Study of Nicotinamide Riboside Supplementation in Allogeneic Hematopoietic Cell Transplantation

NCT04332341 | Recruiting Early Phase 1 DMC FDA Drug

• 1 other identifier: CASE2Z20
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Allogeneic Hematopoietic Cell Transplantation

Outcome Measures

Primary Outcomes (6)

• 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

  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

  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

  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

  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

  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

  day +21

Secondary Outcomes (2)

• Median days to neutrophil recovery after HCT

  Up to 100 days from start of treatment

• Median days to platelet recovery after HCT

  Up to 100 days from start of treatment

Study Arms (1)

Nicotinamide riboside (NR)

EXPERIMENTAL

Drug: Nicotinamide riboside (NR)

Interventions

Nicotinamide riboside (NR) DRUG

Nicotinamide riboside, PO, 500mgtwice daily for 21, 28, and 35 days

Nicotinamide riboside (NR)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Case Comprehensive Cancer Center: lead

Study Sites (1)

Cleveland Clinic, Case Comprehensive Cancer Center

Cleveland, Ohio, 44195, United States

RECRUITING

MeSH Terms

Interventions

nicotinamide-beta-riboside

Study Officials

• Ronald Sobecks, MD

  Cleveland Clinic, Case Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ronald Sobecks, MD

CONTACT

1-866-223-8100; TaussigResearch@ccf.org

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2020
• First Posted: April 2, 2020
• Study Start: May 19, 2020
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: February 18, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)