X4P-001-103 A Study of Mavorixafor in Patients with WHIM Syndrome
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension
IRAS ID
272576
Contact name
Sorena Kiani-Alikhan
Contact email
Sponsor organisation
X4 Pharmaceuticals Incorporated
Eudract number
2019-001153-10
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
129092, FDA IND Number
Duration of Study in the UK
2 years, 0 months, 18 days
Research summary
Research Summary
This study is being done to learn about an investigational drug called mavorixafor. The condition being studied is warts, hypogammaglobulinaemia, infections, and myelokathexis (WHIM) Syndrome, a disease that affects the body’s ability to fight off infections. Mavorixafor is a drug that is intended to release certain types of white blood cells (neutrophils and lymphocytes) from the bone marrow. The more of these cells existent in the blood, the better the body can fight infection.
The purpose of the study is to test mavorixafor when it is given to patients diagnosed with WHIM Syndrome. The goals of the study are to determine whether mavorixafor is safe, how it is tolerated, and what effect it may have on the body and WHIM Syndrome.Summary of Results
Why was this research needed?
WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome is a disease that affects the body’s ability to fight off infections. In WHIM syndrome, the body’s immune system does not function properly and the levels of certain white blood cells (cells that protect the body against infections) drop, increasing the risk for infections.
When this clinical study was done, there were no approved medicines that targeted the main cause of the disease. In this trial, a drug called mavorixafor was studied for WHIM syndrome. Mavorixafor is designed to release certain types of white blood cells (neutrophils and lymphocytes) from the bone marrow.
Who took part in the study?
Researchers asked for the help of people with WHIM syndrome. There were 31 participants in the study, including 13 men and 18 women between 12 and 72 years old. The participants were from:
• US (6 participants)
• Spain (4 participants)
• France, Italy, Netherlands, Russia, and UK (3 participants each)
• Australia (2 participants)
• Austria, Denmark, Israel, and Korea (1 participant each).
What treatments did the participants take?
Participants took either mavorixafor or placebo in this study. The placebo in this study matches the look of mavorixafor but contains no active ingredient. Both treatments were in capsule form taken by mouth What happened during the study?
This study had 2 parts: the first part is called Randomized Period (RP), and the second part is called Open-Label Period (OLP).
During RP, participants were assigned at random to receive mavorixafor or placebo. Participants had a 50-50 chance of getting either treatment. None of the participants, the study doctors, or other study staff knew which treatment the participants were taking. Participants took their assigned treatment once a day for 52 weeks. Participants also had a final health check about 4 weeks after their last dose. Of the 31 participants who joined RP, 14 took mavorixafor and 17 took the placebo.
Eligible participants from RP were offered the opportunity to take mavorixafor in the OLP. During this part, the participants, the study doctors, and other study staff knew that all participants were taking mavorixafor. Participants who continued to take part in the OLP were in the study until mavorixafor became available in the market, the researchers ended the study, or the participants decided to stop taking part in the study, whichever came first.
A total of 27 participants continued to the OLP (11 participants took mavorixafor and 16 participants took the placebo during the RP).
The study ran for about 6 years, from November 2019 to December 2025.
What were the main goals of the study?
The main goal of the study was to learn whether mavorixafor helps to increase neutrophil levels in participants with WHIM syndrome. Researchers calculated the length of time that the participants’ neutrophil levels remained at the ideal level (500 cells/microliter of blood or higher) during the RP.
Another main goal of the study was to learn about any side effects the participants had during the study.
What were the results?
Did participants who took mavorixafor maintain ideal neutrophil levels for a longer period of time?
Yes. Participants who took mavorixafor were able to maintain the ideal neutrophil levels for a longer period of time than those who took the placebo.
Did participants have any side effects?
The study doctors kept track of any new or worsening health issues the participants had after they started treatment in the study. In research studies, these health issues are called adverse events (AEs). The study doctors made note of any AEs that they thought might be related to the study treatment. These AEs are called side effects in this summary. A side effect is considered “serious” when it is life threatening or requires hospital care.
During RP, of the 31 participants:
• none had a serious side effect or stopped treatment due to a side effect.
• 10 participants (32%) had at least 1 side effect that was not serious.
These nonserious side effects included:
• skin conditions (like rashes, dry skin, itching)
• nervous system conditions (like headache*, dizziness)
• stomach problems (like feeling queasy, vomiting, indigestion)
• infection*
• dry eye
• kidney problem
• product issues (product tastes bad)
*Side effect happened in placebo group only.
None of these side effects occurred in more than 1 participant.
During OLP, of the 27 participants:
• 1 participant (4%) had a serious side effect of “oligoarthritis”, a type of joint inflammation. This participant stopped taking mavorixafor because of this serious side effect.
• 14 participants (52%) had at least 1 side effect that was not serious.
The most common nonserious side effects were diarrhea and nausea. These were the only nonserious side effects that happened to at least 10% of participants in OLP. There were other side effects, but they happened to fewer participants.
None of the participants died during the entire study.REC name
London - Central Research Ethics Committee
REC reference
20/LO/1075
Date of REC Opinion
21 Dec 2020
REC opinion
Further Information Favourable Opinion