WHIM SyndromePhase 2NCT03005327

A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

A Phase 2 study testing X4P-001 in people with WHIM Syndrome, run by X4 Pharmaceuticals at 2 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: WHIM Syndrome
What is being tested?: X4P-001
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 8 participants
Where: 2 sitesin 2 countries
Study dates: Dec 2016to 16 Jun 2022
Phase: Phase 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 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Participants with a clinical diagnosis of WHIM syndrome must meet all of the following criteria to be eligible for study participation:
Be at least 18 years of age.
Has signed the current approved informed consent form.
Has a genotype-confirmed mutation of chemokine receptor type 4 (CXCR4) consistent with WHIM syndrome.
Agree to use effective contraception.
Be willing and able to comply with this protocol.
Has confirmed ANC less than or equal to (≤) 400/µL or ALC ≤650/µL or both.

Who cannot join

Participants with any of the following will be excluded from participation in the study:
Has known systemic hypersensitivity to the mavorixafor drug substance or its inactive ingredients.
Is pregnant or nursing.
Has a known history of a positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immunodeficiency syndrome (AIDS).
Has, at Screening, laboratory tests meeting one or more of the following criteria:
A positive antibody test for hepatitis C virus (HCV), unless documented to have no detectable viral load on 2 independent samples.
A positive test for hepatitis B surface antigen (HBsAg).
Has any medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the participant, or may preclude the participant's successful completion of the clinical study.

Trial locations and contacts

2 sites in 2 countries.

Country

City, postcode or hospital

2 sites in 2 countries

United States1 site

University of Washington Medical Center
Seattle, Washington, 98195

Other trials in United States

Australia1 site

St. Vincent's Hospital
Fitzroy, Victoria, 3065

Other trials in Australia

About this study

Official title: A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome

This is a Phase 2 study with an initial 24-week Treatment Period and an Extension Phase. The primary objectives of this Phase 2 study are to determine the safety, tolerability, and dose selection of mavorixafor in participants with WHIM syndrome. Participants may continue treatment in an Extension Phase, if regionally applicable, until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the Sponsor for any reason.

Conditions: WHIM Syndrome
Being tested: X4P-001
Medical subject headings: WHIM syndrome

What participants receive

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

X4P-001Drug
Also called AMD11070
Mavorixafor will be provided as either 25 mg or 100 mg capsules.

Study groups in NCT03005327
Study group	Role	What they receive
X4P-001	Experimental	Initial Treatment Phase: Participants will initiate treatment with mavorixafor at 50 milligrams (mg) once daily (QD) orally or a higher dose, with potential escalation based on area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values to a maximum total daily dose of 400 mg. Participants are expected to receive treatment for 24 weeks in the initial Treatment Period or until development of a treatment-limiting toxicity (TLT). Extension Phase: All participants will receive mavorixafor; the dose will not exceed 400 mg. In the Extension Phase, treatment may continue until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the sponsor. Receives: Drug: X4P-001

What is being measured

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

Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC)
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute neutrophil count (ANC) clinically meaningful threshold was defined as ANC ≥ 600/microliter (μL).
Measured: Time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
All Visits: Average Per-Participant Value of the AUCANC
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ANC clinically meaningful threshold was defined as ANC ≥ 600/μL.
Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCANC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
Measured: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Lymphocyte Count (AUCALC)
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute lymphocyte count (ALC) clinically meaningful threshold was defined as ALC ≥ 1000/μL.
Measured: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
All Visits: Average Per-Participant Value of the AUCALC
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ALC clinically meaningful threshold was defined as ALC ≥ 1000/μL.
Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCALC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
Measured: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and did not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as any AE that began or worsened in severity or frequency on or after the start of study drug through 10 days after the last dose of the study drug. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
Measured: From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days)

Timeline

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

Dec 201616 Jun 2022

First posted29 Dec 2016 (estimated)
Study startDec 2016
Primary completion16 Jun 2022
Study completion16 Jun 2022

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for WHIM SyndromeStudies 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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 8 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03005327
Sponsor's protocol ID: X4P-001-MKKA
Other IDs: 2016-005028-26, 2016
Status last verified: Oct 2024
First submitted: 20 Dec 2016
Last updated: 30 Oct 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Dale DC, Firkin F, Bolyard AA, Kelley M, Makaryan V, Gorelick KJ, Ebrahim T, Garg V, Tang W, Jiang H, Skerlj R, Beaussant Cohen S. Results of a phase 2 trial of an oral CXCR4 antagonist, mavorixafor, for treatment of WHIM syndrome. Blood. 2020 Dec 24;136(26):2994-3003. doi: 10.1182/blood.2020007197.
PubMed 32870250

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1/2
Diagnostic Value of CXCR4-targeted PET/CT in ACTH-dependent and Independent Cushing's Syndrome
Cushing s Syndrome
Enrolling by invitationPhase 1/2
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM
WHIM, Warts
RecruitingPhase 3
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
Neutropenia
RecruitingObservational study
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
Shwachman-Diamond Syndrome, SDS
RecruitingObservational study
The Role of Cxcr4Hi neutrOPhils in InflueNza
Influenza, Acute Respiratory Distress Syndrome

All WHIM Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome
ClinicalTrials.gov ID: NCT03005327
Acronym / sponsor ID: X4P-001-MKKA
Conditions: WHIM Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 8 participants (actual)
Lead sponsor: X4 PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Dec 2016 (actual)
Primary completion: 16 Jun 2022 (actual)
Study completion: 16 Jun 2022 (actual)
First posted: 29 Dec 2016 (estimated)
Results: Posted 30 Oct 2024

Clinical summary

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

Study design

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

Arm	Type	Receives
X4P-001	Experimental	Drug: X4P-001Initial Treatment Phase: Participants will initiate treatment with mavorixafor at 50 milligrams (mg) once daily (QD) orally or a higher dose, with potential escalation based on area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values to a maximum total daily dose of 400 mg. Participants are expected to receive treatment for 24 weeks in the initial Treatment Period or until development of a treatment-limiting toxicity (TLT). Extension Phase: All participants will receive mavorixafor; the dose will not exceed 400 mg. In the Extension Phase, treatment may continue until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the sponsor.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Participants with a clinical diagnosis of WHIM syndrome must meet all of the following criteria to be eligible for study participation:
Be at least 18 years of age.
Has signed the current approved informed consent form.
Has a genotype-confirmed mutation of chemokine receptor type 4 (CXCR4) consistent with WHIM syndrome.
Agree to use effective contraception.

Key exclusion criteria (8 listed)

Has known systemic hypersensitivity to the mavorixafor drug substance or its inactive ingredients.
Is pregnant or nursing.
Has a known history of a positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immunodeficiency syndrome (AIDS).
Has, at Screening, laboratory tests meeting one or more of the following criteria:
Has any medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the participant, or may preclude the participant's successful completion of the clinical study.

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
X4P-001Also: AMD11070Mavorixafor will be provided as either 25 mg or 100 mg capsules.	Drug	X4P-001

Primary outcome

Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC)
Time frame: Time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute neutrophil count (ANC) clinically meaningful threshold was defined as ANC ≥ 600/microliter (μL).
All Visits: Average Per-Participant Value of the AUCANC
Time frame: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ANC clinically meaningful threshold was defined as ANC ≥ 600/μL. Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCANC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Lymphocyte Count (AUCALC)
Time frame: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute lymphocyte count (ALC) clinically meaningful threshold was defined as ALC ≥ 1000/μL.
All Visits: Average Per-Participant Value of the AUCALC
Time frame: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ALC clinically meaningful threshold was defined as ALC ≥ 1000/μL. Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCALC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days)
An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and did not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as any AE that began or worsened in severity or frequency on or after the start of study drug through 10 days after the last dose of the study drug. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.

Results and safety

Results posted to the registry on 30 Oct 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
X4P-001 50 to up to 400 mg/kg	2	2
X4P-001 100 to up to 400 mg/kg	2	1
X4P-001 200 to up to 400 mg/kg	2	1
X4P-001 300 to up to 400 mg/kg	2	2

Primary outcome: Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC)

Time frame: Time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21. Mean (Standard Deviation). Unit: cells*hour/μL

Measure	X4P-001 50 mg n = 2	X4P-001 100 mg n = 2	X4P-001 150 mg n = 2	X4P-001 200 mg n = 2	X4P-001 300 mg n = 4	X4P-001 400 mg n = 0	X4P-001 300/400 mg n = 4
Week 5	-12574.63 (156.094)	-6206.75 (4238.280)	–	-6949.58	24175.00 (777.817)	–	24175.00 (777.817)
Week 13	–	-11291.96 (1509.496)	–	-7575.21 (2701.443)	-2602.50	–	-2602.50
Week 21	–	–	-8723.00 (2695.962)	–	58118.02 (112851.505)	–	58118.02 (112851.505)

Population analysed: Overall Number of Participants Analyzed=number of participants in safety population (all participants who received at least 1 dose of study medication). 'Number analyzed'=number of participants with data above clinically meaningful thresholds at specified timepoints. Participants included in 1 category may be different than participants in other categories. Data for this Outcome Measure were collected by dose level; therefore, participants may have been included in more than 1 arm (dose level).

Primary outcome: All Visits: Average Per-Participant Value of the AUCANC

Time frame: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21. Mean (Standard Deviation). Unit: cells*hour/μL

Measure	X4P-001 300/400 mg n = 5
Value	27477.00 (55792.377)

Population analysed: The Safety Population included all participants who received at least 1 dose of the study medication (mavorixafor). Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Primary outcome: Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Lymphocyte Count (AUCALC)

Time frame: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21. Mean (Standard Deviation). Unit: cells*hour/μL

Measure	X4P-001 50 mg n = 2	X4P-001 100 mg n = 2	X4P-001 150 mg n = 2	X4P-001 200 mg n = 2	X4P-001 300 mg n = 4	X4P-001 400 mg n = 0	X4P-001 300/400 mg n = 4
Week 5	-5839.54 (6485.407)	6978.25 (7435.935)	–	4783.33	21837.50 (24236.085)	–	21837.50 (24236.085)
Week 13	–	-1006.50 (10546.262)	–	7023.83 (1603.482)	2862.50	–	2862.50
Week 21	–	–	-2216.17 (8285.642)	–	15331.35 (21250.202)	–	15331.35 (21250.202)

Primary outcome: All Visits: Average Per-Participant Value of the AUCALC

Time frame: Time 0 (-15 min pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21. Mean (Standard Deviation). Unit: cells*hour/μL

Measure	X4P-001 300/400 mg n = 5
Value	14232.17 (16789.700)

Adverse events

Time frame: From first dose of study drug through 10 days after the last dose of the study drug (Maximum exposure: 1712 days). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
X4P-001 50 mg	0 of 2 (0%)	2 of 2 (100%)	0 of 2
X4P-001 100 mg	0 of 4 (0%)	2 of 4 (50%)	0 of 4
X4P-001 150 mg	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
X4P-001 200 mg	0 of 4 (0%)	2 of 4 (50%)	0 of 4
X4P-001 300 mg	2 of 7 (28.6%)	6 of 7 (85.7%)	0 of 7
X4P-001 400 mg	1 of 5 (20%)	3 of 5 (60%)	0 of 5
Overall Participants	3 of 8 (37.5%)	7 of 8 (87.5%)	0 of 8

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 32870250Dale DC, Firkin F, Bolyard AA, Kelley M, Makaryan V, Gorelick KJ, Ebrahim T, Garg V, Tang W, Jiang H, Skerlj R, Beaussant Cohen S. Results of a phase 2 trial of an oral CXCR4 antagonist, mavorixafor, for treatment of WHIM syndrome. Blood. 2020 Dec 24;136(26):2994-3003. doi: 10.1182/blood.2020007197.

Related trials for WHIM Syndrome

RecruitingPhase 1/2
Diagnostic Value of CXCR4-targeted PET/CT in ACTH-dependent and Independent Cushing's Syndrome
Cushing s Syndrome
Enrolling by invitationPhase 1/2
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM
WHIM, Warts
RecruitingPhase 3
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
Neutropenia
RecruitingObservational study
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
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The Role of Cxcr4Hi neutrOPhils in InflueNza
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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.

The record lists no contact. Use the official record to reach the sponsor.

2 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing X4P-001 in people with WHIM Syndrome, run by X4 Pharmaceuticals at 2 sites in 2 countries.

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 7 inclusion and 8 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: finished, as of the sponsor's last update on 30 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States, Australia. 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 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 30 Oct 2024). 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?

Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) (time frame: Time 0 (-15 minutes [min] pre-dose), 30, 60, and 90 min (each ± 5 min) and 2, 3, 4, 8, 12, 16, and 24 hours (each ±15 min) at Weeks 5, 13, and 21). 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 NCT03005327, which also lists 1 linked publication.

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 30 Oct 2024.