Allogeneic Hematopoietic Cell TransplantationEarly Phase 1NCT04332341
Study of Nicotinamide Riboside Supplementation in Allogeneic Hematopoietic Cell Transplantation
A Early Phase 1 study testing Nicotinamide riboside (NR) in people with Allogeneic Hematopoietic Cell Transplantation, run by Case Comprehensive Cancer Center at 1 site in United States.
Trial summary
The purpose of this study is to find a safe and tolerable way to improve engraftment after transplant. Research studies have shown that adding nicotinamide riboside to donor cells has the potential to increase blood stem cell numbers and potentially decrease the time to engraftment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Allogeneic Hematopoietic Cell Transplantation
- What is being tested?
- Nicotinamide riboside (NR)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 19 May 2020to Sep 2026
- Phase
- Early Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All disease indications for allogeneic hematopoietic cell transplantation except for myelofibrosis.
- Subject must meet program eligibility criteria* for myeloablative conditioning alloHCT and plan to undergo myeloablative conditioning.
- HLA-identical related donor or unrelated human donor source with bone marrow graft.
- Subjects must provide a written informed consent.
Who cannot join
- History of allergy or intolerance to NR precursor compounds, including niacin or nicotinamide
- Pregnant or breastfeeding women are excluded from this study since allogeneic HCT is a strict contraindication.
- Subjects with uncontrolled intercurrent illness or psychiatric illness/social situations that would limit compliance with study requirements.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cleveland Clinic, Case Comprehensive Cancer Center
Cleveland, Ohio, 44195
Navneet Majhail, MD, 866-223-8100, TaussigResearch@ccf.org
Navneet Majhail, MD
About this study
Official title: A Pilot Study of Nicotinamide Riboside Supplementation in Allogeneic Hematopoietic Cell Transplantation
The purpose of this study is to find a safe and tolerable way to improve engraftment after transplant. Research studies have shown that adding nicotinamide riboside to donor cells has the potential to increase blood stem cell numbers and potentially decrease the time to engraftment. Also, nicotinamide riboside, TRU NIAGEN (the study drug) is a type of vitamin B supplement that the general public can get without a prescription and is well tolerated.
Read the detailed description
This is a single-center, pilot feasibility study of NR supplementation in allogeneic (donor) HCT in which the study team is seeking to obtain preliminary data on any beneficial effect of increasing NAD+ levels in-vivo to facilitate engraftment. Engraftment is defined as the process during which transplanted stem cells begin to grow in the bone marrow and produce new white blood cells, red blood cells, and platelets. It takes about 14 to 24 days for donor cells to engraft after infusion, and the time between which blood counts fall to the time when they recover is a very critical period. Transfusions are frequently required to prevent bleeding and antibiotics are needed to prevent infections during this critical time period. Shortening the time of engraftment decreases these risks and can improve long-term health status.
The primary objectives of this study is to evaluate safety and tolerability of NR supplementation
The secondary objective of this study is to evaluate neutrophil and platelet recovery after HCT
- Being tested
- Nicotinamide riboside (NR)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nicotinamide riboside (NR)Drug
Nicotinamide riboside, PO, 500mgtwice daily for 21, 28, and 35 days
| Study group | Role | What they receive |
|---|---|---|
| Nicotinamide riboside (NR) | Experimental | Not described Receives: Drug: Nicotinamide riboside (NR) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent of participants who receive >75% of scheduled doses.
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive >75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Measured: day +7
Percent of participants who receive >75% of scheduled doses.
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive >75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Measured: day +14
Percent of participants who receive >75% of scheduled doses.
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive >75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Measured: day +21
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
Measured: day +7
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
Measured: day +14
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
Measured: day +21
Secondary outcomes (2)
Median days to neutrophil recovery after HCT
Median and range of days to neutrophil recovery after HCT. Recovery defined as first day of three consecutive days with absolute neutrophil count 500 cells/mm3 (0.5 x 109/L) or greater
Measured: Up to 100 days from start of treatment
Median days to platelet recovery after HCT
Median and range of days to Platelet recovery after HCT. Recovery defined as first day of three consecutive days with platelet count 20,000/mm3 (20 x 109/L) or greater and unsupported by platelet transfusions.
Measured: Up to 100 days from start of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Apr 2020
- Study start19 May 2020
- Primary completionSep 2026 (estimated)
- Study completionSep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Allogeneic Hematopoietic Cell TransplantationStudies of the same condition, open and under way.
- More trials run by Case Comprehensive 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Case Comprehensive Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ronald Sobecks, MD, Principal investigator, Cleveland Clinic, Case Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04332341
- Sponsor's protocol ID
- CASE2Z20
- Status last verified
- Aug 2026
- First submitted
- 31 Mar 2020
- Last updated
- 6 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: IPD will not be shared publicly
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Nicotinamide Riboside Supplementation in Allogeneic Hematopoietic Cell Transplantation
- ClinicalTrials.gov ID
- NCT04332341
- Acronym / sponsor ID
- CASE2Z20
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Case Comprehensive Cancer CenterUniversity, hospital or other
- Study start
- 19 May 2020 (actual)
- Primary completion
- Sep 2026 (estimated)
- Study completion
- Sep 2026 (estimated)
- First posted
- 2 Apr 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 purpose of this study is to find a safe and tolerable way to improve engraftment after transplant. Research studies have shown that adding nicotinamide riboside to donor cells has the potential to increase blood stem cell numbers and potentially decrease the time to engraftment. Also, nicotinamide riboside, TRU NIAGEN (the study drug) is a type of vitamin B supplement that the general public can get without a prescription and is well tolerated.
Detailed description
This is a single-center, pilot feasibility study of NR supplementation in allogeneic (donor) HCT in which the study team is seeking to obtain preliminary data on any beneficial effect of increasing NAD+ levels in-vivo to facilitate engraftment. Engraftment is defined as the process during which transplanted stem cells begin to grow in the bone marrow and produce new white blood cells, red blood cells, and platelets. It takes about 14 to 24 days for donor cells to engraft after infusion, and the time between which blood counts fall to the time when they recover is a very critical period. Transfusions are frequently required to prevent bleeding and antibiotics are needed to prevent infections during this critical time period. Shortening the time of engraftment decreases these risks and can improve long-term health status.
The primary objectives of this study is to evaluate safety and tolerability of NR supplementation
The secondary objective of this study is to evaluate neutrophil and platelet recovery after HCT
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nicotinamide riboside (NR) | Experimental | Drug: Nicotinamide riboside (NR) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- All disease indications for allogeneic hematopoietic cell transplantation except for myelofibrosis.
- Subject must meet program eligibility criteria* for myeloablative conditioning alloHCT and plan to undergo myeloablative conditioning.
- HLA-identical related donor or unrelated human donor source with bone marrow graft.
- Subjects must provide a written informed consent.
Key exclusion criteria (3 listed)
- History of allergy or intolerance to NR precursor compounds, including niacin or nicotinamide
- Pregnant or breastfeeding women are excluded from this study since allogeneic HCT is a strict contraindication.
- Subjects with uncontrolled intercurrent illness or psychiatric illness/social situations that would limit compliance with study requirements.
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 |
|---|---|---|
| Nicotinamide riboside (NR)Nicotinamide riboside, PO, 500mgtwice daily for 21, 28, and 35 days | Drug | Nicotinamide riboside (NR) |
Primary outcome
Percent of participants who receive >75% of scheduled doses.
Time frame: day +7
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive >75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Percent of participants who receive >75% of scheduled doses.
Time frame: day +14
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive >75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Percent of participants who receive >75% of scheduled doses.
Time frame: day +21
Tolerability of NR in allogeneic HCT recipients, defined as 8 or more of 15 participants (i.e. at least 50% of enrolled subjects) able to receive >75% of their scheduled doses (overall rate 53%, 95% confidence intervals 33%-86%). This will include both patients who discontinue NR due to study drug related toxicity and patients who are not able to stay on NR due to transplant related toxicity that limits ability to take the drug
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
Time frame: day +7
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
Time frame: day +14
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
Percent of participants experiencing a CTCAE 5.0 grade 3 or higher related to the study drug
Time frame: day +21
Safety of NR in allogeneic HCT recipients as measured by percent of participants experiencing a CTCAE 5.0 grade 3 or higher that is related to the study drug
2 secondary outcomes
Median days to neutrophil recovery after HCT
Time frame: Up to 100 days from start of treatment
Median days to platelet recovery after HCT
Time frame: Up to 100 days from start of treatment
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 6 Aug 2026.
- Individual participant dataSharing planned: No. IPD will not be shared publicly
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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.
Ronald Sobecks, MD principal investigator
Cleveland Clinic, Case Comprehensive Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Nicotinamide riboside (NR) in people with Allogeneic Hematopoietic Cell Transplantation, run by Case Comprehensive 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 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: looking for participants now, as of the sponsor's last update on 6 Aug 2026. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 Ronald Sobecks, MD (1-866-223-8100, TaussigResearch@ccf.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent of participants who receive >75% of scheduled doses. (time frame: day +7). The study lists 6 primary outcomes in all. 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 6 Aug 2026.