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Progeria clinical trials

View clinical trials related to Progeria.

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NCT ID: NCT04512963 Completed - Clinical trials for Hutchinson-Gilford Progeria Syndrome

Phase I Study of Progerinin in Healthy Volunteers

Start date: August 24, 2020
Phase: Phase 1
Study type: Interventional

PRG-PRO-001 is a Phase I, Randomized, Double-blind, Placebo-Controlled, Single Ascending Dose (SAD) Study including a food interaction study, followed by a Multiple Ascending Dose (MAD) Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Profile of Progerinin in Healthy Volunteers. This is a first-in-human study. The study aims to determine the safety and tolerability of Progerinin after single and multiple doses in healthy volunteers and to evaluate the pharmacokinetics (PK) of Progerinin after single and multiple dose administrations in healthy volunteers.

NCT ID: NCT03871972 Completed - Clinical trials for Hutchinson-Gilford Progeria Syndrome

Umbilical Cord Blood Transfusion in Progeria Syndrome

Start date: March 5, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS). This is an 1 year trial with 3 IV infusions (4 months apart from each infusion) of umbilical cord blood units with oral Sirolimus to see the safety and efficacy.

NCT ID: NCT00879034 Completed - Progeria Clinical Trials

A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

Start date: March 2009
Phase: Phase 2
Study type: Interventional

This is an open label single arm feasibility trial. A combination of two oral agents (pravastatin and lonafarnib) and one intravenous (IV) agent (zoledronic acid) will be administered at doses and schedule currently applied in pediatrics. These agents all target farnesylation pathways at different points. Our goal is to inhibit farnesylation of abnormal lamin, the disease-causing protein in Hutchinson-Gilford Progeria Syndrome and progeroid laminopathies (henceforth "progeria"). The drugs will include the intravenous bisphosphonate zoledronic acid, oral HMG co-reductase inhibitor pravastatin and the oral farnesyltransferase inhibitor (FTI) lonafarnib (SCH 66336). Patients with genetically confirmed progeria will be eligible for this protocol. Treatment will be initiated for 4 weeks duration and may be extended depending on tolerability. This study will assess the feasibility of this treatment regimen in the first 4 weeks. If tolerated for 4 weeks, patients can be treated with this regimen for up to 6 months.

NCT ID: NCT00731016 Completed - Clinical trials for Hutchinson-Gilford Progeria Syndrome

Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid

Start date: October 2008
Phase: Phase 2
Study type: Interventional

We suggest treating the Hutchinson-Gilford Progeria Syndrome by two molecules (zoledronic acid and pravastatin).The therapeutic approach which we propose has for objectives to reduce, to prevent or to delay the gravest infringements of the disease, to prolong the life of the children, and in a more general way, aim at improving their living conditions.

NCT ID: NCT00425607 Completed - Progeria Clinical Trials

Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria

Start date: May 2007
Phase: Phase 2
Study type: Interventional

This is an open label dose adjusted phase II trial of the oral farnesyltransferase inhibitor (FTI) lonafarnib (SCH66336) for patients with HGPS and progeroid laminopathies.

NCT ID: NCT00094393 Completed - Progeria Clinical Trials

Clinical Studies of Progeria

Start date: October 14, 2004
Phase:
Study type: Observational

This study will examine children with Hutchinson-Gilford Progeria syndrome, a genetic disease that causes many changes to the body over time, including heart disease, bone changes, hair loss, and joint and skin changes. Often called a "premature aging" disease, progeria does not mimic aging completely. This study will examine which body systems are affected in progeria and how each system is affected over time in order to try to develop new treatments. Patients with progeria who are between 6 months and 70 years of age and who are able to travel to the NIH in Bethesda, Md., may be eligible for this 5- to 10-year study. Participants come to the NIH Clinical Center for evaluation every 2 years. Each 4-5 day visit includes the following tests and procedures: - Medical history and physical examination - Blood tests to analyze cardiovascular risk factors, blood counts, blood chemistries, and for research - Urine tests for sugar and proteins - Photographs to study growth problems - X-ray studies to determine bone density and body composition, such as body fat and muscle - Electrocardiogram (EKG) and echocardiogram (heart ultrasound) to study the heart and blood vessels - Lung function tests to measure energy consumption and lung capacity - Skin biopsy (surgical removal of a small skin sample) to examine cellular changes - Hearing tests - Eye examination to evaluate eyesight, eye pressure and structures of the eye - Physical therapy evaluation with stretching and exercises to measure how the joints bend and straighten - Dental examination, including X-rays - Meeting with a nutritionist who will track the patient's food intake and take body measurements - Magnetic resonance imaging (MRI) for patients who are old enough to undergo the procedure without sedation. This test uses a magnetic field and radio waves to examine body organs. For this test, the patient must lie still in the scanner, a narrow cylindrical tube. Patients are provided the results of their medical tests. Information about the patient is submitted to the PRF Cell and Tissue Bank in Peabody, Massachusetts.