Clinical trials: Hutchinson-Gilford Progeria Syndrome
Hutchinson-Gilford Progeria Syndrome, Werner Syndrome
Phase I Study of Progerinin in Healthy Volunteers
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…
- Trial name
- NCT04512963
- Testing
- Progerinin
- Where
- United States
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- PRG Science & Technology Co., Ltd.
Hutchinson-Gilford Progeria Syndrome
A Study Observing Disease Progression in a Patient With Hutchinson-Gilford Progeria Syndrome (HGPS)
This study is a prospective, non-interventional, single-patient observational study following one patient with Hutchinson-Gilford Progeria Syndrome (HGPS), a rare disease that causes rapid, premature aging, over a 3-year period…
- Trial name
- progeria (NCT07839351)
- Where
- South Korea
- Volunteers
- 1 people
- Sponsor
- Bundang CHA Hospital
Progeria, Hutchinson-Gilford Syndrome
A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
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…
- Trial name
- NCT00879034
- Testing
- Lonafarnib, Zoledronic Acid, Pravastatin
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Boston Children's Hospital
Progeria
Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
Hutchinson-Gilford Progeria Syndrome (Progeria) is a rare autosomal disease that results in premature death at a median age of 13 years due to cardiovascular and cerebralvascular compromise.
- Trial name
- NCT00916747
- Testing
- Lonafarnib, Zoledronic Acid, and Pravastatin
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Boston Children's Hospital
Hutchinson-Gilford Progeria Syndrome
Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
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…
- Trial name
- NCT00731016
- Testing
- Zoledronic acid, pravastatin
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hutchinson-Gilford Progeria Syndrome
Creating Stem Cells From a Progeria Patient's Blood to Test Candidate Treatments in the Lab
This study aims to establish induced pluripotent stem cell (iPSC) lines from the blood of one pediatric patient with Hutchinson-Gilford Progeria Syndrome (HGPS), a rare disease that causes rapid, premature aging, in order to…
- Trial name
- NCT07803432
- Volunteers
- 1 people
- Sponsor
- Bundang CHA Hospital
Progeria, Hutchinson-Gilford Syndrome
Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria
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.
- Trial name
- NCT00425607
- Testing
- Lonafarnib
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Monica E. Kleinman
Hutchinson-Gilford Progeria Syndrome
Umbilical Cord Blood Transfusion in Progeria Syndrome
This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS).
- Trial name
- NCT03871972
- Testing
- Umbilical Cord Blood Unit
- Where
- South Korea
- Volunteers
- 2 people
- Sponsor
- Bundang CHA Hospital
Progeria, HGPS
Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy
This treatment IND protocol will allow patients with HGPS and progeroid laminopathies access to lonafarnib, the only compound shown to have an effect on the HGPS disease process resulting in improved outcomes (Gordon et al…
- Trial name
- NCT03895528
- Testing
- Lonafarnib
- Sponsor
- Eiger BioPharmaceuticals
Hutchinson-Gilford Progeria Syndrome
Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)
Researchers will compare treatment with progerinin plus lonafarnib vs lonafarnib alone to assess optimal dosing, safety, tolerability, and pharmacokinetics in patients with Hutchinson-Gilford Progeria Syndrome (HGPS).
- Trial name
- NCT06775041
- Testing
- Progerinin, Lonafarnib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- PRG Science & Technology Co., Ltd.
Progeria
Clinical Studies of Progeria
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.
- Trial name
- NCT00094393
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Human Genome Research Institute (NHGRI)