X4P-001 for WHIM Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
St. Vincent's Hospital, Fitzroy, Australia
WHIM Syndrome+1 More
X4P-001 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug can be used to treat WHIM syndrome.

See full description

Eligible Conditions

  • WHIM Syndrome

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for WHIM Syndrome

Study Objectives

This trial is evaluating whether X4P-001 will improve 1 primary outcome in patients with WHIM Syndrome. Measurement will happen over the course of 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.

Week 5
Mean AUCANC and/or AUCALC

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for WHIM Syndrome

Trial Design

1 Treatment Group

X4P-001
1 of 1
Experimental Treatment

This trial requires 15 total participants across 1 different treatment group

This trial involves a single treatment. X4P-001 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

X4P-001
Drug
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AMD-070
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 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
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 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 for reporting.

Closest Location

University of Washingington Medical Center - Seattle, WA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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.
Has confirmed ANC less than or equal to (≤) 400/µL or ALC ≤650/µL or both.
Agree to use effective contraception.
Be willing and able to comply with this protocol.

Patient Q&A Section

What are common treatments for syndrome?

"There is no cure for syndrome, however treatment revolves around the symptoms of the condition. There are a number of treatments which have been shown to be useful in some people. In particular, cognitive behavioural therapy (CBT) is used as treatment for most people with syndrome. CBT addresses the core symptom of inattention, which is the focus of this paper.\n" - Anonymous Online Contributor

Unverified Answer

What are the signs of syndrome?

"The symptom group's inclusion in a syndrome with specific signs is consistent with clinical evidence. This syndromic grouping of symptoms seems to be specific for a variety of health conditions and may permit syndromic syndromes to be targeted therapeutically." - Anonymous Online Contributor

Unverified Answer

Can syndrome be cured?

"At least for some patients, syndrome can be cured at certain points in time. This is also true for patients with other diagnoses of chronic diseases. There is a possibility that it might happen with symptoms of heart disease and asthma." - Anonymous Online Contributor

Unverified Answer

What is syndrome?

"In most cases, patients do not describe a specific pattern of symptoms, such as a clear-cut sequence of diseases with associated symptoms. Rather, doctors and patients focus on the symptoms themselves (which can include many types of disorders such as fatigue, pain, weakness, nausea or an array of others). Thus, the syndrome is best used to describe specific symptoms (e.g. “I wake at night not remembering,” “The left eye is dim,” etc.). syndrome\n\nA diagnostic checklist is available to help doctors make a diagnosis.\n\nThere are several screening tools which measure several features of depression, but none are standard in use." - Anonymous Online Contributor

Unverified Answer

What causes syndrome?

"Many syndromes are a complex of diseases caused by disturbances in one or more systems. The differential diagnosis of disorders in systemic diseases requires good clinical and laboratory observations and information on the clinical history of the patient." - Anonymous Online Contributor

Unverified Answer

How many people get syndrome a year in the United States?

"In any given year, over 3 million men are bothered by sex symbol syndrome. There are more women bothered by the syndrome than there are men bothered. On average, women are affected more often than men, though this difference is relatively small. For both genders, the syndrome afflicts men and women, and most often manifests in their mid- to late-thirties. Syndromes are especially common in women with high levels of mental fitness--in this group, more women are bothered by the syndrome than men. In women with low mental fitness, more men are bothered by the syndrome. There appears to be a pattern among women; although the syndrome afflicts them more often, younger women are more bothered than older women." - Anonymous Online Contributor

Unverified Answer

What does x4p-001 usually treat?

"X4p-001 is a medicine that is marketed in the United States that is typically used to treat X-linked ichthyosis. In these reports, there were very few cases in which the drug caused [any adverse effects]. The main side effects of using X4p-001 have been a rare skin condition called erythroderma, which can be temporary and disappear when the patient's skin adjusts to the drug. Also, some people might suffer [abnormal itchiness from itchiness drug side effects] and might [not react to a dose adjustment of X4p-001]. Most side effects of X4p-001 are minor and tend to disappear on their own." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in x4p-001 for therapeutic use?

"X4p-001 has proved to be a versatile reagent in the field of drug discovery. In this context, X4p001 was successful in the identification of a new group of protein kinase (PKC) inhibitors which exhibit activity and selectivity in both in vitro and in vivo studies. These pharmacokinetic properties have been shown to be important in the treatment of human tumors. Based on these observations, we conclude that X4p-001 has potential therapeutic value for treatment of a variety of tumors and possibly for treatment of metastatic tumors of the breast and other organs." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating syndrome?

"There have been many treatments for patients who suffer with [DSM-5] chronic fatigue syndrome, but more studies need to be done on the new symptoms, the new treatments, and the possible ways of treating syndromes in the future." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of x4p-001?

"X4p-001 showed clinical toxicity; its safety was assessed by evaluating its safety profile in trials conducted in patients with cancer or HIV and in healthy volunteers. In clinical trials, common side effects of x4p-001 included (1) fatigue; (2) nausea and vomiting; (3) loss of appetite; (4) constipation; (5) loss of hair; (6) rash; and (7) headache. Overall, the frequency of these common side effects was <5%. In a phase I study conducted in patients with HIV, the drug was well tolerated." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets syndrome?

"Most diagnosed patients are 65 years old or older. This is probably due to late diagnosis and the lack of awareness among health care providers that syndrome might be a cause for many medical problems." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of syndrome?

"The cause of syndrome depends on which type of syndrome is present. The main causes of each type of syndrome are [[lung cancer](https://www.withpower.com/clinical-trials/lung-cancer), lung disease, and [lung disease with lung cancer]], which are mainly [seen in the case of pneumonia, bronchiectasis, and fibrosis syndromes. Because of the rarity of this syndrome, it is hard to figure out what the cause is. The exact mechanisms by which the syndrome is caused is yet to be worked out, but it is thought to originate from the immune system." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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