Wiskott-Aldrich SyndromePhase 1NCT00160355
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
A Phase 1 study testing Hematopoietic stem cell transplantation, Miltenyi CliniMACS selection system and Fludarabine, Melphalan, Thiotepa in people with Wiskott-Aldrich Syndrome, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
Wiskott - Aldrich syndrome (WAS) is a rare disorder curable only through allogeneic hematopoietic stem cell transplantation. A mismatched family member is an option when no human leukocyte antigen (HLA-immune system type) matched related or matched unrelated donor is available. Read more about this study
- Who is it for?
- Ages Up to 18 years, men only
- Conditions the study is for
- Wiskott-Aldrich Syndrome
- What is being tested?
- Hematopoietic stem cell transplantation, Miltenyi CliniMACS selection system and Fludarabine, Melphalan, Thiotepa
- Type of treatment
- Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- May 2005to Feb 2009
- Phase
- Phase 1
- 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 Up to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Genotypical diagnosis of Wiskott-Aldrich Syndrome.
- Less than 18 years of age at time of transplant.
- Must meet two of the eight following clinical criteria:
- Eczema that is refractory to standard therapy.
- Thrombocytopenia as defined by a platelet count < 50,000/mm3.
- Significant risk for or presence of opportunistic infection.
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- Autoimmune disease.
- Malignancy or pre-malignant condition.
- Family history as defined as a family member with WAS who died before 10 years of age.
- Does not have a suitable, available 6/6 HLA-matched sibling donor available for donation.
- Does not have a suitable, available 10/10 HLA-allele matched unrelated donor identified through the National Marrow Donor Program (NMDP).
Who cannot join
- If any of the following clinical indicators are met within 45 days prior to transplant, the research participant will not be eligible for the study:
- Symptomatic cardiac disease or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction < 30%).
- Creatinine clearance or Tc 99 less than or equal 40ml/min/1.73 m2.
- SGPT greater than or equal 500 U/L.
- Karnofsky or Lansky Performance Score of < 50.
- Pulmonary function tests: FVC < 50% of predicted value if age appropriate to perform the testing adequately or an O2 saturation less than or equal to 92% on room air at rest.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study
Wiskott - Aldrich syndrome (WAS) is a rare disorder curable only through allogeneic hematopoietic stem cell transplantation. A mismatched family member is an option when no human leukocyte antigen (HLA-immune system type) matched related or matched unrelated donor is available.
This study will evaluate a novel therapeutic strategy for patients with WAS who undergo haploidentical transplantation using a parental donor. To reduce the risk of transplant-related toxicities, participants will receive a reduced intensity chemotherapy and antibody regimen (conditioning treatment). Participants will then receive an infusion of donor stem cells depleted of certain white blood cells called T- and B-lymphocytes. The stem cell depletion processing will be done through the use of the investigational CliniMACS device. A certain number of T-lymphocytes will be added back to the processed stem cell graft prior to infusion into the recipient.
The primary objective of this study is to determine the safety of haploidentical transplantation in WAS patients using this specified conditioning regimen and engineered graft. Safety will be defined in terms of engraftment (meaning how well the graft grows and functions after infusion) and regimen-related toxicity within the first 100 days after transplant.
Read the detailed description
Secondary Objectives in this trial include the following:
- To estimate the survival of study recipients at one year after infusion of the T- and B-lymphocyte depleted stem cell graft.
- To assess if the study treatment enables the recipient to generate normal donor-derived B-cell numbers and endogenous IgM, IgG, and IgA production, resulting in a reduction/elimination of the need for intravenous immunoglobulin infusions.
- To determine if the study treatment results in the ability of the research participant to generate normal donor-derived T cell response and natural killer (NK) cell numbers and function.
- To describe the incidence of Epstein-Barr virus-lymphoproliferative disease (EBV-LPD) in these transplant recipients.
- Conditions
- Wiskott-Aldrich Syndrome
- Being tested
- Hematopoietic stem cell transplantation, Miltenyi CliniMACS selection system, Fludarabine, Melphalan, Thiotepa
- Keywords
- Wiskott-Aldrich Syndrome, Immunodeficiency, Haploidentical transplantation
- Medical subject headings
- Wiskott-Aldrich Syndrome, Immunologic Deficiency Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoietic stem cell transplantationProcedure or surgery
Also called Haploidentical stem cell transplant, Allogeneic stem cell transplant, Mismatched family member donor transplant, T and B cell depletion
To determine the safety in regards to engraftment and toxicity within 100 days of infusing a haploidentical T- and B-cell depleted hematopoietic stem cell graft into patients with Wiskott-Aldrich syndrome who have received a reduced intensity conditioning regimen.
Miltenyi CliniMACS selection systemDevice
This system depletes the hematopoietic stem cell graft of T and B lymphocytes.
Fludarabine, Melphalan, ThiotepaDrug
Participants will receive a reduced intensity conditioning regimen consisting of Fludarabine, Melphalan, Thiotepa, and OKT3 prior to receipt of the haploidentical stem cell graft. Rituximab will be given in an effort to prevent PTLPD. In addition to T-cell depletion of the donor product, cyclosporine will be given for GVHD prophylaxis.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Other | Not described Receives: Procedure: Hematopoietic stem cell transplantation; Device: Miltenyi CliniMACS selection system; Drug: Fludarabine, Melphalan, Thiotepa |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine safety in regards to engraftment and toxicity within 100 days post-haploidentical T- and B-cell depleted hematopoietic stem cell transplantation for patients with Wiskott-Aldrich syndrome who received a reduced intensity conditioning
Measured: March 2010
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2005 (estimated)
- Study startMay 2005
- Primary completionJul 2008
- Study completionFeb 2009
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.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kimberly Kasow, DO, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00160355
- Sponsor's protocol ID
- WASHAP
- Status last verified
- Feb 2009
- First submitted
- 8 Sep 2005
- Last updated
- 26 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study
- ClinicalTrials.gov ID
- NCT00160355
- Acronym / sponsor ID
- WASHAP
- Conditions
- Wiskott-Aldrich Syndrome
- Keywords
- Wiskott-Aldrich Syndrome, Immunodeficiency, Haploidentical transplantation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- May 2005
- Primary completion
- Jul 2008 (actual)
- Study completion
- Feb 2009 (actual)
- First posted
- 12 Sep 2005 (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.
Wiskott - Aldrich syndrome (WAS) is a rare disorder curable only through allogeneic hematopoietic stem cell transplantation. A mismatched family member is an option when no human leukocyte antigen (HLA-immune system type) matched related or matched unrelated donor is available.
This study will evaluate a novel therapeutic strategy for patients with WAS who undergo haploidentical transplantation using a parental donor. To reduce the risk of transplant-related toxicities, participants will receive a reduced intensity chemotherapy and antibody regimen (conditioning treatment). Participants will then receive an infusion of donor stem cells depleted of certain white blood cells called T- and B-lymphocytes. The stem cell depletion processing will be done through the use of the investigational CliniMACS device. A certain number of T-lymphocytes will be added back to the processed stem cell graft prior to infusion into the recipient.
The primary objective of this study is to determine the safety of haploidentical transplantation in WAS patients using this specified conditioning regimen and engineered graft. Safety will be defined in terms of engraftment (meaning how well the graft grows and functions after infusion) and regimen-related toxicity within the first 100 days after transplant.
Detailed description
Secondary Objectives in this trial include the following:
- To estimate the survival of study recipients at one year after infusion of the T- and B-lymphocyte depleted stem cell graft.
- To assess if the study treatment enables the recipient to generate normal donor-derived B-cell numbers and endogenous IgM, IgG, and IgA production, resulting in a reduction/elimination of the need for intravenous immunoglobulin infusions.
- To determine if the study treatment results in the ability of the research participant to generate normal donor-derived T cell response and natural killer (NK) cell numbers and function.
- To describe the incidence of Epstein-Barr virus-lymphoproliferative disease (EBV-LPD) in these transplant recipients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Other | Procedure: Hematopoietic stem cell transplantation, Device: Miltenyi CliniMACS selection system, Drug: Fludarabine, Melphalan, Thiotepa |
Who may qualify
- Ages
- Up to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Genotypical diagnosis of Wiskott-Aldrich Syndrome.
- Less than 18 years of age at time of transplant.
- Eczema that is refractory to standard therapy.
- Thrombocytopenia as defined by a platelet count < 50,000/mm3.
- Significant risk for or presence of opportunistic infection.
Key exclusion criteria (6 listed)
- If any of the following clinical indicators are met within 45 days prior to transplant, the research participant will not be eligible for the study:
- Symptomatic cardiac disease or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction < 30%).
- Creatinine clearance or Tc 99 less than or equal 40ml/min/1.73 m2.
- SGPT greater than or equal 500 U/L.
- Karnofsky or Lansky Performance Score of < 50.
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 |
|---|---|---|
| Hematopoietic stem cell transplantationAlso: Haploidentical stem cell transplant, Allogeneic stem cell transplant, Mismatched family member donor transplant, T and B cell depletionTo determine the safety in regards to engraftment and toxicity within 100 days of infusing a haploidentical T- and B-cell depleted hematopoietic stem cell graft into patients with Wiskott-Aldrich syndrome who have received a reduced intensity conditioning regimen. | Procedure or surgery | 1 |
| Miltenyi CliniMACS selection systemThis system depletes the hematopoietic stem cell graft of T and B lymphocytes. | Device | 1 |
| Fludarabine, Melphalan, ThiotepaParticipants will receive a reduced intensity conditioning regimen consisting of Fludarabine, Melphalan, Thiotepa, and OKT3 prior to receipt of the haploidentical stem cell graft. Rituximab will be given in an effort to prevent PTLPD. In addition to T-cell depletion of the donor product, cyclosporine will be given for GVHD prophylaxis. | Drug | 1 |
Primary outcome
To determine safety in regards to engraftment and toxicity within 100 days post-haploidentical T- and B-cell depleted hematopoietic stem cell transplantation for patients with Wiskott-Aldrich syndrome who received a reduced intensity conditioning
Time frame: March 2010
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Apr 2017.
- St. Jude Children's Research HospitalLink provided by the sponsor.
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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.
Kimberly Kasow, DO principal investigator
St. Jude Children's Research Hospital
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 Hematopoietic stem cell transplantation, Miltenyi CliniMACS selection system and Fludarabine, Melphalan, Thiotepa in people with Wiskott-Aldrich Syndrome, run by St. Jude Children's Research Hospital 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 11 inclusion and 6 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 26 Apr 2017. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To determine safety in regards to engraftment and toxicity within 100 days post-haploidentical T- and B-cell depleted hematopoietic stem cell transplantation for patients with Wiskott-Aldrich syndrome who received a reduced intensity conditioning (time frame: March 2010). 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 Apr 2017.