Clinical trials · Pennsylvania
Inborn Errors of Metabolism clinical trials in Pennsylvania
We’re tracking 4 recruiting inborn errors of metabolism studies with a site in Pennsylvania — including Early Phase 1 and Phase 1/Phase 2 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT00237315Recruiting
Long-term study of urea cycle disorders in affected families
This study follows people with urea cycle disorders (and some people who are very likely to have one) over time. It aims to better understand how these rare enzyme problems affect health, using medical tests and family information.
Philadelphia, PennsylvaniaAges Any age - NCT06839456RecruitingPhase 1/Phase 2
Stem cell addback to prevent infections after transplant
This trial tests whether adding back certain immune cells after a stem cell transplant can help prevent serious viral or fungal infections. It's for children and young adults up to age 25 who need a transplant for a blood cancer or other curable disease.
Philadelphia, PennsylvaniaAges 1 month–25 years - NCT00078078Recruiting
Understanding Methylmalonic Acidemia and Cobalamin Disorders
This research study follows patients with methylmalonic acidemia (a rare metabolic disorder affecting how the body breaks down certain proteins) or cobalamin (vitamin B12) disorders to better understand how these conditions develop and progress over time. Researchers will collect medical information, blood samples, and eye exams to help improve future treatments.
Pittsburgh, PennsylvaniaAges 1 month–115 years - NCT02356653RecruitingEarly Phase 1
Stem cell transplant help for children lacking a matching sibling
This expanded-access study offers a specific type of stem cell transplant preparation that removes certain immune cells (CD3 and CD19) from donor stem cells. It is for children who need a transplant but do not have a fully matched sibling donor and whose team cannot use the hospital’s usual matching device protocol.
Philadelphia, PennsylvaniaAges Up to 30 years
Where these studies are running in Pennsylvania
Institutions with a site for the recruiting inborn errors of metabolism studies listed above.
- Children's Hospital of Philadelphia · 3 studies
- UPMC Children's Hospital of Pittsburgh
What taking part in a inborn errors of metabolism study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Common questions
- Are there inborn errors of metabolism clinical trials in Pennsylvania?
- Yes. We're currently tracking 4 recruiting inborn errors of metabolism studies with a site in Pennsylvania, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a inborn errors of metabolism study in Pennsylvania?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Pennsylvania to take part?
- Not necessarily. These studies have a site in Pennsylvania, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a inborn errors of metabolism trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New inborn errors of metabolism studies open in Pennsylvania regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.