Diagnoses That Require Stem Cell TransplantPhase 1/2NCT02167451

Maraviroc as GVHD Prophylaxis in Transplant Recipients

A Phase 1/2 study testing Maraviroc in people with Diagnoses That Require Stem Cell Transplant and Graft Versus Host Disease (GVHD), run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

The purpose is to determine if the addition of Maraviroc to a standard transplant regimen will reduce the incidence of graft versus host disease in children and young adults after a stem cell transplant. Read more about this study

Who is it for?: Ages 5 to 40 years
Conditions the study is for: Diagnoses That Require Stem Cell Transplant, Graft Versus Host Disease (GVHD)
What is being tested?: Maraviroc
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 31 participants
Where: 1 siteUnited States
Study dates: Jul 2014to Sep 2018
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 5 to 40 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Ages 5 years and </= 40 years
All diagnoses
Peripheral blood stem cells, marrow or cord blood
All conditioning regimens
Patient must be planned to receive a calcineurin inhibitor (cyclosporine or tacrolimus) together with steroid, methotrexate or mycophenolate mofetil as GVHD prophylaxis.

Who cannot join

Documented anaphylaxis to Maraviroc
Ex vivo T-cell (type of white blood cell) depleted grafts
Abnormal Alanine Aminotransferase (ALT) (>/=10X ULN) on day -3. (Assessed at study enrollment and confirmed again prior to the first dose of maraviroc.)

Trial locations and contacts

1 site in 1 country.

United States1 site

Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229

Other trials in United States

About this study

Official title: Maraviroc as Graft Versus Host Disease Prophylaxis in Pediatric and Adult Stem Cell Transplant Recipients

Read the detailed description

In the first stage, drug levels will be obtained to establish appropriate dosing. In the second stage of the study the investigators will study the effects of using Maraviroc in these patients.

Conditions: Diagnoses That Require Stem Cell Transplant, Graft Versus Host Disease (GVHD)
Being tested: Maraviroc
Keywords: stem cell transplant, GVHD prevention
Medical subject headings: Graft vs Host Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

MaravirocDrug
Also called Selzentry

Study groups in NCT02167451
Study group	Role	What they receive
Maraviroc	Experimental	Maraviroc administration will start on day -3 and will end on day +30 after stell cell transplant, making the total number of days of drug administration 34 days. Maraviroc will be administered twice daily orally or via enteral tube. Dosing of Maraviroc will be based on body surface area (starting with 100mg twice a day for BSA of 0.2 and up to 300mg twice a day for BSA greater than 1.73). Receives: Drug: Maraviroc

What is being measured

The primary outcome is the main question the study is designed to answer.

Feasibility of Maraviroc
The ability to administer twice daily oral maraviroc in children and adults undergoing stem cell transplant in addition to routine standard graft versus host disease prophylaxis.
Measured: Up to day +100
GVHD Incidence
Incidence of GVHD by day+100
Measured: By day +100
Area Under The Concentration-Time Curve (AUC) of Maraviroc
pK target >100ng/ml at day zero at the following time points : before the dose and 1, 2, 4, 6, 8, and 12 hours after maraviroc administration
Measured: Day 0
Incidence of Visceral GVHD
determine the number of patients who develop visceral GVHD by day+100
Measured: day+100
Area Under The Concentration-Time Curve (AUC) of Maraviroc
pK target >100ng/ml at day 10 at the following time points : before the dose and 1, 2, 4, 6, 8, and 12 hours after maraviroc administration
Measured: Day 10

Secondary outcomes (7)

Overall Survival
Overall survival for patients who were enrolled and received maraviroc
Measured: By day +100
Graft Failure
Failure to engraft and loss of graft.
Measured: By day +100
Primary Disease Relapse
Measured: By day +100
Toxicities
Incidence of toxicities due to drug
Measured: Up to day +100
Infectious Complications
Infections complications which include asymptomatic viremias for EBV, Adenovirus, CMV, and/or viral disease, bacterial and fungal infections as documented by blood cultures.
Measured: Up to day +100
Time to Neutrophil
Neutrophil engraftment is defined as the first of three consecutive measurements of ANC>500mcL over 3 or more days.
Measured: Up to day +100
Time to Platelet Engraftment
Time to achieve platelets count of 20,000 without transfusions
Measured: days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jul 2014Sep 2018

First posted19 Jun 2014 (estimated)
Study startJul 2014
Primary completionSep 2018
Study completionSep 2018

Why it stopped: slow recruitment and no further drug supply

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Diagnoses That Require Stem Cell TransplantStudies of the same condition, open and under way.
More trials run by Children's Hospital Medical Center, CincinnatiThe 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 1/2
Primary purpose: Prevention
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 31 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02167451
Sponsor's protocol ID: 2014-1221
Status last verified: Feb 2020
First submitted: 13 Jun 2014
Last updated: 10 Mar 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Study at a glance

From the registry record, last updated by the sponsor on 10 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Maraviroc as Graft Versus Host Disease Prophylaxis in Pediatric and Adult Stem Cell Transplant Recipients
ClinicalTrials.gov ID: NCT02167451
Acronym / sponsor ID: 2014-1221
Conditions: Diagnoses That Require Stem Cell Transplant, Graft Versus Host Disease (GVHD)
Keywords: stem cell transplant, GVHD prevention
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: slow recruitment and no further drug supply
Enrolment: 31 participants (actual)
Lead sponsor: Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
Study start: Jul 2014
Primary completion: Sep 2018 (actual)
Study completion: Sep 2018 (actual)
First posted: 19 Jun 2014 (estimated)
Results: Posted 10 Mar 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

In the first stage, drug levels will be obtained to establish appropriate dosing. In the second stage of the study the investigators will study the effects of using Maraviroc in these patients.

Study design

Primary purpose: Prevention
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Maraviroc	Experimental	Drug: MaravirocMaraviroc administration will start on day -3 and will end on day +30 after stell cell transplant, making the total number of days of drug administration 34 days. Maraviroc will be administered twice daily orally or via enteral tube. Dosing of Maraviroc will be based on body surface area (starting with 100mg twice a day for BSA of 0.2 and up to 300mg twice a day for BSA greater than 1.73).

Who may qualify

Ages: 5 to 40 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (5 listed)

Ages 5 years and </= 40 years
All diagnoses
Peripheral blood stem cells, marrow or cord blood
All conditioning regimens
Patient must be planned to receive a calcineurin inhibitor (cyclosporine or tacrolimus) together with steroid, methotrexate or mycophenolate mofetil as GVHD prophylaxis.

Key exclusion criteria (3 listed)

Documented anaphylaxis to Maraviroc
Ex vivo T-cell (type of white blood cell) depleted grafts
Abnormal Alanine Aminotransferase (ALT) (>/=10X ULN) on day -3. (Assessed at study enrollment and confirmed again prior to the first dose of maraviroc.)

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
MaravirocAlso: Selzentry	Drug	Maraviroc

Primary outcome

Feasibility of Maraviroc
Time frame: Up to day +100
The ability to administer twice daily oral maraviroc in children and adults undergoing stem cell transplant in addition to routine standard graft versus host disease prophylaxis.
GVHD Incidence
Time frame: By day +100
Incidence of GVHD by day+100
Area Under The Concentration-Time Curve (AUC) of Maraviroc
Time frame: Day 0
pK target >100ng/ml at day zero at the following time points : before the dose and 1, 2, 4, 6, 8, and 12 hours after maraviroc administration
Incidence of Visceral GVHD
Time frame: day+100
determine the number of patients who develop visceral GVHD by day+100
Area Under The Concentration-Time Curve (AUC) of Maraviroc
Time frame: Day 10
pK target >100ng/ml at day 10 at the following time points : before the dose and 1, 2, 4, 6, 8, and 12 hours after maraviroc administration

7 secondary outcomes

Overall Survival
Time frame: By day +100
Graft Failure
Time frame: By day +100
Primary Disease Relapse
Time frame: By day +100
Toxicities
Time frame: Up to day +100
Infectious Complications
Time frame: Up to day +100
Time to Neutrophil
Time frame: Up to day +100
Time to Platelet Engraftment
Time frame: days

Results and safety

Results posted to the registry on 10 Mar 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Maraviroc	31	30

Primary outcome: Feasibility of Maraviroc

Time frame: Up to day +100. Count of participants. Unit: Participants

Measure	Maraviroc n = 31
Value	23

Primary outcome: GVHD Incidence

Time frame: By day +100. Count of participants. Unit: Participants

Measure	Maraviroc n = 31
Value	11

Primary outcome: Area Under The Concentration-Time Curve (AUC) of Maraviroc

Time frame: Day 0. Mean (Standard Deviation). Unit: hour *ng/mL

Measure	Maraviroc n = 12
Value	4805 (3265)

Primary outcome: Incidence of Visceral GVHD

Time frame: day+100. Count of participants. Unit: Participants

Measure	Maraviroc n = 31
Value	6

Population analysed: all patients enrolled on trial

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: day+ 100 after BMT or 30 days after last maraviroc administration if patients withdrew from study. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Maraviroc	11 of 31 (35.5%)	16 of 31 (51.6%)	4 of 31

Limitations and caveats (from the sponsor): early termination leading to small number of subjects analyzed.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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.

Pooja Khandelwal, MD principal investigator

Children's Hospital Medical Center, Cincinnati

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 5 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 10 Mar 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 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 combined 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.

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?

Yes. Results are posted in the registry (first posted 10 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Feasibility of Maraviroc (time frame: Up to day +100). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02167451.

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 10 Mar 2020.