Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
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.
• 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
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.
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
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.
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
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.
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.