Is NCT07845825 recruiting in San Antonio, TX?

Yes, NCT07845825 is currently recruiting participants at its San Antonio, TX location. This clifahdd clinical trial is actively enrolling qualified participants.

How do I join the clifahdd clinical trial in San Antonio?

To join this clinical trial in San Antonio, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this San Antonio clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

CLIFAHDD Clinical Trial in San Antonio, TX

About This CLIFAHDD Study in San Antonio

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.

Sponsor: The University of Texas Health Science Center at San Antonio

Who Can Participate

Inclusion Criteria

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

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).

Exclusion Criteria

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)

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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in San Antonio?

Yes, this clinical trial (NCT07845825) has an active research site in San Antonio, TX that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

CLIFAHDD Treatment Options in San Antonio, TX

If you're searching for clifahdd treatment options in San Antonio, TX, this clinical trial (NCT07845825) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our San Antonio research site is actively enrolling participants for this early phase 1 clinical trial. You'll receive care from experienced clifahdd specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all clifahdd clinical trials near you to find additional studies recruiting in your area.

CLIFAHDD Clinical Trial in San Antonio, TX

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