Stem Cell Transplant ComplicationsNCT04970004
Study in Adult and Pediatric Patients With HSCT-TMA
An observational study of Stem Cell Transplant Complications and Thrombotic Microangiopathies, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
This is an observational, retrospective study designed to assess outcomes in patients diagnosed with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) who were not treated with complement component (C5) inhibitor therapy. Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Stem Cell Transplant Complications, Thrombotic Microangiopathies
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Jul 2021to 1 Apr 2022
- 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 1 month and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Adult and pediatric patients who were diagnosed with TMA within 6 months after a HSCT procedure
Who may be able to join
- Body weight ≥ 5 kg at the time of HSCT-TMA diagnosis
- Documented TMA diagnosis within 6 months from the HSCT
- Evidence of renal dysfunction
- Presence of hypertension
Who cannot join
- History or presence of familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity < 5%)
- Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
- Positive direct Coombs test
- Diagnosis of disseminated intravascular coagulation
- History or presence of bone marrow/graft failure
- Diagnosis of veno-occlusive disease
- Received a complement inhibitor (eg, eculizumab) post-HSCT through 12 months post TMA diagnosis
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Retrospective Observational Study of Adult and Pediatric Patients With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)
This is an observational, retrospective study designed to assess outcomes in patients diagnosed with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) who were not treated with complement component (C5) inhibitor therapy. Data required to evaluate study outcomes will be abstracted from the medical records of all patients who meet study eligibility criteria.
- Being tested
- No intervention
- Keywords
- Hematopoietic Stem Cell Transplant, Transplant-associated TMA, Thrombotic Microangiopathy (TMA)
- Medical subject headings
- Thrombotic Microangiopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
No study intervention will be administered as part of this study.
| Study group | Role | What they receive |
|---|---|---|
| Patients Diagnosed with HSCT-TMA | Not described Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients achieving TMA Response
TMA Response is defined as platelet count ≥ 50,000/mm^3, lactate dehydrogenase < 1.5 upper limit of normal, absence of schistocytes (if present at baseline), and increase in eGFR ≥ 30% from baseline or discontinuation of dialysis (for patients on dialysis at baseline)
Measured: During the 26-week period after HSCT-TMA diagnosis
Secondary outcomes (4)
Proportion of patients achieving TMA response
Measured: During the 52-week period after HSCT-TMA diagnosis
Changes in individual components of TMA response
Platelets, lactate dehydrogenase, eGFR, chronic kidney disease stage, dialysis status
Measured: From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis
Overall survival
Measured: At 26 weeks and 52 weeks after HSCT-TMA diagnosis
Nonrelapse mortality
Death due to any cause during the study, with the exception of death due to underlying disease progression or relapse
Measured: At 26 weeks and 52 weeks after HSCT-TMA diagnosis
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2021
- Study start12 Jul 2021
- Primary completion1 Apr 2022
- Study completion1 Apr 2022
Why it stopped: Sponsor decision to not proceed.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stem Cell Transplant ComplicationsStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The 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
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04970004
- Sponsor's protocol ID
- ALX-TMA-501
- Status last verified
- May 2022
- First submitted
- 16 Jul 2021
- Last updated
- 12 May 2022
- Results posted
- No
- Data monitoring committee
- No
- 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 12 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Observational Study of Adult and Pediatric Patients With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)
- ClinicalTrials.gov ID
- NCT04970004
- Acronym / sponsor ID
- ALX-TMA-501
- Keywords
- Hematopoietic Stem Cell Transplant, Transplant-associated TMA, Thrombotic Microangiopathy (TMA)
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: Sponsor decision to not proceed.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Jul 2021 (actual)
- Primary completion
- 1 Apr 2022 (actual)
- Study completion
- 1 Apr 2022 (actual)
- First posted
- 21 Jul 2021 (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.
This is an observational, retrospective study designed to assess outcomes in patients diagnosed with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) who were not treated with complement component (C5) inhibitor therapy. Data required to evaluate study outcomes will be abstracted from the medical records of all patients who meet study eligibility criteria.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients Diagnosed with HSCT-TMA | – | Other: No intervention |
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Adult and pediatric patients who were diagnosed with TMA within 6 months after a HSCT procedure
Key inclusion criteria (4 listed)
- Body weight ≥ 5 kg at the time of HSCT-TMA diagnosis
- Documented TMA diagnosis within 6 months from the HSCT
- Evidence of renal dysfunction
- Presence of hypertension
Key exclusion criteria (7 listed)
- History or presence of familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity < 5%)
- Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
- Positive direct Coombs test
- Diagnosis of disseminated intravascular coagulation
- History or presence of bone marrow/graft failure
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 |
|---|---|---|
| No interventionNo study intervention will be administered as part of this study. | Other | Patients Diagnosed with HSCT-TMA |
Primary outcome
Proportion of patients achieving TMA Response
Time frame: During the 26-week period after HSCT-TMA diagnosis
TMA Response is defined as platelet count ≥ 50,000/mm^3, lactate dehydrogenase < 1.5 upper limit of normal, absence of schistocytes (if present at baseline), and increase in eGFR ≥ 30% from baseline or discontinuation of dialysis (for patients on dialysis at baseline)
4 secondary outcomes
Proportion of patients achieving TMA response
Time frame: During the 52-week period after HSCT-TMA diagnosis
Changes in individual components of TMA response
Time frame: From baseline to 26 weeks and to 52 weeks after HSCT-TMA diagnosis
Overall survival
Time frame: At 26 weeks and 52 weeks after HSCT-TMA diagnosis
Nonrelapse mortality
Time frame: At 26 weeks and 52 weeks after HSCT-TMA diagnosis
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2022.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Stem Cell Transplant Complications and Thrombotic Microangiopathies, run by Alexion Pharmaceuticals, 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 4 inclusion and 7 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: withdrawn, as of the sponsor's last update on 12 May 2022. 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.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Proportion of patients achieving TMA Response (time frame: During the 26-week period after HSCT-TMA diagnosis). 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 May 2022.