CLIFAHDDEarly Phase 1NCT07845825
Aprepitant for the Treatment of CLIFAHDD
A Early Phase 1 study testing Aprepitant Powder for Oral Suspension in people with CLIFAHDD and Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, run by The University of Texas Health Science Center at San Antonio at 1 site in United States.
Trial summary
There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare and severe neurodevelopmental disease. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, NALCN Channelopathy
- What is being tested?
- Aprepitant Powder for Oral Suspension
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Oct 2026to 31 Jul 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of CLIFAHDD (confirmed by clinical and genetic report per best practices)
- NALCN variant with predicted NALCN gain-of-function in established functional assays and/or computational assessments
- Age >6 months at the time of initial consent/assent
- Weight >6kg at the time of initial consent/assent
- Ability of participant or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.
- For children (<18y at time of consent/assent), informed assent (if developmentally appropriate) and parental informed consent to participate in the study
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- Willingness to comply with all study-related requirements, including Aprepitant regimen and travel to San Antonio, TX for specified visits
- Has adequate organ functions as defined by the following laboratory parameters at baseline (laboratory parameters outside of these ranges that are deemed clinically insignificant should be discussed with the Independent Safety Monitor):
- Absolute neutrophil count ≥1000 cells/uL;
- hemoglobin ≥8.5 g/dL;
- platelet count ≥100,000/mm3;
- aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 x upper limit of normal (ULN);
- total bilirubin ≤ 1.5 x institutional ULN (exception: subjects with known Gilbert's syndrome and total bilirubin ≤2 x institutional ULN at screening or anytime during the prior 6 months are eligible);
- adequate renal function defined as calculated creatinine clearance >60 mL/min using the Cockcroft Gault Method;
- acceptable coagulation parameters including international normalized ratio (INR) <1.4 and partial thromboplastin time (PTT) ≤ 1.5 x institutional ULN;
- serum albumin ≥3.0 g/dL
- Women of child-bearing age participants must use highly effective forms of birth control defined as those with a failure rate of < 1% per year. Acceptable methods include: complete sexual abstinence (anticipated given severity of neurodevelopmental symptoms in CLIFAHDD), combined hormonal contraceptives, progestogen-only hormonal contraceptives, intrauterine devices (IUDs), intrauterine hormone-releasing systems (IUS), bilateral tubal occlusion, or vasectomized partner (provided the partner is the sole sexual partner).
Who cannot join
- Known hypersensitivity to any component of the study treatment formulation(s)
- Concomitant use of pimozide, terfenadine, astemizole, cisapride, flibanserin, lomitapide (contraindicated CYP3A4 substrate)
- Concomitant use of strong (Clarithromycin, telithromycin, nefazodone, itraconazole, ketoconazole, atazanavir, darunavir, indinavir, lopinavir, nelfinavir, ritonavir, saquinavir, tipranavir) or moderate (amiodarone, erythromycin, fluconazole, miconazole, diltiazem, verapamil, delavirdine, amprenavir, fosamprenavir, conivaptan, danazol, ketoconazole) CYP3A4 inhibitors and strong CYP3A4 inducers (apalutamide, carbamazepine, dexamethasone, enzalutamide, fosphenytoin, lumacaftor, midostaurin, mitotane, pentobarbital, phenobarbital, rifampin, phenytoin) or CYP3A4 substrates (Docetaxel, paclitaxel, etoposide, irinotecan, ifosfamide, imatinib, vinorelbine, vinblastine, vincristine, colchicine, axitinib). Participants on one of these medications but unable to discontinue for the trial and considered otherwise low risk should be discussed with the Independent Safety Monitor but may be considered for enrollment.
- Concomitant use of high-risk cytochrome P450 family 2 subfamily C member 9 (CYP2C9) substrates (e.g., warfarin or phenytoin or tolbutamide)
- Patients who are medically unstable or have been hospitalized for an acute medical condition in the 3 months prior to the first active drug visit
- Patients who are acutely ill in the hospital or intensive care unit (ICU)
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- Any other history of clinically significant acute or chronic neurologic disease, respiratory disease, cardiovascular disease, gastrointestinal disease, liver disease, renal disease, endocrine disorder, infectious disease, or any other medical or psychiatric condition that in the opinion of the investigator or study clinician will significantly increase the safety risk for the subject or confound the interpretation of the study data.
- Currently receiving any other investigational agents or has received study treatment or used an investigational device within 30 days of the first dose of treatment
- Caregivers unwilling to follow study procedures or follow protocol as outlined.
- Pregnancy
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Emily Durham, PhD contact
United States1 site, 1 recruiting
- Recruiting
UT Health San Antonio - Center for Brain Health
San Antonio, Texas, 78229
Megan Iammarino, DPT, 440-382-3366, iammarinom@uthscsa.edu
Randee Kent-Baron, 210-450-0524, kentbaron@uthscsa.edu
About this study
Official title: Channeling Hope Trial 1: A Phase 1, Open-label, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) of Chronic Aprepitant Use in Individuals With Congenital Contractures of the Limbs and Face, Hypotonia, and Development Delay (CLIFAHDD)
There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare and severe neurodevelopmental disease. The investigators have learned more about the cause of the disease and researched possible treatments based on both the cause of the disease and the way certain drugs work. The investigators are interested in understanding the safety of using aprepitant (a drug already approved by the United States Food \& Drug Administration [FDA] to treat nausea and vomiting for kids and adults who are receiving chemotherapy) on people diagnosed with CLIFAHDD. The overall goal of this Phase 1 study is to study whether this drug is safe for daily use in individuals with CLIFAHDD and to find more information on how it impacts daily functioning and quality of life. The total study will take around 8 months total. This includes the 90 days before the drug starts and the 21 days after the drug ends.
Read the detailed description
The sodium leak ion channel NALCN is responsible for a background sodium influx that contributes to the resting membrane potential and serves as a key regulator in neuronal and cell excitability. Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay (CLIFAHDD; OMIM 616266) is a debilitating and ultra-rare neurodevelopmental disease with early infancy onset caused by a de novo autosomal dominant mutation in NALCN, often associated with NALCN gain-of-function (GOF). CLIFAHDD is associated with global neurodevelopmental delay, hyperkinetic movement disorders (e.g., dystonia, chorea, ataxia), seizures, apnea, constipation, insomnia, and increased risk of early childhood mortality. There are currently no drug treatments for CLIFAHDD. A multidisciplinary drug repurposing pipeline identified aprepitant as a promising lead candidate with converging functional, in silico, in vitro, and in vivo preclinical data support.
The primary objective of this Phase 1 clinical trial is to determine the safety, tolerability and pharmacokinetics (PK) of chronic aprepitant use in individuals with CLIFAHDD. The hypothesis is that aprepitant will be safe, tolerable, and amendable to chronic use in patients with CLIFAHDD based on prior safety and PK data on aprepitant use in pediatric and adult populations, including off-label chronic use. The exploratory objectives are: (1) to identify potential pharmacodynamic biomarkers reflecting NALCN pathway modulation; (2) to assess preliminary signals of clinical efficacy and outcome measures for future trials.
The study is designed as a single-center, Phase 1, open-label, dose-escalation study designed to evaluate the safety, tolerability, and PK of chronically administered oral aprepitant in individuals with CLIFAHDD (n=3-5, with protocol-defined criteria allowing expansion to a max n=5).
Participants will undergo evaluations of disease severity and symptoms over a 90 day lead-in period. For the baseline visit and initiation of cycle 1, participants will travel to UTHSA for onsite clinical monitoring, collection of baseline clinical outcome assessments, and PK assessments. Following the initial dose, each participant's biological and pharmacokinetic response will be evaluated over a 72-hour period. PK sampling will include an 8-point plasma concentration-time profile followed by additional blood draws. After the initial PK assessment, participants will enter a structured dose escalation period, during which aprepitant will be increased in 1mg/kg every 4 weeks. PK analyses with an 8-point plasma concentration-time profile will be performed at the end of each 4-week cycle to establish steady state drug availability with chronic dosing. Safety evaluations, clinical outcome assessments, and pharmacodynamic biomarkers will be assessed at the end of each 4-week cycle. Safety data (rather than PK analyses) will be used to guide dose escalation and will be reviewed in real time by the study clinician and Principal Investigator, without a planned pause in study conduct. Escalation will continue until either a maximum tolerated dose (MTD) is established or the maximum FDA-approved dose is reached. At the maximum dose or end of Cycle 3, participants will undergo an 8-point plasma concentration-time profile to characterize dose-dependent PK changes and elimination. Participants will undergo details safety, clinical, and pharmacodynamic biomarker assessments. Remote safety monitoring will continue for 3 weeks
- Conditions
- CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, NALCN Channelopathy, Ataxia - Other, Neurodevelopmental Disorder (Diagnosis)
- Being tested
- Aprepitant Powder for Oral Suspension
- Keywords
- CLIFAHDD, Sodium leak ion channel, NALCN, Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay, Aprepitant, Channelopathy, Neurodevelopmental Disorder, Neurogenetic Disorder, Ataxia
- Medical subject headings
- Facies, Muscle Hypotonia, Learning Disabilities, Neurodevelopmental Disorders, Disease, Channelopathies, Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Aprepitant Powder for Oral SuspensionDrug
Also called Emend
Administration will begin at 1mg/kg up to a maximum of 40mg in weeks 1-4, then escalate to 2mg/kg up to a maximum of 80mg in weeks 5-8, then 3mg/kg up to a maximum of 125mg in weeks 9-12
| Study group | Role | What they receive |
|---|---|---|
| Aprepitant treatment group | Experimental | Participant enrolled in the study will be administered oral aprepitant to evaluate safety and tolerability Receives: Drug: Aprepitant Powder for Oral Suspension |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse events
Adverse events will be assessed for relatedness to the study drug and assessed according to Common Terminology Criteria for Adverse Events (CTCAE) grade
Measured: Baseline to 24 weeks
Dose-limiting toxicity (DLT)
Number of events that lead to specified worsening of patients neurologic or non-neurologic condition during the treatment period which may lead to study treatment interruption for more than 14 days
Measured: Baseline to 24 weeks
Maximum administered dose
The highest dose at which no more than one instance of DLT is observed, or the maximum labeled dose
Measured: Baseline to 24 weeks
Secondary outcomes (1)
Area under the concentration-time curve (AUC)
The area under the concentration vs. time curve is a useful description of exposure of drug following administration. Following the initial dose, each participant's biological and pharmacokinetic response will be evaluated over a 72-hour period over a single dosing interval.
Measured: Dose time to 72 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2026
- Study start31 Oct 2026 (estimated)
- Primary completion31 May 2027 (estimated)
- Study completion31 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CLIFAHDDStudies of the same condition, open and under way.
- More trials run by The University of Texas Health Science Center at San AntonioThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single-center, one arm, open-label, dose-escalation clinical trial
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Channeling Hope Foundation, Cures Within Reach
- Responsible party
- Principal investigator, Megan Iammarino, The University of Texas Health Science Center at San Antonio
- Investigators
- Megan Iammarino, DPT, Principal investigator, The University of Texas Health Science Center at San Antonio
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07845825
- Sponsor's protocol ID
- STUDY00002877
- Status last verified
- Nov 2026
- First submitted
- 21 Sep 2026
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual Participant Data will be deidentified and shared with Cures Within Reach and Channeling Hope Foundation (study sponsors). Aggregated data will be shared as summary results on ClinicalTrials.gov and in a peer review journal. All de-identified individual participant data will be shared with external investigators with reasonable requests and agreements in place.
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Channeling Hope Trial 1: A Phase 1, Open-label, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) of Chronic Aprepitant Use in Individuals With Congenital Contractures of the Limbs and Face, Hypotonia, and Development Delay (CLIFAHDD)
- ClinicalTrials.gov ID
- NCT07845825
- Acronym / sponsor ID
- STUDY00002877
- Conditions
- CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, NALCN Channelopathy, Ataxia - Other, Neurodevelopmental Disorder (Diagnosis)
- Keywords
- CLIFAHDD, Sodium leak ion channel, NALCN, Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay, Aprepitant, Channelopathy, Neurodevelopmental Disorder, Neurogenetic Disorder
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- The University of Texas Health Science Center at San AntonioUniversity, hospital or other
- Collaborators
- Channeling Hope Foundation, Cures Within Reach
- Study start
- 31 Oct 2026 (estimated)
- Primary completion
- 31 May 2027 (estimated)
- Study completion
- 31 Jul 2027 (estimated)
- First posted
- 29 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare and severe neurodevelopmental disease. The investigators have learned more about the cause of the disease and researched possible treatments based on both the cause of the disease and the way certain drugs work. The investigators are interested in understanding the safety of using aprepitant (a drug already approved by the United States Food \& Drug Administration [FDA] to treat nausea and vomiting for kids and adults who are receiving chemotherapy) on people diagnosed with CLIFAHDD. The overall goal of this Phase 1 study is to study whether this drug is safe for daily use in individuals with CLIFAHDD and to find more information on how it impacts daily functioning and quality of life. The total study will take around 8 months total. This includes the 90 days before the drug starts and the 21 days after the drug ends.
Detailed description
The sodium leak ion channel NALCN is responsible for a background sodium influx that contributes to the resting membrane potential and serves as a key regulator in neuronal and cell excitability. Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay (CLIFAHDD; OMIM 616266) is a debilitating and ultra-rare neurodevelopmental disease with early infancy onset caused by a de novo autosomal dominant mutation in NALCN, often associated with NALCN gain-of-function (GOF). CLIFAHDD is associated with global neurodevelopmental delay, hyperkinetic movement disorders (e.g., dystonia, chorea, ataxia), seizures, apnea, constipation, insomnia, and increased risk of early childhood mortality. There are currently no drug treatments for CLIFAHDD. A multidisciplinary drug repurposing pipeline identified aprepitant as a promising lead candidate with converging functional, in silico, in vitro, and in vivo preclinical data support.
The primary objective of this Phase 1 clinical trial is to determine the safety, tolerability and pharmacokinetics (PK) of chronic aprepitant use in individuals with CLIFAHDD. The hypothesis is that aprepitant will be safe, tolerable, and amendable to chronic use in patients with CLIFAHDD based on prior safety and PK data on aprepitant use in pediatric and adult populations, including off-label chronic use. The exploratory objectives are: (1) to identify potential pharmacodynamic biomarkers reflecting NALCN pathway modulation; (2) to assess preliminary signals of clinical efficacy and outcome measures for future trials.
The study is designed as a single-center, Phase 1, open-label, dose-escalation study designed to evaluate the safety, tolerability, and PK of chronically administered oral aprepitant in individuals with CLIFAHDD (n=3-5, with protocol-defined criteria allowing expansion to a max n=5).
Participants will undergo evaluations of disease severity and symptoms over a 90 day lead-in period. For the baseline visit and initiation of cycle 1, participants will travel to UTHSA for onsite clinical monitoring, collection of baseline clinical outcome assessments, and PK assessments. Following the initial dose, each participant's biological and pharmacokinetic response will be evaluated over a 72-hour period. PK sampling will include an 8-point plasma concentration-time profile followed by additional blood draws. After the initial PK assessment, participants will enter a structured dose escalation period, during which aprepitant will be increased in 1mg/kg every 4 weeks. PK analyses with an 8-point plasma concentration-time profile will be performed at the end of each 4-week cycle to establish steady state drug availability with chronic dosing. Safety evaluations, clinical outcome assessments, and pharmacodynamic biomarkers will be assessed at the end of each 4-week cycle. Safety data (rather than PK analyses) will be used to guide dose escalation and will be reviewed in real time by the study clinician and Principal Investigator, without a planned pause in study conduct. Escalation will continue until either a maximum tolerated dose (MTD) is established or the maximum FDA-approved dose is reached. At the maximum dose or end of Cycle 3, participants will undergo an 8-point plasma concentration-time profile to characterize dose-dependent PK changes and elimination. Participants will undergo details safety, clinical, and pharmacodynamic biomarker assessments. Remote safety monitoring will continue for 3 weeks
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single-center, one arm, open-label, dose-escalation clinical trial
| Arm | Type | Receives |
|---|---|---|
| Aprepitant treatment group | Experimental | Drug: Aprepitant Powder for Oral SuspensionParticipant enrolled in the study will be administered oral aprepitant to evaluate safety and tolerability |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (17 listed)
- Diagnosis of CLIFAHDD (confirmed by clinical and genetic report per best practices)
- NALCN variant with predicted NALCN gain-of-function in established functional assays and/or computational assessments
- Age >6 months at the time of initial consent/assent
- Weight >6kg at the time of initial consent/assent
- Ability of participant or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.
Key exclusion criteria (10 listed)
- Known hypersensitivity to any component of the study treatment formulation(s)
- Concomitant use of pimozide, terfenadine, astemizole, cisapride, flibanserin, lomitapide (contraindicated CYP3A4 substrate)
- Concomitant use of strong (Clarithromycin, telithromycin, nefazodone, itraconazole, ketoconazole, atazanavir, darunavir, indinavir, lopinavir, nelfinavir, ritonavir, saquinavir, tipranavir) or moderate (amiodarone, erythromycin, fluconazole, miconazole, diltiazem, verapamil, delavirdine, amprenavir, fosamprenavir, conivaptan, danazol, ketoconazole) CYP3A4 inhibitors and strong CYP3A4 inducers (apalutamide, carbamazepine, dexamethasone, enzalutamide, fosphenytoin, lumacaftor, midostaurin, mitotane, pentobarbital, phenobarbital, rifampin, phenytoin) or CYP3A4 substrates (Docetaxel, paclitaxel, etoposide, irinotecan, ifosfamide, imatinib, vinorelbine, vinblastine, vincristine, colchicine, axitinib). Participants on one of these medications but unable to discontinue for the trial and considered otherwise low risk should be discussed with the Independent Safety Monitor but may be considered for enrollment.
- Concomitant use of high-risk cytochrome P450 family 2 subfamily C member 9 (CYP2C9) substrates (e.g., warfarin or phenytoin or tolbutamide)
- Patients who are medically unstable or have been hospitalized for an acute medical condition in the 3 months prior to the first active drug 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 |
|---|---|---|
| Aprepitant Powder for Oral SuspensionAlso: EmendAdministration will begin at 1mg/kg up to a maximum of 40mg in weeks 1-4, then escalate to 2mg/kg up to a maximum of 80mg in weeks 5-8, then 3mg/kg up to a maximum of 125mg in weeks 9-12 | Drug | Aprepitant treatment group |
Primary outcome
Number of Adverse events
Time frame: Baseline to 24 weeks
Adverse events will be assessed for relatedness to the study drug and assessed according to Common Terminology Criteria for Adverse Events (CTCAE) grade
Dose-limiting toxicity (DLT)
Time frame: Baseline to 24 weeks
Number of events that lead to specified worsening of patients neurologic or non-neurologic condition during the treatment period which may lead to study treatment interruption for more than 14 days
Maximum administered dose
Time frame: Baseline to 24 weeks
The highest dose at which no more than one instance of DLT is observed, or the maximum labeled dose
1 secondary outcome
Area under the concentration-time curve (AUC)
Time frame: Dose time to 72 hours
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Study sponsor and 501c3 patient advocacy group to advance care and develop treatments for NALCN Channel Related DiseasesLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Individual Participant Data will be deidentified and shared with Cures Within Reach and Channeling Hope Foundation (study sponsors). Aggregated data will be shared as summary results on ClinicalTrials.gov and in a peer review journal. All de-identified individual participant data will be shared with external investigators with reasonable requests and agreements in place.
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.
Emily Durham, PhD contact
Megan Iammarino, DPT principal investigator
The University of Texas Health Science Center at San Antonio
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Aprepitant Powder for Oral Suspension in people with CLIFAHDD and Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, run by The University of Texas Health Science Center at San Antonio 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 17 inclusion and 10 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: looking for participants now, as of the sponsor's last update on 5 Nov 2026. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Carolyn Paiz (210-450-7547, paizc@uthscsa.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Adverse events (time frame: Baseline to 24 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Nov 2026.