Aplastic AnemiaPhase 4NCT01995331
Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
A Phase 4 study testing cyclophosphamide,cyclosporine A in people with Aplastic Anemia, run by Xiaofan Zhu at 1 site in China.
Trial summary
Severe aplastic anemia (SAA)is characterized by the depletion of hematopoietic precursors associated with life-threatening complications. High-dose cyclophosphamide has been found to yield a complete response (CR) in adults and children with SAA.However, the optimal dosage of cyclophosphamide for patients in childhood… Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- cyclophosphamide,cyclosporine A
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- Mar 2012to Mar 2015
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Acquired Childhood Severe Aplastic Anemia (SAA)
Who cannot join
- not Childhood and Acquired Severe Aplastic Anemia
Trial locations and contacts
1 site in 1 country.
China1 site
Department of Pediatrics,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin, Tianjin Municipality, 300020
About this study
Severe aplastic anemia (SAA)is characterized by the depletion of hematopoietic precursors associated with life-threatening complications. High-dose cyclophosphamide has been found to yield a complete response (CR) in adults and children with SAA.However, the optimal dosage of cyclophosphamide for patients in childhood remains unclear. So we explore the ideal dosage of cyclophosphamide for the treatment of children with SAA.
Read the detailed description
Tisdale et al. (2000,2002) attempted to compare immunosuppression using ATG/CSA with high-dose cyclophosphamide (50 mg/kg/d for 4 consecutive days) plus CSA in a randomized trial of newly diagnosed adults with SAA. Both groups received CSA as part of the treatment regimen. However, the trial was terminated prematurely due to excessive morbidity among the patients treated in the cyclophosphamide arm. They documented that invasive fungal infections were severe among the cyclophosphamide group. Between January 2008 through May 2009, in our department, nine pediatric patients with a diagnosis of SAA were enrolled a study with lower dose of cyclophosphamide with 30mg/kg/day for 4 consecutive days and combination with CSA, this study shows promise for children with severe aplastic anemia.Now we want explore the dosage of cyclophosphamide.
- Conditions
- Aplastic Anemia
- Being tested
- cyclophosphamide,cyclosporine A
- Keywords
- aplastic anemia;, cyclophosphamide;, immunosuppressive therapy;, cyclosporine A
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cyclophosphamide,cyclosporine ADrug
Also called Drug,Mesnaum
Drug,cyclophosphamide,cyclophosphamide (30 mg/kg/day) administered intravenously (IV) over 1 hr for 4 consecutive days Drug,cyclosporine A,5mg-12mg/kg.d,CSA was administered orally 40 days after the fourth dose of cyclophosphamide and maintained for 3 years. The dose of CSA was adjusted to maintain trough drug concentration above 150 μg/L and peak drug concentration above 300 μg/L.
Drug, human granulocyte colony-stimulating factor (rhG-CSF), 5 μg/kg/day subcutaneously starting 24 hrs after the fourth dose of cyclophosphamide, and it was withdrawed when ANC was >1×109/L.
| Study group | Role | What they receive |
|---|---|---|
| moderate-dose cyclophosphomide | Other | Not described Receives: Drug: cyclophosphamide,cyclosporine A |
What is being measured
The primary outcome is the main question the study is designed to answer.
The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >30×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
Measured: 36 months
Secondary outcomes (1)
The side effect of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
To report the death rate and the death cause , the infection rate and the pathogenic bacteria, especialy the fungal infection rate.
To report other toxicity.
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2013 (estimated)
- Study startMar 2012
- Primary completionMar 2015 (estimated)
- Study completionMar 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xiaofan Zhu
- Responsible party
- Sponsor-investigator, Xiaofan Zhu, Chinese Academy of Medical Sciences
- Investigators
- Xiaifan Zhu, MD, Principal investigator, Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01995331
- Sponsor's protocol ID
- YL20102601
- Status last verified
- Nov 2013
- First submitted
- 21 Nov 2013
- Last updated
- 26 Nov 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
- ClinicalTrials.gov ID
- NCT01995331
- Acronym / sponsor ID
- YL20102601
- Conditions
- Aplastic Anemia
- Keywords
- aplastic anemia;, cyclophosphamide;, immunosuppressive therapy;, cyclosporine A
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Xiaofan Zhu
- Study start
- Mar 2012
- Primary completion
- Mar 2015 (estimated)
- Study completion
- Mar 2015 (estimated)
- First posted
- 26 Nov 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Severe aplastic anemia (SAA)is characterized by the depletion of hematopoietic precursors associated with life-threatening complications. High-dose cyclophosphamide has been found to yield a complete response (CR) in adults and children with SAA.However, the optimal dosage of cyclophosphamide for patients in childhood remains unclear. So we explore the ideal dosage of cyclophosphamide for the treatment of children with SAA.
Detailed description
Tisdale et al. (2000,2002) attempted to compare immunosuppression using ATG/CSA with high-dose cyclophosphamide (50 mg/kg/d for 4 consecutive days) plus CSA in a randomized trial of newly diagnosed adults with SAA. Both groups received CSA as part of the treatment regimen. However, the trial was terminated prematurely due to excessive morbidity among the patients treated in the cyclophosphamide arm. They documented that invasive fungal infections were severe among the cyclophosphamide group. Between January 2008 through May 2009, in our department, nine pediatric patients with a diagnosis of SAA were enrolled a study with lower dose of cyclophosphamide with 30mg/kg/day for 4 consecutive days and combination with CSA, this study shows promise for children with severe aplastic anemia.Now we want explore the dosage of cyclophosphamide.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| moderate-dose cyclophosphomide | Other | Drug: cyclophosphamide,cyclosporine A |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Acquired Childhood Severe Aplastic Anemia (SAA)
Key exclusion criteria (1 listed)
- not Childhood and Acquired Severe Aplastic Anemia
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 |
|---|---|---|
| cyclophosphamide,cyclosporine AAlso: Drug,MesnaumDrug,cyclophosphamide,cyclophosphamide (30 mg/kg/day) administered intravenously (IV) over 1 hr for 4 consecutive days Drug,cyclosporine A,5mg-12mg/kg.d,CSA was administered orally 40 days after the fourth dose of cyclophosphamide and maintained for 3 years. The dose of CSA was adjusted to maintain trough drug concentration above 150 μg/L and peak drug concentration above 300 μg/L. Drug, human granulocyte colony-stimulating factor (rhG-CSF), 5 μg/kg/day subcutaneously starting 24 hrs after the fourth dose of cyclophosphamide, and it was withdrawed when ANC was >1×109/L. | Drug | moderate-dose cyclophosphomide |
Primary outcome
The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Time frame: 36 months
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >30×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
1 secondary outcome
The side effect of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Time frame: 36 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2013.
Related trials for Aplastic Anemia
RecruitingObservational study
Cancer in Inherited Bone Marrow Failure Syndromes
Diamond Blackfan Anemia, Dyskeratosis Congenita
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 1/2
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
Bone Marrow Failure Syndromes, Aplastic Anemias
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.
Xiaifan Zhu, MD principal investigator
Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing cyclophosphamide,cyclosporine A in people with Aplastic Anemia, run by Xiaofan Zhu at 1 site in China.
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 1 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 26 Nov 2013. 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 China. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia (time frame: 36 months). 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 26 Nov 2013.