ThrombocytopeniaPhase 2/3NCT03391856
Prolonged Isolated Thrombocytopenia After Allo-SCT : N-acetyl-L-cysteine (NAC) Versus Supportive Therapy
A Phase 2/3 study testing N-acetyl-L-cysteine (NAC) and supportive therapy in people with Thrombocytopenia and Stem Cell Transplant Complications, run by Peking University People's Hospital at 1 site in China.
Trial summary
Isolated prolonged thrombocytopenia (PT) is a common complication after allogeneic stem cell transplantation with significant poor prognosis. No standard treatment is available. The current study assigned PT randomly to 2 arms: intervention arm with N-acetyl-L-cysteine (NAC) and control arm with supportive… Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Thrombocytopenia, Stem Cell Transplant Complications
- What is being tested?
- N-acetyl-L-cysteine (NAC) and supportive therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- 1 Mar 2018to 30 Dec 2018
- Phase
- Phase 2/3
- 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 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- age>= 14 years
- diagnosed as PT (defined as platelet count in peripheral blood never achieve 20000/miro-liter for 7 consecutive days without transfusion at day 60 post transplantation)
- serum creatine level < ULN (upper limit of normal), serum ALT/AST /TBil<=2 ULN
- without active CMV/EBV/ADV reactivation, active GVHD, without relapse or minimal residual disease at enrollment
Who cannot join
- history of asthma
- allergy to NAC
- refuse to participate
Trial locations and contacts
1 site in 1 country.
China1 site
Peking University People's Hospital
Beijing, Beijing Municipality, 100044
About this study
Official title: A Prospective Randomized Controlled Study for Intervention of Prolonged Isolated Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplantation: N-acetyl-L-cysteine (NAC) Versus Supportive Therapy
Isolated prolonged thrombocytopenia (PT) is a common complication after allogeneic stem cell transplantation with significant poor prognosis. No standard treatment is available. The current study assigned PT randomly to 2 arms: intervention arm with N-acetyl-L-cysteine (NAC) and control arm with supportive therapy.This is a prospective randomized controlled study.
Read the detailed description
- patients diagnosed with PT at day 60 post transplant will be randomized assigned to intervention arm (NAC) or controlled arm (supportive therapy: prophylactic platelet transfusion was given when platelet count <20000/ul.)
- Response will be evaluated at day 90. Response was defined as platelet recovery to >= 20000/ul for 7 consecutive days without transfusion support during the enrollment period. All the other patients not achieved above criteria was defined as no response.
- For those without response in both arms, patients will received NAC plus recombinant human thrombopoietin (rhTPO) for an additional 30 days. rhTPO was given at 300IU/kg/d for 28 consecutive days or platelet > 50000/ul independent of platelet transfusion.
- Being tested
- N-acetyl-L-cysteine (NAC), supportive therapy
- Medical subject headings
- Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetyl-L-cysteine (NAC)Drug
NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant
supportive therapyOther
prophylactic platelet transfusion was given when platelet count <20000/ul
| Study group | Role | What they receive |
|---|---|---|
| intervention arm | Experimental | NAC 400mg p.o tid from day 60 to day 90 post transplant Receives: Drug: N-acetyl-L-cysteine (NAC) |
| controlled arm | Other | Supportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count <20000/ul Receives: Other: supportive therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
response rate
rate of response after 30 days of treatment
Measured: 30 days after the start of enrollement
Secondary outcomes (4)
overall survival
survival proportion at the last followup after transplantation
Measured: the day of last follow-up
non-relapse mortality
non-relapse mortality at the last followup after transplantation
Measured: the day of last follow-up
grade 2-4 acute graft versus host disease
grade 2-4 aGVHD at day 100 after transplant
Measured: day 100 after transplant
chronic graft versus host disease
chronic graft versus host disease at the day of last follow-up
Measured: the day of last follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2018
- Study start1 Mar 2018
- Primary completion30 Dec 2018
- Study completion30 Dec 2018
Why it stopped: protocol changed
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- The First Affiliated Hospital of Soochow University, Nanfang Hospital, Southern Medical University
- Responsible party
- Principal investigator, Xiaojun Huang,MD, Peking University People's Hospital
- Investigators
- Xiao-Jun Huang, MD, Principal investigator, Peiking University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03391856
- Sponsor's protocol ID
- 2017PHB220
- Status last verified
- Sep 2020
- First submitted
- 2 Jan 2018
- Last updated
- 25 Sep 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Randomized Controlled Study for Intervention of Prolonged Isolated Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplantation: N-acetyl-L-cysteine (NAC) Versus Supportive Therapy
- ClinicalTrials.gov ID
- NCT03391856
- Acronym / sponsor ID
- 2017PHB220
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: protocol changed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- The First Affiliated Hospital of Soochow University, Nanfang Hospital, Southern Medical University
- Study start
- 1 Mar 2018 (actual)
- Primary completion
- 30 Dec 2018 (actual)
- Study completion
- 30 Dec 2018 (actual)
- First posted
- 5 Jan 2018 (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.
Isolated prolonged thrombocytopenia (PT) is a common complication after allogeneic stem cell transplantation with significant poor prognosis. No standard treatment is available. The current study assigned PT randomly to 2 arms: intervention arm with N-acetyl-L-cysteine (NAC) and control arm with supportive therapy.This is a prospective randomized controlled study.
Detailed description
- patients diagnosed with PT at day 60 post transplant will be randomized assigned to intervention arm (NAC) or controlled arm (supportive therapy: prophylactic platelet transfusion was given when platelet count <20000/ul.)
- Response will be evaluated at day 90. Response was defined as platelet recovery to >= 20000/ul for 7 consecutive days without transfusion support during the enrollment period. All the other patients not achieved above criteria was defined as no response.
- For those without response in both arms, patients will received NAC plus recombinant human thrombopoietin (rhTPO) for an additional 30 days. rhTPO was given at 300IU/kg/d for 28 consecutive days or platelet > 50000/ul independent of platelet transfusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| intervention arm | Experimental | Drug: N-acetyl-L-cysteine (NAC)NAC 400mg p.o tid from day 60 to day 90 post transplant |
| controlled arm | Other | Other: supportive therapySupportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count <20000/ul |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- age>= 14 years
- diagnosed as PT (defined as platelet count in peripheral blood never achieve 20000/miro-liter for 7 consecutive days without transfusion at day 60 post transplantation)
- serum creatine level < ULN (upper limit of normal), serum ALT/AST /TBil<=2 ULN
- without active CMV/EBV/ADV reactivation, active GVHD, without relapse or minimal residual disease at enrollment
Key exclusion criteria (3 listed)
- history of asthma
- allergy to NAC
- refuse to participate
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 |
|---|---|---|
| N-acetyl-L-cysteine (NAC)NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant | Drug | intervention arm |
| supportive therapyprophylactic platelet transfusion was given when platelet count <20000/ul | Other | controlled arm |
Primary outcome
response rate
Time frame: 30 days after the start of enrollement
rate of response after 30 days of treatment
4 secondary outcomes
overall survival
Time frame: the day of last follow-up
non-relapse mortality
Time frame: the day of last follow-up
grade 2-4 acute graft versus host disease
Time frame: day 100 after transplant
chronic graft versus host disease
Time frame: the day of last follow-up
Results and safety
The study was stopped early (reason given: protocol changed). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2020.
- Individual participant dataSharing planned: No
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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.
Xiao-Jun Huang, MD principal investigator
Peiking University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing N-acetyl-L-cysteine (NAC) and supportive therapy in people with Thrombocytopenia and Stem Cell Transplant Complications, run by Peking University People's Hospital at 1 site in China.
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 4 inclusion and 3 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 25 Sep 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 China. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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: protocol changed). 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?
response rate (time frame: 30 days after the start of enrollement). 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 Sep 2020.