NCT03198195

Brief 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. 1.Conditioning regimen Busulfan 16 mg/kg in total, Fludarabine 160 mg/m2 in total.
  2. 2.GvHD Prophylaxis:

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
5

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Mar 2015

Longer than P75 for all trials

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 10, 2015

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 10, 2017

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

June 22, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 26, 2017

Completed
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 10, 2020

Completed
Last Updated

June 27, 2017

Status Verified

June 1, 2017

Enrollment Period

2 years

First QC Date

June 22, 2017

Last Update Submit

June 26, 2017

Conditions

Wiskott-Aldrich Syndrome

Outcome Measures

Primary Outcomes (1)

  • Rate of aGvHD

    after post transplant cyclophospamide

    3month

Interventions

cyclophosphamidePROCEDURE

on days +3，+4,using cyclophosphamide 50mg/kg

Eligibility Criteria

Age5 Months - 10 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodProbability Sample
Study Population

Patient with Wiskott-Aldrich Syndrome : 1. life-threatening bleeding 2. strong demand of parents

You may qualify if:

  • Patients diagnosed with Wiskott-Aldrich Syndrome with indication of Hematopoietic stem cell transplantation

You may not qualify if:

  • without indication of Hematopoietic stem cell transplantation

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Wiskott-Aldrich Syndrome

Interventions

Cyclophosphamide

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesLymphopeniaLeukopeniaCytopeniaHemorrhagic DisordersLeukocyte DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-LinkedPrimary Immunodeficiency DiseasesImmunologic Deficiency SyndromesImmune System Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
OTHER GOV
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD,PhD

Study Record Dates

First Submitted

June 22, 2017

First Posted

June 26, 2017

Study Start

March 10, 2015

Primary Completion

March 10, 2017

Study Completion

July 10, 2020

Last Updated

June 27, 2017

Record last verified: 2017-06

Data Sharing

IPD Sharing
Will not share