Nijmegen Breakage SyndromePhase 2NCT04400045
Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage Syndrome
A Phase 2 study testing Treosulfan in people with Nijmegen Breakage Syndrome, 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 safety and efficacy of low dose treosulfan based conditioning regimen in HSCT in Nijmegen breakage syndrome Read more about this study
- Who is it for?
- Ages 3 months to 21 years
- Conditions the study is for
- Nijmegen Breakage Syndrome
- What is being tested?
- Treosulfan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteRussia
- Study dates
- 22 May 2020to May 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 3 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients aged ≥ 3 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
None listed.
Trial locations and contacts
1 site in 1 country.
Russia1 site, 1 recruiting
- Recruiting
HSCT department
Moscow, 117198
Dmitry Balashov, MD, PhD, +74952876570 ext. 6534, bala8@yandex.ru
About this study
Official title: Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage Syndrome
The aim of the current study is to evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in HSCT in Nijmegen breakage syndrome
Read the detailed description
Nijmegen breakage syndrome (NBS) is a DNA repair disorder. The only curative option for combine immunodeficiency in NBS is allogeneic hematopoietic stem cell transplantation (HSCT). Standard myeloablative conditioning regimens in DNA repair disorders lead to increased morbidity and mortality after HSCT. Low doses of alkylators are used to reduce toxicity rates, which, however, increase the risks of mixed chimerism and graft failure. The data of treosulfan usage in NBS are sparse. To evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in NBS, treosulfan 21g/m2 in combination with fludarabine 150mg/mg, cyclophosphamide 40mg/kg, thymoglobulin (Genzyme) 5mg/kg and rituximab 100mg/m2 will be used from day -6 to -1 day, followed by stem cell infusion. The primary endpoint is event-free survival, where graft failure, death, and malignancies are considered as events.
- Conditions
- Nijmegen Breakage Syndrome
- Being tested
- Treosulfan
- Medical subject headings
- Nijmegen Breakage Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TreosulfanDrug
Treosulfan 21mg/m2 (days -6, -5, -4)
| Study group | Role | What they receive |
|---|---|---|
| intervention/treatment | Experimental | Fludarabine 150mg/m2 (days -6, -5, -4, -3, -2) Treosulfan 21g/m2 (days -6, -5, -4) Cyclophosphamide 40mg/kg (days -3, -2) Thymoglobulin (Genzyme) 5mg/kg (days -5, -4) Rituximab 100mg/m2 (day -1) Stem cell infusion - day 0 Receives: Drug: Treosulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival
Events: graft failure, death, malignancies
Measured: 3 years after HSCT
Secondary outcomes (9)
Overall survival
Measured: 3 years after HSCT
Cumulative incidence of engraftment
Measured: 100 days
Cumulative incidence of graft failure
Measured: 3 years
Cumulative incidence of viral infections
Measured: 1 year
Cumulative incidence of acute graft versus host disease
Measured: 1 year
Cumulative incidence of chronic graft versus host disease
Measured: 3 years
Incidence of early organ toxicity
Measured: 100 days
Cumulative incidence of transplant related mortality
Measured: 3 years
Incidence of long-term toxicity
malignancies, non-malignant complications
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2020
- Study start22 May 2020
- Primary completionMay 2023 (estimated)
- Study completionMay 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nijmegen Breakage SyndromeStudies 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
- 10 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04400045
- Sponsor's protocol ID
- NCPHOI-2020-04
- Status last verified
- Jun 2020
- First submitted
- 19 May 2020
- Last updated
- 25 Jun 2020
- 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 25 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage Syndrome
- ClinicalTrials.gov ID
- NCT04400045
- Acronym / sponsor ID
- NCPHOI-2020-04
- Conditions
- Nijmegen Breakage Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyUniversity, hospital or other
- Study start
- 22 May 2020 (actual)
- Primary completion
- May 2023 (estimated)
- Study completion
- May 2023 (estimated)
- First posted
- 22 May 2020 (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 safety and efficacy of low dose treosulfan based conditioning regimen in HSCT in Nijmegen breakage syndrome
Detailed description
Nijmegen breakage syndrome (NBS) is a DNA repair disorder. The only curative option for combine immunodeficiency in NBS is allogeneic hematopoietic stem cell transplantation (HSCT). Standard myeloablative conditioning regimens in DNA repair disorders lead to increased morbidity and mortality after HSCT. Low doses of alkylators are used to reduce toxicity rates, which, however, increase the risks of mixed chimerism and graft failure. The data of treosulfan usage in NBS are sparse. To evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in NBS, treosulfan 21g/m2 in combination with fludarabine 150mg/mg, cyclophosphamide 40mg/kg, thymoglobulin (Genzyme) 5mg/kg and rituximab 100mg/m2 will be used from day -6 to -1 day, followed by stem cell infusion. The primary endpoint is event-free survival, where graft failure, death, and malignancies are considered as events.
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: TreosulfanFludarabine 150mg/m2 (days -6, -5, -4, -3, -2) Treosulfan 21g/m2 (days -6, -5, -4) Cyclophosphamide 40mg/kg (days -3, -2) Thymoglobulin (Genzyme) 5mg/kg (days -5, -4) Rituximab 100mg/m2 (day -1) Stem cell infusion - day 0 |
Who may qualify
- Ages
- 3 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Patients aged ≥ 3 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 (0 listed)
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 |
|---|---|---|
| TreosulfanTreosulfan 21mg/m2 (days -6, -5, -4) | Drug | intervention/treatment |
Primary outcome
Event-free survival
Time frame: 3 years after HSCT
Events: graft failure, death, malignancies
9 secondary outcomes
Overall survival
Time frame: 3 years after HSCT
Cumulative incidence of engraftment
Time frame: 100 days
Cumulative incidence of graft failure
Time frame: 3 years
Cumulative incidence of viral infections
Time frame: 1 year
Cumulative incidence of acute graft versus host disease
Time frame: 1 year
Cumulative incidence of chronic graft versus host disease
Time frame: 3 years
Incidence of early organ toxicity
Time frame: 100 days
Cumulative incidence of transplant related mortality
Time frame: 3 years
Incidence of long-term toxicity
Time frame: 3 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2020.
Related trials for Nijmegen Breakage Syndrome
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Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome
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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.
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 Treosulfan in people with Nijmegen Breakage Syndrome, 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 0 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: status not confirmed, as of the sponsor's last update on 25 Jun 2020. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Dmitry Balashov, MD, PhD (+74952876570 ext. 6534, 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: 3 years 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 25 Jun 2020.