Wiskott-Aldrich SyndromeNCT03198195
Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome
An observational study of Wiskott-Aldrich Syndrome, run by Capital Research Institute of Pediatrics.
Trial summary
A protocol named as "CIP-2015" for patients with Wiskott-Aldrich Syndrome may reduce the rate of GvHD. The details of the protocal followed with: 1. Conditioning regimen Busulfan 16 mg/kg in total, Fludarabine 160 mg/m2 in total. 2. Read more about this study
- Who is it for?
- Ages 5 months to 10 years
- Conditions the study is for
- Wiskott-Aldrich Syndrome
- What is being tested?
- cyclophosphamide
- Type of treatment
- Surgery or procedure
- How many people
- 5 participants
- Where
- No sites listed yet
- Study dates
- 10 Mar 2015to 10 Jul 2020
- How long will I take part?
- 5 Years
- Phase
- Observational
- 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 5 months to 10 years
- All sexes
- No healthy volunteers
- Child
Study population: Patient with Wiskott-Aldrich Syndrome : 1. life-threatening bleeding 2. strong demand of parents
Who may be able to join
- -Patients diagnosed with Wiskott-Aldrich Syndrome with indication of Hematopoietic stem cell transplantation
Who cannot join
- without indication of Hematopoietic stem cell transplantation
About this study
Official title: Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome
A protocol named as "CIP-2015" for patients with Wiskott-Aldrich Syndrome may reduce the rate of GvHD.
The details of the protocal followed with:
- Conditioning regimen Busulfan 16 mg/kg in total, Fludarabine 160 mg/m2 in total.
- GvHD Prophylaxis:
Rabbit antihuman thymocyte globulin 7.5 mg/kg post-transplant cyclophosphamide (CY) (50 mg/kg.d on days +3 and +4) Cyclosporine or tacrolimus, mycophenolate mofetil, on days +5
Read the detailed description
Patients were enrolled in CIP-2015 Protocol at the Capital Institute of Pediatrics (Beijing). The conditioning regimen consisted of fludarabine (40 mg/m2) from days -6 to -3, and Busulfan was administered intravenously for 4 days, from days -5 to -2，using dose targeting based on therapeutic drug monitoring. Thymoglobulin (Sanofi, Cambridge, MA) 7.5 to 10 mg/kg (cumulative dose over 4 days) was administered over 4 days, from days -5 to -2. Bone marrow (BM) and PBSC were infused on day 0, followed by post-transplant CY (50 mg/kg/day, on days +3 and +4). To protect against hemorrhagic cystitis, MESNA (2-mercaptoethane sodium sulfonate) was administered at 150% of the CY dose. Post grafting immunosuppression with mycophenolate mofetil and tacrolimus commenced on day +5 and extended until days +28 and +84, respectively. Tacrolimus was tapered off by day +90 if there was no GVHD.
- Conditions
- Wiskott-Aldrich Syndrome
- Being tested
- cyclophosphamide
- Medical subject headings
- Wiskott-Aldrich Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cyclophosphamideProcedure or surgery
on days +3，+4,using cyclophosphamide 50mg/kg
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of aGvHD
after post transplant cyclophospamide
Measured: 3month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2017
- Study start10 Mar 2015
- Primary completion10 Mar 2017
- Study completion10 Jul 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wiskott-Aldrich 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 5 participants (actual)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Capital Research Institute of Pediatrics
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Yan Yue, Capital Research Institute of Pediatrics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03198195
- Sponsor's protocol ID
- CIP-2015-08
- Status last verified
- Jun 2017
- First submitted
- 22 Jun 2017
- Last updated
- 27 Jun 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome
- ClinicalTrials.gov ID
- NCT03198195
- Acronym / sponsor ID
- CIP-2015-08
- Conditions
- Wiskott-Aldrich Syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Capital Research Institute of PediatricsOther government
- Study start
- 10 Mar 2015 (actual)
- Primary completion
- 10 Mar 2017 (actual)
- Study completion
- 10 Jul 2020 (estimated)
- First posted
- 26 Jun 2017 (actual)
- 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.
A protocol named as "CIP-2015" for patients with Wiskott-Aldrich Syndrome may reduce the rate of GvHD.
The details of the protocal followed with:
- Conditioning regimen Busulfan 16 mg/kg in total, Fludarabine 160 mg/m2 in total.
- GvHD Prophylaxis:
Rabbit antihuman thymocyte globulin 7.5 mg/kg post-transplant cyclophosphamide (CY) (50 mg/kg.d on days +3 and +4) Cyclosporine or tacrolimus, mycophenolate mofetil, on days +5
Detailed description
Patients were enrolled in CIP-2015 Protocol at the Capital Institute of Pediatrics (Beijing). The conditioning regimen consisted of fludarabine (40 mg/m2) from days -6 to -3, and Busulfan was administered intravenously for 4 days, from days -5 to -2，using dose targeting based on therapeutic drug monitoring. Thymoglobulin (Sanofi, Cambridge, MA) 7.5 to 10 mg/kg (cumulative dose over 4 days) was administered over 4 days, from days -5 to -2. Bone marrow (BM) and PBSC were infused on day 0, followed by post-transplant CY (50 mg/kg/day, on days +3 and +4). To protect against hemorrhagic cystitis, MESNA (2-mercaptoethane sodium sulfonate) was administered at 150% of the CY dose. Post grafting immunosuppression with mycophenolate mofetil and tacrolimus commenced on day +5 and extended until days +28 and +84, respectively. Tacrolimus was tapered off by day +90 if there was no GVHD.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Target follow-up
- 5 Years
Who may qualify
- Ages
- 5 months to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Patient with Wiskott-Aldrich Syndrome : 1. life-threatening bleeding 2. strong demand of parents
Key inclusion criteria (1 listed)
- -Patients diagnosed with Wiskott-Aldrich Syndrome with indication of Hematopoietic stem cell transplantation
Key exclusion criteria (1 listed)
- without indication of Hematopoietic stem cell transplantation
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 |
|---|---|---|
| cyclophosphamideon days +3，+4,using cyclophosphamide 50mg/kg | Procedure or surgery | – |
Primary outcome
Rate of aGvHD
Time frame: 3month
after post transplant cyclophospamide
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 2017.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Wiskott-Aldrich Syndrome, run by Capital Research Institute of Pediatrics.
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 27 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.
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.
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?
Rate of aGvHD (time frame: 3month). 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 27 Jun 2017.