy (excluding previous azacitidine treatment) or radiotherapy within 4 weeks of randomization (6 weeks for nitrosoureas or mitomycin C).
- Significant electrophysical abnormalities in pre-trial EKG.
- Present history of locally advanced or metastatic malignant disease or leukemia.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Luitpold AzacitidineAlso: Single use vials containing 100 mg azacitidine/vials,.The formulation contained mannitol as an excipient. Lot 090035 and Lot 120011Subcutaneous (SC) at a dose of 75 mg/m^2 per day on days 1 and 2 of a treatment cycle | Drug | Luitpold Azacitidine first, then Vidaza® |
| Vidaza®Also: Vidaza (azacitidine for injection), Celegene Corporation, commercially available formulation. The formulation contained mannitol as a excipient. Lot 91096ASubcutaneous (SC) at a dose of 75 mg/m^2 per day on days 1 and 2 of a treatment cycle | Drug | Vidaza® first, then Luitpold Azacitidine |
Primary outcome
Area Under the Curve (AUC) From Time Zero to the Time of Last Quantifiable Concentration (AUC 0-t)
Time frame: 0.125, 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, and 10 hours after dosing on days 1 and 2.
The AUC is a measure of systemic drug exposure, which is obtained by collecting a series of blood samples and measuring the concentrations of drug in each sample; AUC 0-t is defined as AUC from time 0 to the last data point
Area Under the Curve From Time Zero Extrapolated to Infinity (AUC 0-∞)
Time frame: 0.125, 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, and 10 hours after dosing on days 1 and 2
The AUC is a measure of systemic drug exposure, which is obtained by collecting a series of blood samples and measuring the concentrations of drug in each sample. AUC 0-∞ is defined as area under the concentration vs. time curve from zero to infinity.
Observed Maximal Concentration (Cmax)
Time frame: 0.125, 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, and 10 hours after dosing on days 1 and 2
Cmax refers to the highest measured drug concentration which is obtained by collecting a series of blood samples and measuring the concentrations of drug in each sample.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Myelodysplastic Syndromes
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joseph Perno, MD,PhD study director
Medical Director
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Luitpold Azacitidine and Vidaza® in people with Myelodysplastic Syndrome and Myelofibrosis, run by American Regent, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Area Under the Curve (AUC) From Time Zero to the Time of Last Quantifiable Concentration (AUC 0-t) (time frame: 0.125, 0.25, 0.5, 0.75, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 8, and 10 hours after dosing on days 1 and 2.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jun 2025; AzTrials last refreshed its copy on 8 Nov 2026.