Sickle Cell DiseasePhase 2NCT03367546
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
A Phase 2 study testing Blood and Marrow Transplant in people with Sickle Cell Disease and Thalassemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor. Read more about this study
- Who is it for?
- Ages 0 to 25 years
- Conditions the study is for
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders
- What is being tested?
- Blood and Marrow Transplant
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jul 2018to 19 Dec 2022
- Phase
- Phase 2
- 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 0 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Sickle Cell Disease (SCD)
- If diagnosis of SCD must meet one or more of the following disease characteristics:
- Stroke, CNS hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral MRI or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
- Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
- Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
- Impaired neuropsychological function and abnormal cerebral MRI scan
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- Stage I or II sickle lung disease,
- Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate [GFR] 30-50% of the predicted normal value)
- Bilateral proliferative retinopathy and major visual impairment in at least one eye
- Osteonecrosis of multiple joints with documented destructive changes
- Requirement for chronic transfusions
- RBC alloimmunization
- Transfusion Dependent Alpha- or Beta-Thalassemia
- Other Non-Malignant Hematologic Disorders:
- Transfusion dependent or involve other potential life-threatening cytopenias, including but not limited to Paroxysmal Nocturnal Hemoglobinuria, Glanzmann's Thrombasthenia, Severe Congenital Neutropenia and Shwachman-Diamond Syndrome
- cALD
- Diagnosis of ALD by abnormal plasma very long chain fatty acid (VLCFA) profile or ABCD1 gene mutation
- Cerebral disease on MRI
- Absence of a Major Functional Disability (cortical blindness, loss of communication, wheelchair dependence) on the ALD Neurologic Function Scale
- Other inherited metabolic disorders:
- Any other inherited metabolic disorder for which alloHCT is indicated and for whom, in the opinion of the treating physician, the patient's best treatment option is with a haploidentical donor following non-myeloablatve conditioning.
- Age, Performance Status, Consent
- Age: 0-55 years
- Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
- Consent: voluntary written consent (adult or parental/guardian)
- Adequate Organ Function
- Renal: Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
- Hepatic: Bilirubin and ALT <3 times the upper limit of institutional normal
- Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%.
Who cannot join
- Availability of a suitable HLA-matched related donor
- Uncontrolled infection
- Pregnant or breastfeeding
- HIV positive
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Caner Center at University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant Following Reducing Intensity Conditioning for Patients With Selected High Risk Non-Malignant Disease
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
- Being tested
- Blood and Marrow Transplant
- Keywords
- SCD, cALD
- Medical subject headings
- Anemia, Sickle Cell, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood and Marrow TransplantProcedure or surgery
Reduced intensity conditioning (RIC) with rabbit ATG, fludarabine, cyclophosphamide, thiotepa and low dose (2 Gy) total body irradiation followed by T-cell replete, unmanipulated, haploidentical related donor stem cell transplant (HaploHCT) and post-transplant cyclophosphamide (PTCy)
| Study group | Role | What they receive |
|---|---|---|
| rATG, FLU/CY/TBI, & Thiotepa | Experimental | Anti-Thymocyte Globulin - Rabbit (rATG), Fludarabine (Fludara), Cyclophosphamide (Cytoxan, Neosar), Total Body Irradiation (TBI), \& Thiotepa Receives: Procedure: Blood and Marrow Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neutrophil Recovery
Incidence of neutrophil recovery by day +42
Measured: Day 42
Secondary outcomes (2)
Overall Survival (OS)
Incidence of overall survival at 1 year
Measured: 1 year
Primary Graft Failure (neutropenic and non-neutropenic)
Incidence of primary graft failure (neutropenic and non-neutropenic) by day +42
Measured: Day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2017
- Study start2 Jul 2018
- Primary completion19 Dec 2022
- Study completion19 Dec 2022
Why it stopped: Study no longer needed
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christen L Ebens, MD, MPH, Principal investigator, University of Minnesota - Pediatrics Blood and Marrow Transplant
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03367546
- Sponsor's protocol ID
- 2017LS101
- Other IDs
- MT2017-30
- Status last verified
- Jul 2024
- First submitted
- 5 Dec 2017
- Last updated
- 12 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant Following Reducing Intensity Conditioning for Patients With Selected High Risk Non-Malignant Disease
- ClinicalTrials.gov ID
- NCT03367546
- Acronym / sponsor ID
- 2017LS101
- Conditions
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
- Keywords
- SCD, cALD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study no longer needed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 2 Jul 2018 (actual)
- Primary completion
- 19 Dec 2022 (actual)
- Study completion
- 19 Dec 2022 (actual)
- First posted
- 8 Dec 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.
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rATG, FLU/CY/TBI, & Thiotepa | Experimental | Procedure: Blood and Marrow TransplantAnti-Thymocyte Globulin - Rabbit (rATG), Fludarabine (Fludara), Cyclophosphamide (Cytoxan, Neosar), Total Body Irradiation (TBI), \& Thiotepa |
Who may qualify
- Ages
- 0 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (29 listed)
- Sickle Cell Disease (SCD)
- Transfusion Dependent Alpha- or Beta-Thalassemia
- Other Non-Malignant Hematologic Disorders:
- Transfusion dependent or involve other potential life-threatening cytopenias, including but not limited to Paroxysmal Nocturnal Hemoglobinuria, Glanzmann's Thrombasthenia, Severe Congenital Neutropenia and Shwachman-Diamond Syndrome
- cALD
Key exclusion criteria (4 listed)
- Availability of a suitable HLA-matched related donor
- Uncontrolled infection
- Pregnant or breastfeeding
- HIV positive
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 |
|---|---|---|
| Blood and Marrow TransplantReduced intensity conditioning (RIC) with rabbit ATG, fludarabine, cyclophosphamide, thiotepa and low dose (2 Gy) total body irradiation followed by T-cell replete, unmanipulated, haploidentical related donor stem cell transplant (HaploHCT) and post-transplant cyclophosphamide (PTCy) | Procedure or surgery | rATG, FLU/CY/TBI, & Thiotepa |
Primary outcome
Neutrophil Recovery
Time frame: Day 42
Incidence of neutrophil recovery by day +42
2 secondary outcomes
Overall Survival (OS)
Time frame: 1 year
Primary Graft Failure (neutropenic and non-neutropenic)
Time frame: Day 42
Results and safety
The study was stopped early (reason given: Study no longer needed). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2024.
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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.
Christen L Ebens, MD, MPH principal investigator
University of Minnesota - Pediatrics Blood and Marrow Transplant
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Blood and Marrow Transplant in people with Sickle Cell Disease and Thalassemia, run by Masonic Cancer Center, University of Minnesota 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 29 inclusion and 4 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: stopped early, as of the sponsor's last update on 12 Jul 2024. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: Study no longer needed). 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?
Neutrophil Recovery (time frame: Day 42). 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 Jul 2024.