Stem Cell TransplantationPhase 2NCT05293509
Pharmacologic Pretransplant Immunosuppression (PTIS) + Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation in Inherited Hematologic Disorders
A Phase 2 study testing Fludarabine, Dexamethasone and Cyclophosphamide in people with Stem Cell Transplantation, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
To assess the outcomes of NRM when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC) and an allogeneic stem cell transplant (allo-SCT) and post-transplant graft-versus-host disease prophylaxis based on post-transplant cyclophosphamide… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Stem Cell Transplantation
- What is being tested?
- Fludarabine, Dexamethasone and Cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Mar 2022to 18 Sep 2023
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The first six patients will be ages >12 years old and <35 years old. Thereafter in a second stage, patients ages 2 to 50 years old will be included.
- Patient with a matched related donor or who has a related haploidentical donor identified.
- Performance score of at least 70 by Karnofsky or 0 to 1 by ECOG (age > 12 years), or Zubrod or Lansky Play Performance Scale of at least 70 (age <12 years).
- Adequate major organ system function as demonstrated by:
- Serum creatinine clearance equal or more than 50 ml/min (calculated with Cockroft-Gault formula).
- Bilirubin equal or less than 1.5 mg/dl except for Gilbert's disease. ALT and/or AST equal or less than 3x institutional ULN. Conjugated (direct) bilirubin less than 2x upper limit of normal.
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- Left ventricular ejection fraction equal or greater than 50%.
- Diffusing capacity for carbon monoxide (DLCO) equal or greater than 50%
- Predicted, corrected for hemoglobin. For children < 7 years of age who are unable to perform PFT, oxygen saturation > 92% on room air by pulse oximetry.
- Patient or the patient's legal representative, parent(s) or guardian should be able to provide written informed consent. Assent of a minor if participant's age is at least seven and less than eighteen years.
- Sexually active males and females of childbearing potential must agree to use a form of contraception considered effective and medically acceptable by the Investigator.
Who cannot join
- HIV positive; active hepatitis B or C.
- Uncontrolled infections.
- Liver cirrhosis. However mild fibrosis will be allowed i.e. fine reticulin or Grade 1, with bridging fibrosis.
- CNS involvement within 3 months.
- Positive pregnancy test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.
- Inability to comply with medical therapy or follow-up.
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- Will restrict eligibility to a maximum BMI of ≤40
- Patient with a known history of allergic reactions to any constituents of the cell product, including a known history of allergic reactions to DMSO.
- Prior allo-SCT
- Other active malignancy/cancer diagnosis in remission for at least 2yrs. Malignancies not being excluded are as follows: Ductal carcinoma in situ (DCIS), Basal cell carcinoma (BCC), Cervical intraepithelial neoplasia (CIN)
Trial locations and contacts
1 site in 1 country.
About this study
To assess the outcomes of NRM when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC) and an allogeneic stem cell transplant (allo-SCT) and post-transplant graft-versus-host disease prophylaxis based on post-transplant cyclophosphamide (PT-Cy) in patients with inherited blood disorders.
Read the detailed description
Objectives
Primary:
To estimate the 100-day non-relapse mortality (NRM) rate when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC) and an allogeneic stem cell transplant (allo-SCT) and post-transplant graft-versus-host disease prophylaxis based on post-transplant cyclophosphamide (PT-Cy) in patients with inherited blood disorders.
Secondary outcomes include the following:
- Immune reconstitution ii. Infectious complications iii. Quality of life (QOL) at 3 months,100 days, and 1 year post-transplant iv. OS, EFS, and GRFS v. Incidence of aGVHD at day 100. vi. Rate of chronic GVHD within the first-year post transplantation. vii. Rate of Graft failure
- Conditions
- Stem Cell Transplantation
- Being tested
- Fludarabine, Dexamethasone, Cyclophosphamide, Bortezomib, Rituximab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
Also called Fludara™
40 mg/m2/day i.v.- by vein
DexamethasoneDrug
25 mg/m2/day i.v.-by vein
CyclophosphamideDrug
Also called Cytoxan™
100 mg/m2 IV-by vein
BortezomibDrug
Also called Velcade®
Four doses of bortezomib at a dose of 1.3 mg/m2 -injection under the skin
RituximabDrug
Also called Rituxan®
Four doses of rituximab at a dose of 375 mg/m2- by vein
BusulfanDrug
Also called Busulfex™
110 mg/m2 i.v-by vein
Cyclophosphamide (Cy)Drug
Also called Cytoxan™
by vein
Tacrolimus (or cyclosporine)Drug
by vein
Mycophenolate mofetil (MMF)Drug
given by PO
Rabbit ATGDrug
by vein
| Study group | Role | What they receive |
|---|---|---|
| Phase I: Sequential Pharmacological PTIS | Experimental | Not described Receives: Drug: Fludarabine; Drug: Dexamethasone; Drug: Cyclophosphamide; Drug: Bortezomib; Drug: Rituximab; Drug: Busulfan; Drug: Cyclophosphamide (Cy); Drug: Tacrolimus (or cyclosporine) |
| Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy | Experimental | Not described Receives: Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Busulfan; Drug: Cyclophosphamide (Cy); Drug: Tacrolimus (or cyclosporine); Drug: Mycophenolate mofetil (MMF); Drug: Rabbit ATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the 100-day non-relapse mortality (NRM) rate when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2022
- Study start2 Mar 2022
- Primary completion18 Sep 2023
- Study completion18 Sep 2023
Why it stopped: 0 accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stem Cell TransplantationStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jeremy Ramdial, Ramdial, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05293509
- Sponsor's protocol ID
- 2021-0477
- Other IDs
- NCI-2022-02150
- Status last verified
- Sep 2023
- First submitted
- 4 Mar 2022
- Last updated
- 21 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacologic Pretransplant Immunosuppression (PTIS) + Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation in Inherited Hematologic Disorders
- ClinicalTrials.gov ID
- NCT05293509
- Acronym / sponsor ID
- 2021-0477
- Conditions
- Stem Cell Transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: 0 accrual
- Enrolment
- 0 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 2 Mar 2022 (actual)
- Primary completion
- 18 Sep 2023 (actual)
- Study completion
- 18 Sep 2023 (actual)
- First posted
- 24 Mar 2022 (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.
To assess the outcomes of NRM when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC) and an allogeneic stem cell transplant (allo-SCT) and post-transplant graft-versus-host disease prophylaxis based on post-transplant cyclophosphamide (PT-Cy) in patients with inherited blood disorders.
Detailed description
Objectives
Primary:
To estimate the 100-day non-relapse mortality (NRM) rate when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC) and an allogeneic stem cell transplant (allo-SCT) and post-transplant graft-versus-host disease prophylaxis based on post-transplant cyclophosphamide (PT-Cy) in patients with inherited blood disorders.
Secondary outcomes include the following:
- Immune reconstitution ii. Infectious complications iii. Quality of life (QOL) at 3 months,100 days, and 1 year post-transplant iv. OS, EFS, and GRFS v. Incidence of aGVHD at day 100. vi. Rate of chronic GVHD within the first-year post transplantation. vii. Rate of Graft failure
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phase I: Sequential Pharmacological PTIS | Experimental | Drug: Fludarabine, Drug: Dexamethasone, Drug: Cyclophosphamide, Drug: Bortezomib, Drug: Rituximab, Drug: Busulfan, Drug: Cyclophosphamide (Cy), Drug: Tacrolimus (or cyclosporine) |
| Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy | Experimental | Drug: Fludarabine, Drug: Cyclophosphamide, Drug: Busulfan, Drug: Cyclophosphamide (Cy), Drug: Tacrolimus (or cyclosporine), Drug: Mycophenolate mofetil (MMF), Drug: Rabbit ATG |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- The first six patients will be ages >12 years old and <35 years old. Thereafter in a second stage, patients ages 2 to 50 years old will be included.
- Patient with a matched related donor or who has a related haploidentical donor identified.
- Performance score of at least 70 by Karnofsky or 0 to 1 by ECOG (age > 12 years), or Zubrod or Lansky Play Performance Scale of at least 70 (age <12 years).
- Adequate major organ system function as demonstrated by:
- Patient or the patient's legal representative, parent(s) or guardian should be able to provide written informed consent. Assent of a minor if participant's age is at least seven and less than eighteen years.
Key exclusion criteria (10 listed)
- HIV positive; active hepatitis B or C.
- Uncontrolled infections.
- Liver cirrhosis. However mild fibrosis will be allowed i.e. fine reticulin or Grade 1, with bridging fibrosis.
- CNS involvement within 3 months.
- Positive pregnancy test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.
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 |
|---|---|---|
| FludarabineAlso: Fludara™40 mg/m2/day i.v.- by vein | Drug | Phase I: Sequential Pharmacological PTIS, Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy |
| Dexamethasone25 mg/m2/day i.v.-by vein | Drug | Phase I: Sequential Pharmacological PTIS |
| CyclophosphamideAlso: Cytoxan™100 mg/m2 IV-by vein | Drug | Phase I: Sequential Pharmacological PTIS, Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy |
| BortezomibAlso: Velcade®Four doses of bortezomib at a dose of 1.3 mg/m2 -injection under the skin | Drug | Phase I: Sequential Pharmacological PTIS |
| RituximabAlso: Rituxan®Four doses of rituximab at a dose of 375 mg/m2- by vein | Drug | Phase I: Sequential Pharmacological PTIS |
| BusulfanAlso: Busulfex™110 mg/m2 i.v-by vein | Drug | Phase I: Sequential Pharmacological PTIS, Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy |
| Cyclophosphamide (Cy)Also: Cytoxan™by vein | Drug | Phase I: Sequential Pharmacological PTIS, Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy |
| Tacrolimus (or cyclosporine)by vein | Drug | Phase I: Sequential Pharmacological PTIS, Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy |
| Mycophenolate mofetil (MMF)given by PO | Drug | Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy |
| Rabbit ATGby vein | Drug | Phase II: RTC Regimen and GVHD Prophylaxis Based on Post-Cy |
Primary outcome
To determine the 100-day non-relapse mortality (NRM) rate when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC
Time frame: through study completion, an average of 1 year
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 21 Sep 2023.
- M D Anderson Cancer CenterLink 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.
Jeremy Ramdial, Ramdial principal investigator
M.D. Anderson Cancer Center
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 Fludarabine, Dexamethasone and Cyclophosphamide in people with Stem Cell Transplantation, run by M.D. Anderson Cancer Center 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 10 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 21 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 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 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?
To determine the 100-day non-relapse mortality (NRM) rate when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC (time frame: through study completion, an average of 1 year). 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 21 Sep 2023.