Steroid-refractory Acute Graft-versus-Host DiseasePhase 1NCT03327857
Neihulizumab (ALTB-168) in Patients With Steroid-refractory Acute Graft-versus-host Disease or Treatment-refractory Acute Graft-versus-host Disease
A Phase 1 study testing Neihulizumab (ALTB-168) in people with Steroid-refractory Acute Graft-versus-Host Disease and Treatment-refractory Acute Graft-versus-Host Disease, run by AltruBio Inc. at 13 sites in United States.
Trial summary
A Phase I study to establish the pharmacokinetics, pharmacodynamics, safety and efficacy profiles of Neihulizumab in patients with steroid-refractory or treatment refractory acute graft-versus-host disease (SR/TR-aGVHD) Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Steroid-refractory Acute Graft-versus-Host Disease, Treatment-refractory Acute Graft-versus-Host Disease
- What is being tested?
- Neihulizumab (ALTB-168)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 13 sitesUnited States
- Study dates
- 31 May 2018to 31 Dec 2022
- Phase
- Phase 1
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Inclusion Criteria (must meet all of the following criteria):
- Patients must have clinical aGVHD and pathologic findings consistent with the diagnosis by biopsy of at least 1 involved site, and
- progressed after 3 days of treatment with methylprednisolone (MP) 2 mg/kg/day equivalent, or
- did not improve after 7 days of treatment with MP 2 mg/kg/day equivalent, or
- progressed to involve a new organ after treatment with MP 1 mg/kg/day equivalent for skin and upper gastrointestinal (GI) GVHD, or
- recurred during or after a steroid taper
- For single dose phase: Patients must have erythematous manifestations of cutaneous aGVHD. Characteristics of the rash must indicate active inflammation (red coloration) as distinct from resolving inflammation (brown coloration).
- For single dose phase: Providers and patients must be willing to defer new systemic or cutaneous topical treatment of aGVHD for at least 36 hr after administration of Neihulizumab.
- Patient must give informed consent and sign an approved consent form prior to any study procedures.
- Females of childbearing potential must have a negative pregnancy test result before enrollment. Males and females of childbearing potential must agree to use a highly effective method of birth control during the study for at least 30 days after enrollment in the study.
Exclusion Criteria (may not meet any of the following criteria):
- For single dose phase: Prior administration of anti-lymphocyte globulin or anti- thymocyte globulin for treatment of aGVHD.
- For multiple dose phase: Has received any systemic treatment in addition to corticosteroids for aGVHD.
- Stage 4 lower GI GVHD, defined by the presence of ileus, severe abdominal pain, or overt GI bleeding.
- Uncontrolled infections not responding to antimicrobial therapy or requiring intensive critical care or vasopressors.
- Evidence of end-organ cytomegalovirus (CMV) or adenovirus infection.
- Known to have adenovirus, or Epstein Barr virus (EBV) viremia from screening according to institutional standard practice. Patients receiving appropriate antiviral treatment for CMV, HHV6 or hepatitis viremia are eligible on a case-by-case basis.
- HIV infection or a known HIV-related malignancy.
- Tuberculosis, history of tuberculosis or a known positive Quantiferon test for tuberculosis.
- Unplanned donor lymphocyte infusion (DLI) for residual or relapsed malignancy or mixed chimerism. DLI as part of the planned HCT protocol is allowed.
- Known relapsed or progressive malignancy after transplant, posttransplant lymphoproliferative disease or any secondary malignancy diagnosed after HCT.
- Absolute neutrophil count (ANC) <1000/mm3.
- Total serum bilirubin concentration >3.0 mg/dL UNLESS attributed to GVHD.
- Creatinine clearance < 30 mL/min calculated by Cockcroft-Gault equation.
- Sodium (Na) concentration < 130 mmol/L.
- Karnofsky Performance Status (KPS) or Lansky Performance Status < 20%.
- Intensive care unit (ICU) care, life expectancy of less than 28 days, ongoing or unresolved hepatic sinusoidal obstruction syndrome, unstable hemodynamics, or evidence of current or previous clinically significant disease, medical condition or finding (including vital signs and ECG) that in the opinion of the Investigator, would compromise the safety of the patient or the quality of the data.
- History of allergy or hypersensitivity to any systemically administered antibody agent or its excipients.
- Pregnancy or nursing.
- Less than 12 years of age.
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory University
Atlanta, Georgia, 30322
Dana Farber Cancer Center
Boston, Massachusetts, 02215
University of Chicago
Chicago, Illinois, 60637
University Hospitals Seidman Cancer Center
Cleveland, Ohio, 44106
City of Hope
Duarte, California, 91010
Baylor College of Medicine-Houston Methodist & Texas Children's Hospital
Houston, Texas, 77030
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095
University of Miami - Sylvester Comprehensive Cancer Center
Miami, Florida, 33136
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota
Minneapolis, Minnesota, 55455
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
The University of Kansas Cancer Center
Westwood, Kansas, 66205
About this study
Official title: A Phase I Study of Neihulizumab (ALTB-168) in Patients With Steroid-refractory Acute Graft-versus-host Disease (SR-aGVHD) or Treatment-refractory Acute Graft-versus-host Disease (TR-aGVHD)
A Phase I study to establish the pharmacokinetics, pharmacodynamics, safety and efficacy profiles of Neihulizumab in patients with steroid-refractory or treatment refractory acute graft-versus-host disease (SR/TR-aGVHD)
Read the detailed description
Neihulizumab (ALTB-168) is an immune checkpoint agonist antibody that regulates T cell homeostasis. The unique mechanism of action provides a natural regulation of T cell homeostasis that induces cell death preferentially in late-stage activated T cells without affecting resting T cells and early-activated T cells. Because pathogenic T cells underlying the inflammatory conditions are usually in late-stage activated state, eliminating this population of cells can potentially result in controlling autoimmune inflammation of T cell associated diseases, such as GvHD.
- Conditions
- Steroid-refractory Acute Graft-versus-Host Disease, Treatment-refractory Acute Graft-versus-Host Disease
- Being tested
- Neihulizumab (ALTB-168)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Neihulizumab (ALTB-168)Biological
Single dose phase: Patients will receive single dose of Neihulizumab based on the protocol escalation criteria.
Multiple dose phase: Parients will receive weekly doses of Neihulizumab for 4 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Neihulizumab (ALTB-168) | Experimental | Intravenous doses of Neihulizumab (ALTB-168) Receives: Biological: Neihulizumab (ALTB-168) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetics of Neihulizumab - AUC
Including AUC0-t, AUC0-tz, AUC 0-inf
Measured: Up to Day 56
Pharmacokinetics of Neihulizumab - Cmax
Maximum plasma concentration
Measured: Up to Day 56
Pharmacokinetics of Neihulizumab - tmax
Time to reach Cmax
Measured: Up to Day 56
Pharmacokinetics of Neihulizumab - Lambda-z
Terminal phase elimination rate constant
Measured: Up to Day 56
Pharmacokinetics of Neihulizumab - t1/2
Half life
Measured: Up to Day 56
Pharmacokinetics of Neihulizumab - MRT
Mean Residence Time
Measured: Up to Day 56
Pharmacokinetics of Neihulizumab - Vz and Vss
Volume of distribution and volume of distribution at steady state
Measured: Up to Day 56
Secondary outcomes (8)
Adverse Events (AEs)
AEs graded according to CTCAE v4.03
Measured: Up to Day 180
To measure the Receptor Occupancy (RO)
Receptor occupancy will be monitored using a flow cytometry based method
Measured: Up to Day 56
To measure regenerating islet-derived 3-alpha (REG3α) and suppression of tumorigenicity 2 (ST2) as Pharmacodynamics (PD) biomarkers.
Receptor occupancy will be monitored using a flow cytometry based method
Measured: Up to Day 56
Complete Response (CR)
To assess the rate of complete response (CR) at Day 28 in patients treated with Neihulizumab
Measured: Day 28
Overall Response Rate (ORR)
To assess the Overall Response Rate (ORR) at Day 28: CR+PR
Measured: Day 28
Duration of Response
For subjects with CR at Day 28, duration of response will be assessed according to the time interval from Day 28 to the first occurrence of (1) resumption of Neihulizumab administration or initiation of new systemic treatment for aGvHD or (for patients who have tapered steroids) an increase in corticosteroids to methylprednisolone 2 mg/kg (+/-10%) equivalent or more, or (2) death.
Measured: Up to Day 180
Non Relapse Mortality (NRM)
Patients will be followed-up for survival for 6 months after the first Neihulizumab treatment
Measured: Day 180
Immunogenicity
Immunogenicity will be monitored by anti-drug antibody (ADA) ELISA
Measured: Up to Day 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2017
- Study start31 May 2018
- Primary completion30 Sep 2022
- Study completion31 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Steroid-refractory Acute Graft-versus-Host DiseaseStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- AltruBio Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shih-Yao Lin, MD, PhD, Study director, AltruBio, Inc. (formerly AbGenomics International)Paul Martin, MD, Principal investigator, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03327857
- Sponsor's protocol ID
- 2017.002.01
- Status last verified
- Jan 2023
- First submitted
- 13 Oct 2017
- Last updated
- 19 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Neihulizumab (ALTB-168) in Patients With Steroid-refractory Acute Graft-versus-host Disease (SR-aGVHD) or Treatment-refractory Acute Graft-versus-host Disease (TR-aGVHD)
- ClinicalTrials.gov ID
- NCT03327857
- Acronym / sponsor ID
- 2017.002.01
- Conditions
- Steroid-refractory Acute Graft-versus-Host Disease, Treatment-refractory Acute Graft-versus-Host Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- AltruBio Inc.Industry (pharma, biotech, devices)
- Study start
- 31 May 2018 (actual)
- Primary completion
- 30 Sep 2022 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 1 Nov 2017 (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.
A Phase I study to establish the pharmacokinetics, pharmacodynamics, safety and efficacy profiles of Neihulizumab in patients with steroid-refractory or treatment refractory acute graft-versus-host disease (SR/TR-aGVHD)
Detailed description
Neihulizumab (ALTB-168) is an immune checkpoint agonist antibody that regulates T cell homeostasis. The unique mechanism of action provides a natural regulation of T cell homeostasis that induces cell death preferentially in late-stage activated T cells without affecting resting T cells and early-activated T cells. Because pathogenic T cells underlying the inflammatory conditions are usually in late-stage activated state, eliminating this population of cells can potentially result in controlling autoimmune inflammation of T cell associated diseases, such as GvHD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Neihulizumab (ALTB-168) | Experimental | Biological: Neihulizumab (ALTB-168)Intravenous doses of Neihulizumab (ALTB-168) |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| Neihulizumab (ALTB-168)Single dose phase: Patients will receive single dose of Neihulizumab based on the protocol escalation criteria. Multiple dose phase: Parients will receive weekly doses of Neihulizumab for 4 weeks. | Biological | Neihulizumab (ALTB-168) |
Primary outcome
Pharmacokinetics of Neihulizumab - AUC
Time frame: Up to Day 56
Including AUC0-t, AUC0-tz, AUC 0-inf
Pharmacokinetics of Neihulizumab - Cmax
Time frame: Up to Day 56
Maximum plasma concentration
Pharmacokinetics of Neihulizumab - tmax
Time frame: Up to Day 56
Time to reach Cmax
Pharmacokinetics of Neihulizumab - Lambda-z
Time frame: Up to Day 56
Terminal phase elimination rate constant
Pharmacokinetics of Neihulizumab - t1/2
Time frame: Up to Day 56
Half life
Pharmacokinetics of Neihulizumab - MRT
Time frame: Up to Day 56
Mean Residence Time
Pharmacokinetics of Neihulizumab - Vz and Vss
Time frame: Up to Day 56
Volume of distribution and volume of distribution at steady state
8 secondary outcomes
Adverse Events (AEs)
Time frame: Up to Day 180
To measure the Receptor Occupancy (RO)
Time frame: Up to Day 56
To measure regenerating islet-derived 3-alpha (REG3α) and suppression of tumorigenicity 2 (ST2) as Pharmacodynamics (PD) biomarkers.
Time frame: Up to Day 56
Complete Response (CR)
Time frame: Day 28
Overall Response Rate (ORR)
Time frame: Day 28
Duration of Response
Time frame: Up to Day 180
Non Relapse Mortality (NRM)
Time frame: Day 180
Immunogenicity
Time frame: Up to Day 56
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2023.
- 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.
Shih-Yao Lin, MD, PhD study director
AltruBio, Inc. (formerly AbGenomics International)
Paul Martin, MD principal investigator
Fred Hutchinson Cancer Center
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Neihulizumab (ALTB-168) in people with Steroid-refractory Acute Graft-versus-Host Disease and Treatment-refractory Acute Graft-versus-Host Disease, run by AltruBio Inc. at 13 sites 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?
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: finished, as of the sponsor's last update on 19 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites 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?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Pharmacokinetics of Neihulizumab - AUC (time frame: Up to Day 56). The study lists 7 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 19 Jan 2023.