Bone Marrow Failure SyndromePhase 1/2NCT03513328
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
A Phase 1/2 study testing Thiotepa--single daily dose and Thiotepa--escalated dose in people with Bone Marrow Failure Syndrome and Thalassemia, run by University of Florida at 1 site in United States.
Trial summary
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem… Read more about this study
- Who is it for?
- Ages 3 months to 39 years
- Conditions the study is for
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease
- What is being tested?
- Thiotepa--single daily dose and Thiotepa--escalated dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jun 2018to 19 Feb 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 months to 39 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnoses:
- Hemoglobinopathies (e.g. thalassemia or sickle cell disease),
- Cytopenias (e.g.Diamond-Blackfan anemia, congenital or acquired neutropenia, congenital or acquired thrombocytopenia, congenital or acquired anemia, and others, regardless clonality),
- Hemophagocytic lymphohistiocytosis,
- Primary immunodeficiencies (e.g. Wiscott Aldrich Syndrome, chronic granulomatous disease, common variable immune deficiency, X-linked lymphoproliferative disease, NK+ severe combined immune deficiencies),
- Metabolic disorders (Hurler's syndrome, mannosidosis, adrenal leuko-dystrophy)
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- Other non-malignant disorders for which there is published evidence that HSCT (hematopoietic stem cell transplant) is a curative therapy.
- Donor Requirements
- Related or unrelated donor who is suitable and willing to donate bone marrow or peripheral blood stem cells. HLA typing should be done by high-resolution typing at A, B, C, DrB1 and DQ loci and the donor should be at a minimum ≥8/10 match (with one antigen/allele mismatch allowed at A, B, or C-loci and other at DQ loci).
- Cord blood units must be matched at a minimum of 6/8 antigens/alleles at A, B, C and DrB1 loci. High resolution typing at all loci is required. The minimum TNC dose pre-cryopreservation must be ≥3.7 x10^7/kg of recipient's weight, if a single cord blood unit is used, or at least 2x10^7/kg per unit, if two cord blood units are used. The mismatches cannot be at the same loci (e.g. double A mismatch).
- Haploidentical related stem cell donor who is suitable and willing to donate peripheral blood stem cells. T-cell depletion is required if haploidentical donors are used. Pharmacologic GVHD prophylaxis will not be used for T-cell depleted transplant recipients.
- Adequate organ function defined as:
- Cardiac: ejection fraction ≥55% or shortening fraction ≥30%
- creatinine clearance ≥70 ml/min/1.73m2
- Pulse oximetry >95% on room air or FEV1/DLCO >60%
- LFTs < 3 x ULN, Total bilirubin <3 mg/dl (unless due to non-hepatic cause (e.g. Gilbert's syndrome or hemolysis)
- Lansky/Karnofsky score ≥60%
- Written informed consent obtained from the subject or parental/guardian permission ± child's assent per institutional guidelines
- Women of childbearing potential (WOCBP) must be using an adequate method of contraception to avoid pregnancy for at least 1 month after completion of conditioning. WOCBP include any woman who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or who is not post-menopausal. Post-menopause is defined as:
- Amenorrhea that has lasted for ≥ 12 consecutive months without another cause, or
- For women with irregular menstrual periods who are taking hormone replacement therapy (HRT), a documented serum follicle-stimulating hormone (FSH) level of greater than 35 mIU/mL.
- Males with female partners of childbearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, or vasectomy) for at least one month after completion of conditioning.
Who cannot join
- Diagnoses that do not require myeloablative transplant for cure (e.g. NK- SCID patients), unless the subject previously did not engraft with non-myeloablative or reduced intensity conditioning transplant.
- Known or suspected sensitivity to chemotherapy or radiation (e.g Fanconi's anemia, Dyskeratosis congenita, Ligase IV deficiency, etc).
- Subjects with fast-progressing neurodegenerative disorders (e.g. Krabbe disease or adrenal leukodystrophy with Loes score of ≥10)
- Cytopenias with increased blasts (>5%)
- Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer (by complement-dependent cytotoxicity or flow cytometric testing) or the presence of anti-donor HLA antibody to the high expression loci HLA-A, B, C, DRB1 with mean fluorescence intensity (MFI)>3000 by solid phase
- Prior allogeneic stem cell transplant, except for patients with immune deficiencies who underwent previous non-myeloablative or reduced intensity transplants.
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- Haploidentical donor using in vivo T-cell depletion (e.g. post-transplant cyclophosphamide).
- Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment.
- Seropositive for HIV
- Active Hepatitis B or C determined by a detectable viral load of HBV or HCV by PCR
- Bridging fibrosis or liver cirrhosis
- Females or males of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for at least 1 months after the end of conditioning
- Females who are pregnant or breastfeeding
- History of any other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of protocol therapy or that might affect the interpretation of the results of the study or that puts the subject at high risk for treatment complications, in the opinion of the treating physician.
- Subjects demonstrating an inability to understand the study and comply with the study and/or follow-up procedures
Trial locations and contacts
1 site in 1 country.
United States1 site
UF Health Shands Children's Hospital
Gainesville, Florida, 32608
About this study
Official title: PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.
Read the detailed description
Hematopoietic stem cell transplantation is the only curative choice for a number of inherited bone marrow failure syndromes, hemoglobinopathies, metabolic disorders and primary immune deficiencies. While survival of these patients is typically better than survival of patients with malignancies, toxicities of conditioning regimens and failure of engraftment remain challenges. Most children with non-malignant disorders present with normocellular or even hypercellular bone marrow, posing a barrier to engraftment and requiring intensive conditioning. Commonly used backbone of busulfan and fludarabine, although well tolerated, results in variable engraftment, in particular with mismatched unrelated donors and cord blood recipients. In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) in order to determine the minimum effective dose required for reliable engraftment. Subjects are stratified in groups A and B based the risk of graft failure.
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic
- Being tested
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Keywords
- human leukocyte antigen (HLA)
- Medical subject headings
- Bone Marrow Failure Disorders, Thalassemia, Anemia, Sickle Cell, Anemia, Diamond-Blackfan, Anemia, Hemolytic, Lymphohistiocytosis, Hemophagocytic, Wiskott-Aldrich Syndrome, Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thiotepa--single daily doseDrug
Conditioning regimen for hematopoietic stem-cell transplant. Single daily IV dose of Thiotepa at 5 mg/kg.
Thiotepa--escalated doseDrug
Twice daily IV dose of Thiotepa at 5 mg/kg, twelve hours apart, 10mg/kg total.
| Study group | Role | What they receive |
|---|---|---|
| Group A--Thiotepa single dose | Experimental | Fully matched 10/10 subjects with lower risk of graft failure. Subjects will undergo 10/10 HLA (human leukocyte antigen) matched bone marrow and peripheral blood transplant. Subjects receive combination of single daily dose thiotepa (5 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG). Receives: Drug: Thiotepa--single daily dose |
| Group A--Thiotepa escalated dose | Experimental | Fully matched 10/10 subjects with lower risk of graft failure. Subjects will undergo 10/10 HLA (human leukocyte antigen) matched bone marrow and peripheral blood transplant. Subjects receive combination of escalated dose of thiotepa (10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG). Receives: Drug: Thiotepa--escalated dose |
| Group B--Thiotepa single dose | Active comparator | Subjects with higher risk of graft failure. Subjects will undergo transplant with <10/10 bone marrow or peripheral blood match, or receiving cord blood transplant. Subjects receive combination of single daily dose thiotepa (5 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG). Receives: Drug: Thiotepa--single daily dose |
| Group B--Thiotepa escalated dose | Active comparator | Subjects with higher risk of graft failure. Subjects will undergo transplant with <10/10 bone marrow or peripheral blood match, or receiving cord blood transplant. Subjects receive combination of escalated dose of thiotepa (10 mg/kg)added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG). Receives: Drug: Thiotepa--escalated dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of Minimum Effective Dose (MED) of Thiotepa
Assess the MED of thiotepa in combination with reduced-dose busulfan, fludarabine and rATG required to achieve engraftment in >90% subjects undergoing hematopoietic stem cell transplantation for non-malignant disorders.
Measured: Day 42
Secondary outcomes (7)
Percentage of Subjects With Graft Rejection/Failure.
Percentage of all subjects who initiated conditioning regimen and have sustained engraftment failure.
Measured: Day 42; Day 365
Percentage of Subjects Without Disease Recurrence Who Are Alive at 24 Months Post Transplant
Percentage of subjects who initiated conditioning regimen and are without evidence of underlying disease (DFS).
Measured: Month 24
Percentage of Subjects Alive at 24 Months Post Transplant (OS)
Percentage of subjects who initiated conditioning regimen and are alive at 24 months post transplant (OS).
Measured: Month 24
Evaluation of Transplant-related Mortality
Percentage of subjects who initiated conditioning regimen and who died due to a cause unrelated to the underlying disease.
Measured: Month 12
Number of Participants With Grade 2-4 Acute Graft-versus-host Disease (GVDH)
Graft-versus host disease symptoms measured using Modified Glucksberg Staging Criteria. (Scale 0-4; with 4 being most severe)
Measured: Month 12
Percentage of Participants With Chronic Graft-versus-host Disease (cGVHD)
Measures the frequency of chronic graft-vs-host disease in Group A participants
Measured: Month 24
Percentage of Participants With Transplant-related Complications
Complications gathered via CIBMTR (Center for International Blood \& Marrow Transplant Research) post-transplant form was tabulated and described by treatment received.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2018
- Study start15 Jun 2018
- Primary completion17 Feb 2022
- Study completion19 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Bone Marrow Failure SyndromeStudies of the same condition, open and under way.
- More trials run by University of FloridaThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- The study builds in rules for escalation of thiotepa dose based on graft failure by day 42. Patients are stratified in two groups A (lower risk of graft failure) and B (higher risk of graft failure). Patients undergoing 10/10 HLA matched bone marrow and peripheral blood transplants are assigned to Group A, and patients receiving \<10/10 matched bone marrow or peripheral blood, or receiving cord blood, even if fully matched, are assigned to Group B. If criteria for thiotepa dose escalation are met first in Group A, which has a lower risk of graft failure, thiotepa dose will be escalated for all subjects (Groups A and B). If criteria for thiotepa dose escalation are met first in Group B, dose will be escalated only in Group B and Group A will continue enrolling patients at a lower dose level.
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Live Like Bella Pediatric Cancer Research
- Responsible party
- Sponsor
- Investigators
- Biljana Horn, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03513328
- Sponsor's protocol ID
- IRB201800798
- Other IDs
- PEDS024, OCR17838, AWD05935
- Status last verified
- Sep 2023
- First submitted
- 19 Apr 2018
- Last updated
- 15 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: There is no plan at this time.
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders
- ClinicalTrials.gov ID
- NCT03513328
- Acronym / sponsor ID
- IRB201800798
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic
- Keywords
- human leukocyte antigen (HLA)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Live Like Bella Pediatric Cancer Research
- Study start
- 15 Jun 2018 (actual)
- Primary completion
- 17 Feb 2022 (actual)
- Study completion
- 19 Feb 2023 (actual)
- First posted
- 1 May 2018 (actual)
- Results
- Posted 15 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.
Detailed description
Hematopoietic stem cell transplantation is the only curative choice for a number of inherited bone marrow failure syndromes, hemoglobinopathies, metabolic disorders and primary immune deficiencies. While survival of these patients is typically better than survival of patients with malignancies, toxicities of conditioning regimens and failure of engraftment remain challenges. Most children with non-malignant disorders present with normocellular or even hypercellular bone marrow, posing a barrier to engraftment and requiring intensive conditioning. Commonly used backbone of busulfan and fludarabine, although well tolerated, results in variable engraftment, in particular with mismatched unrelated donors and cord blood recipients. In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) in order to determine the minimum effective dose required for reliable engraftment. Subjects are stratified in groups A and B based the risk of graft failure.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- The study builds in rules for escalation of thiotepa dose based on graft failure by day 42. Patients are stratified in two groups A (lower risk of graft failure) and B (higher risk of graft failure). Patients undergoing 10/10 HLA matched bone marrow and peripheral blood transplants are assigned to Group A, and patients receiving <10/10 matched bone marrow or peripheral blood, or receiving cord blood, even if fully matched, are assigned to Group B. If criteria for thiotepa dose escalation are met first in Group A, which has a lower risk of graft failure, thiotepa dose will be escalated for all subjects (Groups A and B). If criteria for thiotepa dose escalation are met first in Group B, dose will be escalated only in Group B and Group A will continue enrolling patients at a lower dose level.
| Arm | Type | Receives |
|---|---|---|
| Group A--Thiotepa single dose | Experimental | Drug: Thiotepa--single daily doseFully matched 10/10 subjects with lower risk of graft failure. Subjects will undergo 10/10 HLA (human leukocyte antigen) matched bone marrow and peripheral blood transplant. Subjects receive combination of single daily dose thiotepa (5 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG). |
| Group A--Thiotepa escalated dose | Experimental | Drug: Thiotepa--escalated doseFully matched 10/10 subjects with lower risk of graft failure. Subjects will undergo 10/10 HLA (human leukocyte antigen) matched bone marrow and peripheral blood transplant. Subjects receive combination of escalated dose of thiotepa (10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG). |
| Group B--Thiotepa single dose | Active comparator | Drug: Thiotepa--single daily doseSubjects with higher risk of graft failure. Subjects will undergo transplant with <10/10 bone marrow or peripheral blood match, or receiving cord blood transplant. Subjects receive combination of single daily dose thiotepa (5 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG). |
| Group B--Thiotepa escalated dose | Active comparator | Drug: Thiotepa--escalated doseSubjects with higher risk of graft failure. Subjects will undergo transplant with <10/10 bone marrow or peripheral blood match, or receiving cord blood transplant. Subjects receive combination of escalated dose of thiotepa (10 mg/kg)added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG). |
Who may qualify
- Ages
- 3 months to 39 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (22 listed)
- Diagnoses:
- Donor Requirements
- Adequate organ function defined as:
- Lansky/Karnofsky score ≥60%
- Written informed consent obtained from the subject or parental/guardian permission ± child's assent per institutional guidelines
Key exclusion criteria (15 listed)
- Diagnoses that do not require myeloablative transplant for cure (e.g. NK- SCID patients), unless the subject previously did not engraft with non-myeloablative or reduced intensity conditioning transplant.
- Known or suspected sensitivity to chemotherapy or radiation (e.g Fanconi's anemia, Dyskeratosis congenita, Ligase IV deficiency, etc).
- Subjects with fast-progressing neurodegenerative disorders (e.g. Krabbe disease or adrenal leukodystrophy with Loes score of ≥10)
- Cytopenias with increased blasts (>5%)
- Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer (by complement-dependent cytotoxicity or flow cytometric testing) or the presence of anti-donor HLA antibody to the high expression loci HLA-A, B, C, DRB1 with mean fluorescence intensity (MFI)>3000 by solid phase
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 |
|---|---|---|
| Thiotepa--single daily doseConditioning regimen for hematopoietic stem-cell transplant. Single daily IV dose of Thiotepa at 5 mg/kg. | Drug | Group A--Thiotepa single dose, Group B--Thiotepa single dose |
| Thiotepa--escalated doseTwice daily IV dose of Thiotepa at 5 mg/kg, twelve hours apart, 10mg/kg total. | Drug | Group A--Thiotepa escalated dose, Group B--Thiotepa escalated dose |
Primary outcome
Assessment of Minimum Effective Dose (MED) of Thiotepa
Time frame: Day 42
Assess the MED of thiotepa in combination with reduced-dose busulfan, fludarabine and rATG required to achieve engraftment in >90% subjects undergoing hematopoietic stem cell transplantation for non-malignant disorders.
7 secondary outcomes
Percentage of Subjects With Graft Rejection/Failure.
Time frame: Day 42; Day 365
Percentage of Subjects Without Disease Recurrence Who Are Alive at 24 Months Post Transplant
Time frame: Month 24
Percentage of Subjects Alive at 24 Months Post Transplant (OS)
Time frame: Month 24
Evaluation of Transplant-related Mortality
Time frame: Month 12
Number of Participants With Grade 2-4 Acute Graft-versus-host Disease (GVDH)
Time frame: Month 12
Percentage of Participants With Chronic Graft-versus-host Disease (cGVHD)
Time frame: Month 24
Percentage of Participants With Transplant-related Complications
Time frame: 24 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. There is no plan at this time.
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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.
Biljana Horn, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Thiotepa--single daily dose and Thiotepa--escalated dose in people with Bone Marrow Failure Syndrome and Thalassemia, run by University of Florida 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 22 inclusion and 15 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 15 Sep 2023. 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 combined 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.
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?
Yes. Results are posted in the registry (first posted 15 Sep 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Assessment of Minimum Effective Dose (MED) of Thiotepa (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 15 Sep 2023.