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4 Study Matches

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease (GATEWAY)

The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult patients with Wilson's Disease prior to and following background WD therapy withdrawal.
Call 214-648-5005
studyfinder@utsouthwestern.edu, DOLORES.SANTOYO@UTSouthwestern.edu
William Lee
14217
All
18 Years to 65 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04537377
STU-2022-0533
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Main
Inclusion Criteria:

• Male or female aged between 18 and 65 years
• Confirmed diagnosis of WD
• Treated for WD according to international recommendations with no current evidence for inadequate treatment
• Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder and (ii) Stable laboratory parameters used to assess copper metabolism Main
Exclusion Criteria:

• ALT level ≥ 2 ULN that is not readily explained by extrinsic factors
• Total bilirubin > 1.5 x ULN in the absence of proven Gilbert's syndrome; in case of Gilbert's syndrome, direct bilirubin > ULN
• INR > ULN
• Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrolment visit
• Patient has moderate or severe renal impairment defined as eGFR CKD-EPI < 60 mL/min/1.73 m2, or patient has nephritis or nephrotic syndrome
• Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection
• Any history or current evidence of hepatitis B infection
• Any history of hepatitis C infection, unless previous viral RNA assays in two samples, collected at least 6 months apart, are negative
• Positive QuantiFERON®-TB Gold tuberculosis test result
• Any concomitant disorder/condition
•including hepatic disorders
•or treatment possibly interfering with the conduct or evaluation of the study
• Any history of diabetes
• Pregnancy or breastfeeding
• Body Mass Index ≥ 35 kg/m2 Other protocol defined Inclusion/ Exclusion criteria may apply
Genetic: VTX-801
Wilson's Disease
UT Southwestern
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A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

The purpose of this study is to evaluate the safety and tolerability of pegtarviliase in approximately 36 subjects with homocystinuria due to CBS deficiency.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Juana.Luevano@UTSouthwestern.edu
Markey McNutt
59152
All
12 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT05154890
STU-2022-0242
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Inclusion Criteria:
1. Diagnosis of homocystinuria due to CBS deficiency 2. Capable of providing signed informed consent/assent and to comply with all study related procedures 3. Is ≥12 years of age (≥18 in the US) at the time of signing the informed consent/assent 4. Plasma tHcy ≥50 µM (rounded to the nearest whole number) and documentation of previous tHcy ≥80 µM 5. Female subjects of child-bearing potential must have a negative serum pregnancy test during the screening period and a negative urine pregnancy test prior to dosing on the first day of treatment 6. If the subject (male or female) is engaging in sexual activity, he/she must be unable to become pregnant/cause pregnancy or must agree to use highly effective contraception 7. Subjects receiving pyridoxine and/or betaine must be on the same dose of the medication(s) for at least 6 weeks prior to the first administration of study drug and be willing and able to remain on a stable dose for the duration of the study. Similarly, those on prescribed dietary therapy must be on a consistent dietary regimen for at least 6 weeks prior to study drug and should maintain this regimen for the duration of the study
Exclusion Criteria:
1. Other medical conditions or co-morbidity(ies) that, in the opinion of the investigator, would put the subject at increased medical risk or interfere with study compliance or data interpretation (eg, severe intellectual disability that precludes completion of the required study assessments) 2. Currently participating in another therapeutic clinical study or has received any investigational agent within 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug in this study 3. Surgery requiring general anesthesia within 8 weeks prior to the first dose of study drug or planned surgery druing the treatment period 4. Active infection requiring anti-infective therapy <2 weeks prior to the first dose of study drug in this study; anti-infective therapy that completes ≥2 weeks prior to first dose of study drug is acceptable 5. Pregnant or nursing 6. Females of child-bearing potential who are using or plan to use estrogen-containing contraception during the study (unless the subject currently using estrogen-containing contraceptives is willing to switch to a non-estrogen-containing contraceptive at least 1 week before dosing and for the duration of the study) and for 30 days after the last dose 7. History of hypersensitivity to polyethylene glycol (PEG) that, in the judgment of the investigator, puts the subject at unacceptable risk for adverse events (AEs) 8. Serum creatinine level >1.5× the upper limit of normal (ULN) 9. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin level > 2× the ULN
Drug: Pegtarviliase, Drug: Pegtarviliase
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
UT Southwestern
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Mindfulness and Yoga Therapy for Acute Pain in Sickle Cell Disease

Patients with sickle cell disease suffer from acute and chronic pain that diminishes their quality of life. The purpose of this study is to assess the feasibility and acceptability of mindfulness meditation, breathing exercises, and gentle yoga therapy as supportive measures for the management of acute vaso-occlusive pain crises in the inpatient setting.
Call 214-648-5005
studyfinder@utsouthwestern.edu, KASIA.HARRAH@UTSouthwestern.edu
Siayareh Rambally
59781
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT05572294
STU-2021-0729
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Inclusion criteria:
• Patients with sickle cell disease, any genotype
• Adult patients at least 18 years of age
• English speaking
• Admitted to Clements University Hospital with an acute vaso-occlusive pain crisis Exclusion criteria:
• Unable to follow simple instructions
• Admitted to the intensive care unit
Behavioral: Mindfulness meditation, breathing exercises (including incentive spirometry), and yoga therapy
Sickle Cell Disease, Sickle Cell Crisis
Sickle Cell Disease, Yoga Therapy, Mindfulness
UT Southwestern
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Lysosomal Acid Lipase (LAL) Deficiency Registry (ALX-LALD-501)

This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.
Call 214-648-5005
studyfinder@utsouthwestern.edu, TODD.MORGAN@UTSouthwestern.edu
Thomas Kerr
23175
All
Not specified
This study is NOT accepting healthy volunteers
NCT01633489
STU-2020-1403
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Patients must have a confirmed diagnosis of LAL Deficiency. An Informed Consent and Authorization must be obtained prior to patient enrollment where required under applicable laws and regulations, or a waiver must be obtained by the Institutional Review Board/Independent Ethics Committee. Patients cannot be currently participating in an Alexion-sponsored clinical trial. Patients who have concluded participation in an Alexion-sponsored sebelipase alfa clinical trial are eligible to enroll in this Registry, and enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.
Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2, Acid Lipase Deficiency, LIPA Deficiency, LAL-Deficiency
UT Southwestern
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