Candidiasis, Chronic MucocutaneousPhase 2CAMBNCT02629419
CAMB/MAT2203 in Patients With Mucocutaneous Candidiasis
A Phase 2 study testing Amphotericin B in people with Candidiasis, Chronic Mucocutaneous, run by Matinas BioPharma Nanotechnologies, Inc. at 1 site in United States.
Trial summary
This is an open-label, dose-titration trial to study the efficacy, safety, and pharmacokinetics of oral cochleate amphotericin B (CAMB) in the treatment of mucocutaneous candidiasis infections in patients who are refractory or intolerant to standard non intravenous therapies. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Candidiasis, Chronic Mucocutaneous
- What is being tested?
- Amphotericin B
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Sep 2016to 6 Aug 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have a clinical diagnosis of at least one of the following:
- Persistent oropharyngeal candidiasis (OPC) for greater than or equal to 5 days documented on at least one occasion by potassium hydroxide (KOH) test or fungal stain and confirmed by mycological culture to be azole resistant within the previous 6 months and/or intolerance to standard non intravenous therapies or lack of improvement or worsening of OPC after receipt of appropriately dosed oral azole therapy.
- Esophageal candidiasis (EC) associated with clinical symptoms of retrosternal pain, odynophagia, and/or pain with swallowing and documented by esophageal biopsy or visualization with culture documenting azole resistance within the previous 6 months and/or intolerance to standard non-intravenous therapies or lack of improvement or worsening of EC after appropriately dosed azole therapy.
- Persistent vulvovaginal candidiasis (VVC) for greater than or equal to 5 days as documented by presence of vaginal symptoms and a positive wet mount showing Candida structures and confirmed by a vaginal culture positive for Candida with azole resistance within the previous 6 months and/or intolerance to standard non intravenous therapies or lack of improvement or worsening of VVC after appropriately dosed azole therapy.
- Patient is expected to survive for greater than or equal to 6 months.
- Willing to have samples stored for future research.
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- Agree to use highly effective contraception.
- Contraception: Because the effects of CAMB on the developing human fetus are unknown, sexually active patients of childbearing potential must agree to use highly effective contraception as outlined below before study entry and for the duration of study participation. Females of childbearing potential must have a negative pregnancy test result before receiving CAMB. During the course of the study, if a patient becomes pregnant or suspects they are pregnant, then they should inform the study staff and their primary care physician immediately. Acceptable forms of contraception are:
- Intrauterine device (IUD) or equivalent.
- Hormonal contraceptives (eg, consistent, timely and continuous use of contraceptive pill, patch, ring, implant, or injection that has reached full efficacy prior to dosing). If the patient uses contraceptive pill, patch, or ring, then a barrier method (eg, male/female condom, cap, or diaphragm plus spermicide) must also be used at the time of potentially reproductive sexual activity.
- Be in a stable, long-term monogamous relationship, per principal investigator (PI) assessment, with a partner that does not pose any potential pregnancy risk, eg, has undergone a vasectomy at least 6 months prior to first dose of study agent or is of the same sex as the patient.
- Have had a hysterectomy and/or a bilateral tubal ligation or both ovaries removed.
Who cannot join
- Allergy to any amphotericin B (AMB) product or any component of CAMB (eg, phosphatidylserine)
- Have evidence of systemic fungal infections requiring intravenous antifungal therapy
- Pregnant or nursing women, and women intending to become pregnant during the study period
- Had a concomitant medical condition that could interfere with study drug evaluation or that is a contraindication to the proposed investigational treatment based upon known agent safety profile or toxicities.
- Had any of the following laboratory abnormalities at the screening visit:
- Alanine Transaminase (ALT), Aspartate Transaminase (AST) and Alkaline phosphatase (ALP) > 2.5 times the upper limit of normal (ULN).
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- Total bilirubin level > 2.5 times the ULN
- Serum creatinine level > 2 times the ULN
- Absolute neutrophil count less than 500 cells/microliter
- Potassium level less than 3.5 mmol/L
- Exposure to any investigational agent within 4 weeks prior to Day 0 (Baseline).
- Current or recent history (past 12 months) of drug or alcohol abuse.
- Use of intravenous AMB products within 1-week of start of study drug administration
- Use of non-intravenous AMB products (such as oral AMB swishes) within 72 hours prior to start of study drug administration
- Any other condition the investigator believes would interfere with the patient s ability to provide informed consent, comply with study instructions, or which might confound the interpretation of the study results or put the subject at undue risk.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Allergy and Infectious Disease (NIAID)
Bethesda, Maryland, 20892
About this study
Official title: A Phase 2a Efficacy, Safety, Tolerability, and PK Study of Encochleated Amphotericin B (CAMB/MAT2203) in Patients With Mucocutaneous Candidiasis Who Are Refractory or Intolerant to Standard Non-Intravenous Therapies
This is an open-label, dose-titration trial to study the efficacy, safety, and pharmacokinetics of oral cochleate amphotericin B (CAMB) in the treatment of mucocutaneous candidiasis infections in patients who are refractory or intolerant to standard non intravenous therapies.
Read the detailed description
Patients aged 18 to 75 years with mucocutaneous candidiasis (esophageal, oropharyngeal, or vulvovaginal) who are refractory or intolerant to standard non-intravenous therapies will be enrolled. Patients will initially be treated in a short-term dose titration period, where the dose may be increased in patients that do not respond clinically. Patients who do not respond clinically to the highest dose of drug will discontinue the protocol. Patients that respond to treatment and tolerate the study medication will be eligible to enter a long-term extension (up to 60-months).
- Conditions
- Candidiasis, Chronic Mucocutaneous
- Being tested
- Amphotericin B
- Keywords
- STAT3 deficient Hyper IgE syndrome, Gain of function STAT1 defects, Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED), IL-17/IL-22 autoantibodies from thymoma, Job's Syndrome (Hyperimmunoglobulin E Syndrome, Buckley Syndrome)
- Medical subject headings
- Candidiasis, Chronic Mucocutaneous, Polyendocrinopathies, Autoimmune, Job Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Amphotericin BDrug
Also called Encochleated Amphotericin B, CAMB, MAT2203
Oral lipid nanocrystal formulation of amphotericin B
| Study group | Role | What they receive |
|---|---|---|
| CAMB (Encochleated Amphotericin B) | Experimental | Encochleated Amphotericin B (200 mg, 400 mg, 800 mg) Receives: Drug: Amphotericin B |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Response to Treatment of Mucocutaneous Candidiasis
Number of subjects with a clinical response of clinical cure or improvement. Clinical cure was defined as absence of signs or symptoms of infection.
Clinical improvement was defined as follows:
- OPC or EC: partial resolution defined as greater than or equal to 50% of Baseline signs or symptoms
- VVC: reduction of greater than or equal to 50% of clinical severity score from Baseline Severity of each symptom was graded on a scale from zero (absence of symptoms) to 3 (severe symptoms). The sum of all scores for all symptoms was used as the clinical severity score. Higher scores mean worse outcomes.
Measured: 14-days at highest titrated dose
Secondary outcomes (4)
Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours Postdose
Drug concentration in plasma collected at 0, 1, 2, 4, 8, 10, 12, and 24 hours post-dose
Measured: Single and Multiple Doses (14-days)
Maximum Plasma Concentration (Cmax)
Plasma concentration was measured at 0, 1, 2, 4, 8, 10, 12, and 24 hours post-dose
Measured: Single and Multiple Doses (14-days)
Time to Reach Maximum Plasma Concentration (Tmax)
Plasma collected at 0, 1, 2, 4, 8, 10, 12, and 24 hours post-dose
Measured: Single and Multiple Doses (14-days)
Long-term Adverse Events, Changes in Laboratory Parameters
Incidence of nephrotoxicity, defined as an increase of greater than 100% of baseline serum creatinine.
Incidence of hypokalemia, defined as serum potassium less than or equal to 3mmol/L during or within 3 weeks of completing treatment.
Measured: up to 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2015 (estimated)
- Study start27 Sep 2016
- Primary completion9 Nov 2021
- Study completion6 Aug 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 Candidiasis, Chronic MucocutaneousStudies 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
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Matinas BioPharma Nanotechnologies, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alexandra Freeman, MD, Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02629419
- Sponsor's protocol ID
- MB-70004
- Other IDs
- 16-I-0002
- Acronym
- CAMB
- Status last verified
- Jul 2024
- First submitted
- 16 Nov 2015
- Last updated
- 7 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Desai JV, Urban A, Swaim DZ, Colton B, Kibathi LW, Ferre EMN, Stratton P, Merideth MA, Hunsberger S, Matkovits T, Mannino R, Holland SM, Tramont E, Lionakis MS, Freeman AF. Efficacy of Cochleated Amphotericin B in Mouse and Human Mucocutaneous Candidiasis. Antimicrob Agents Chemother. 2022 Jul 19;66(7):e0030822. doi: 10.1128/aac.00308-22. Epub 2022 Jun 14.
PubMed 35699443
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Efficacy, Safety, Tolerability, and PK Study of Encochleated Amphotericin B (CAMB/MAT2203) in Patients With Mucocutaneous Candidiasis Who Are Refractory or Intolerant to Standard Non-Intravenous Therapies
- ClinicalTrials.gov ID
- NCT02629419
- Acronym / sponsor ID
- CAMB / MB-70004
- Conditions
- Candidiasis, Chronic Mucocutaneous
- Keywords
- STAT3 deficient Hyper IgE syndrome, Gain of function STAT1 defects, Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED), IL-17/IL-22 autoantibodies from thymoma, Job's Syndrome (Hyperimmunoglobulin E Syndrome, Buckley Syndrome)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Matinas BioPharma Nanotechnologies, Inc.Industry (pharma, biotech, devices)
- Study start
- 27 Sep 2016 (actual)
- Primary completion
- 9 Nov 2021 (actual)
- Study completion
- 6 Aug 2022 (actual)
- First posted
- 14 Dec 2015 (estimated)
- Results
- Posted 7 Jun 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, dose-titration trial to study the efficacy, safety, and pharmacokinetics of oral cochleate amphotericin B (CAMB) in the treatment of mucocutaneous candidiasis infections in patients who are refractory or intolerant to standard non intravenous therapies.
Detailed description
Patients aged 18 to 75 years with mucocutaneous candidiasis (esophageal, oropharyngeal, or vulvovaginal) who are refractory or intolerant to standard non-intravenous therapies will be enrolled. Patients will initially be treated in a short-term dose titration period, where the dose may be increased in patients that do not respond clinically. Patients who do not respond clinically to the highest dose of drug will discontinue the protocol. Patients that respond to treatment and tolerate the study medication will be eligible to enter a long-term extension (up to 60-months).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CAMB (Encochleated Amphotericin B) | Experimental | Drug: Amphotericin BEncochleated Amphotericin B (200 mg, 400 mg, 800 mg) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Patients must have a clinical diagnosis of at least one of the following:
- Persistent oropharyngeal candidiasis (OPC) for greater than or equal to 5 days documented on at least one occasion by potassium hydroxide (KOH) test or fungal stain and confirmed by mycological culture to be azole resistant within the previous 6 months and/or intolerance to standard non intravenous therapies or lack of improvement or worsening of OPC after receipt of appropriately dosed oral azole therapy.
- Esophageal candidiasis (EC) associated with clinical symptoms of retrosternal pain, odynophagia, and/or pain with swallowing and documented by esophageal biopsy or visualization with culture documenting azole resistance within the previous 6 months and/or intolerance to standard non-intravenous therapies or lack of improvement or worsening of EC after appropriately dosed azole therapy.
- Persistent vulvovaginal candidiasis (VVC) for greater than or equal to 5 days as documented by presence of vaginal symptoms and a positive wet mount showing Candida structures and confirmed by a vaginal culture positive for Candida with azole resistance within the previous 6 months and/or intolerance to standard non intravenous therapies or lack of improvement or worsening of VVC after appropriately dosed azole therapy.
- Patient is expected to survive for greater than or equal to 6 months.
Key exclusion criteria (15 listed)
- Allergy to any amphotericin B (AMB) product or any component of CAMB (eg, phosphatidylserine)
- Have evidence of systemic fungal infections requiring intravenous antifungal therapy
- Pregnant or nursing women, and women intending to become pregnant during the study period
- Had a concomitant medical condition that could interfere with study drug evaluation or that is a contraindication to the proposed investigational treatment based upon known agent safety profile or toxicities.
- Had any of the following laboratory abnormalities at the screening visit:
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 |
|---|---|---|
| Amphotericin BAlso: Encochleated Amphotericin B, CAMB, MAT2203Oral lipid nanocrystal formulation of amphotericin B | Drug | CAMB (Encochleated Amphotericin B) |
Primary outcome
Clinical Response to Treatment of Mucocutaneous Candidiasis
Time frame: 14-days at highest titrated dose
Number of subjects with a clinical response of clinical cure or improvement. Clinical cure was defined as absence of signs or symptoms of infection. Clinical improvement was defined as follows: * OPC or EC: partial resolution defined as greater than or equal to 50% of Baseline signs or symptoms * VVC: reduction of greater than or equal to 50% of clinical severity score from Baseline Severity of each symptom was graded on a scale from zero (absence of symptoms) to 3 (severe symptoms). The sum of all scores for all symptoms was used as the clinical severity score. Higher scores mean worse outcomes.
4 secondary outcomes
Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours Postdose
Time frame: Single and Multiple Doses (14-days)
Maximum Plasma Concentration (Cmax)
Time frame: Single and Multiple Doses (14-days)
Time to Reach Maximum Plasma Concentration (Tmax)
Time frame: Single and Multiple Doses (14-days)
Long-term Adverse Events, Changes in Laboratory Parameters
Time frame: up to 60 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35699443Desai JV, Urban A, Swaim DZ, Colton B, Kibathi LW, Ferre EMN, Stratton P, Merideth MA, Hunsberger S, Matkovits T, Mannino R, Holland SM, Tramont E, Lionakis MS, Freeman AF. Efficacy of Cochleated Amphotericin B in Mouse and Human Mucocutaneous Candidiasis. Antimicrob Agents Chemother. 2022 Jul 19;66(7):e0030822. doi: 10.1128/aac.00308-22. Epub 2022 Jun 14.
Related trials for Candidiasis, Chronic Mucocutaneous
RecruitingObservational study
Natural History of Individuals With Immune System Problems That Lead to Fungal Infections
Chronic Mucocutaneous Candidiasis, Invasive Aspergillosis
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.
Alexandra Freeman, MD principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Amphotericin B in people with Candidiasis, Chronic Mucocutaneous, run by Matinas BioPharma Nanotechnologies, Inc. at 1 site 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?
The sponsor lists 12 inclusion and 15 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 7 Aug 2024. 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 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 7 Jun 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?
Clinical Response to Treatment of Mucocutaneous Candidiasis (time frame: 14-days at highest titrated dose). 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 NCT02629419, 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 7 Aug 2024.