Inborn Errors of ImmunityPhase 2NCT06199427
PTCy and and Ruxolitinib for GVHD Prophylaxis After HSCT With Thymoglobulin in Conditioning Regimen in Patients With Inborn Errors of Immunity
A Phase 2 study testing Cyclophosphamide and Ruxolitinib in people with Inborn Errors of Immunity, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
Trial summary
The aim of the current study is to evaluate the efficacy of combined regimen of GVHD prophylaxis with thymoglobulin in conditioning regimen and PTCY with ruxolitinib used after HSCT in patients with inborn errors of immunity (IEI) Read more about this study
- Who is it for?
- Ages 0 months to 21 years
- Conditions the study is for
- Inborn Errors of Immunity
- What is being tested?
- Cyclophosphamide and Ruxolitinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteRussia
- Study dates
- 21 Nov 2023to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients aged ≥ 0 months and < 21 years
- Patients diagnosed with NBS eligible for an allogeneic HSCT
- Signed written informed consent signed by a parent or legal guardian
Who cannot join
- Concomitant severe somatic disease associated with an additional risk of severe complications
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Russia1 site, 1 recruiting
- Recruiting
HSCT department
Moscow
Dmitry Balashov, MD, PhD, +74956647091, bala8@yandex.ru
About this study
Official title: Safety and Efficacy of Cyclophosphamide and Ruxolitinib for Graft-versus-host-disease Prophylaxis After Hematopoietic Stem Cell Transplantation With Thymoglobulin Serotherapy in Conditioning Regimen in Patients With Inborn Errors of Immunity
The aim of the current study is to evaluate the efficacy of combined regimen of GVHD prophylaxis with thymoglobulin in conditioning regimen and PTCY with ruxolitinib used after HSCT in patients with inborn errors of immunity (IEI)
Read the detailed description
Hematopoietic stem cell transplantation (HSCT) is widely used in inborn errors of immunity (IEI), and risks of graft-versus-host disease (GVHD) remain high. Use of post-transplant cyclophosphamide (PTCY) for GVHD prophylaxis revolutionized the outcomes of HSCT from mismatched related donor (MMRD). Use of ruxolitinib for GVHD prophylaxis demonstrates promising results in adult patients. Another well-known option for GVHD prevention is antithymocyte globulin. To evaluate the efficacy of combination of thymoglobulin with PTCY and ruxolitinib for GVHD prophylaxis, conditioning regimen containing treosulfan 30-42 g/m2, fludarabine 150 mg/mg, and thiotepa 10 mg/kg or melphalan 140 mg/m2 and GVHD prophylaxis regimen containing cyclophosphamide 50 mg/kg for MMRD, 25 mg/kg for matched unrelated and related donors at days +3, 4 post-HSCT and ruxolitinib at dose 7 mg/m2 from day +5 after HSCT will be used in patients with IEI.
- Conditions
- Inborn Errors of Immunity
- Being tested
- Cyclophosphamide, Ruxolitinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Cyclophosphamide 25mg/kg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg/kg (days +3, +4) after HSCT from MMRD
RuxolitinibDrug
Ruxolitinib 7 mg/m2 from day +5 after HSCT
| Study group | Role | What they receive |
|---|---|---|
| intervention/treatment | Experimental | Conditioning regimen containing treosulfan 30-42 g/m2, fludarabine 150 mg/mg, thymoglobulin 5 mg/kg and thiotepa 10 mg/kg or melphalan 140 mg/m2
GVHD prophylaxis regimen for matched unrelated (MUD) and matched related donors (MRD):
Cyclophosphamide (PTCY) 25 mg/kg/day (days +3, +4) Ruxolitinib 7 mg/m2 from day +5
GVHD prophylaxis regimen for mismatched related donor (MMRD):
Cyclophosphamide (PTCY) 50 mg/kg/day (days +3, +4) Ruxolitinib 7 mg/m2 from day +5 Receives: Drug: Cyclophosphamide; Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival
Events: graft failure, death
Measured: 1 year after HSCT
Secondary outcomes (9)
Overall survival
Measured: 1 year after HSCT
Cumulative incidence of acute graft versus host disease
Measured: 1 year after HSCT
Cumulative incidence of chronic graft versus host disease
Measured: 1 year after HSCT
Cumulative incidence of engraftment
Measured: 100 days
Cumulative incidence of graft failure
Measured: 1 year after HSCT
Incidence of early organ toxicity
Measured: 100 days
Cumulative incidence of transplant related mortality
Measured: 1 year after HSCT
Cumulative incidence of viral infections
Measured: 1 year after HSCT
Investigation of the concentration of ruxolitinib in the blood To investigate the pharmacokinetics of ruxolitinib
The features of pharmacokinetics in children of different ages
Measured: 1 month after HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2024
- Study start21 Nov 2023
- Primary completion21 Nov 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inborn Errors of ImmunityStudies of the same condition, open and under way.
- 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
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dmitry Balashov, MD, PhD, Study chair, National Research Center for Pediatric Hematology, Moscow, Russia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06199427
- Sponsor's protocol ID
- IEI-PTCy 2023
- Status last verified
- Dec 2023
- First submitted
- 28 Dec 2023
- Last updated
- 10 Jan 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 10 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Cyclophosphamide and Ruxolitinib for Graft-versus-host-disease Prophylaxis After Hematopoietic Stem Cell Transplantation With Thymoglobulin Serotherapy in Conditioning Regimen in Patients With Inborn Errors of Immunity
- ClinicalTrials.gov ID
- NCT06199427
- Acronym / sponsor ID
- IEI-PTCy 2023
- Conditions
- Inborn Errors of Immunity
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyUniversity, hospital or other
- Study start
- 21 Nov 2023 (actual)
- Primary completion
- 21 Nov 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 10 Jan 2024 (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.
The aim of the current study is to evaluate the efficacy of combined regimen of GVHD prophylaxis with thymoglobulin in conditioning regimen and PTCY with ruxolitinib used after HSCT in patients with inborn errors of immunity (IEI)
Detailed description
Hematopoietic stem cell transplantation (HSCT) is widely used in inborn errors of immunity (IEI), and risks of graft-versus-host disease (GVHD) remain high. Use of post-transplant cyclophosphamide (PTCY) for GVHD prophylaxis revolutionized the outcomes of HSCT from mismatched related donor (MMRD). Use of ruxolitinib for GVHD prophylaxis demonstrates promising results in adult patients. Another well-known option for GVHD prevention is antithymocyte globulin. To evaluate the efficacy of combination of thymoglobulin with PTCY and ruxolitinib for GVHD prophylaxis, conditioning regimen containing treosulfan 30-42 g/m2, fludarabine 150 mg/mg, and thiotepa 10 mg/kg or melphalan 140 mg/m2 and GVHD prophylaxis regimen containing cyclophosphamide 50 mg/kg for MMRD, 25 mg/kg for matched unrelated and related donors at days +3, 4 post-HSCT and ruxolitinib at dose 7 mg/m2 from day +5 after HSCT will be used in patients with IEI.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| intervention/treatment | Experimental | Drug: Cyclophosphamide, Drug: RuxolitinibConditioning regimen containing treosulfan 30-42 g/m2, fludarabine 150 mg/mg, thymoglobulin 5 mg/kg and thiotepa 10 mg/kg or melphalan 140 mg/m2 GVHD prophylaxis regimen for matched unrelated (MUD) and matched related donors (MRD): Cyclophosphamide (PTCY) 25 mg/kg/day (days +3, +4) Ruxolitinib 7 mg/m2 from day +5 GVHD prophylaxis regimen for mismatched related donor (MMRD): Cyclophosphamide (PTCY) 50 mg/kg/day (days +3, +4) Ruxolitinib 7 mg/m2 from day +5 |
Who may qualify
- Ages
- 0 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Patients aged ≥ 0 months and < 21 years
- Patients diagnosed with NBS eligible for an allogeneic HSCT
- Signed written informed consent signed by a parent or legal guardian
Key exclusion criteria (1 listed)
- Concomitant severe somatic disease associated with an additional risk of severe complications
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 |
|---|---|---|
| CyclophosphamideCyclophosphamide 25mg/kg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg/kg (days +3, +4) after HSCT from MMRD | Drug | intervention/treatment |
| RuxolitinibRuxolitinib 7 mg/m2 from day +5 after HSCT | Drug | intervention/treatment |
Primary outcome
Event-free survival
Time frame: 1 year after HSCT
Events: graft failure, death
9 secondary outcomes
Overall survival
Time frame: 1 year after HSCT
Cumulative incidence of acute graft versus host disease
Time frame: 1 year after HSCT
Cumulative incidence of chronic graft versus host disease
Time frame: 1 year after HSCT
Cumulative incidence of engraftment
Time frame: 100 days
Cumulative incidence of graft failure
Time frame: 1 year after HSCT
Incidence of early organ toxicity
Time frame: 100 days
Cumulative incidence of transplant related mortality
Time frame: 1 year after HSCT
Cumulative incidence of viral infections
Time frame: 1 year after HSCT
Investigation of the concentration of ruxolitinib in the blood To investigate the pharmacokinetics of ruxolitinib
Time frame: 1 month after HSCT
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2024.
Related trials for Inborn Errors of Immunity
RecruitingObservational study
NIAID Centralized Sequencing Protocol
Atopy, Primary Immunodeficiency
RecruitingObservational study
Immune Regulation in Patients With Common Variable Immunodeficiency and Related Inborn Errors of Immunity (IEI)
XLA, CVID
RecruitingObservational study
The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism
Oxidative Phosphorylation Deficiencies, Electron Transport Chain Disorders, Mitochondrial
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
Lymphoproliferative Disorders, Autoimmune Lymphoproliferative
Recruiting
Identification of Clinical, Genetic and Immunological Factors Involved in the Development of Severe Bacterial Infections in Pediatrics
Severe Bacterial Infections
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.
Dmitry Balashov, MD, PhD study chair
National Research Center for Pediatric Hematology, Moscow, Russia
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 Cyclophosphamide and Ruxolitinib in people with Inborn Errors of Immunity, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
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 3 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 10 Jan 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 Russia. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dmitry Balashov, MD, PhD (+74956647091, bala8@yandex.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-free survival (time frame: 1 year after HSCT). 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 10 Jan 2024.