Clinical trials: Exocrine Pancreatic Insufficiency, open to volunteers
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Phase 2 Study to Assess the Safety and Efficacy of ANG003
In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine pancreatic insufficiency (EPI).
- Trial name
- NCT07450547
- Testing
- ANG003 Dose A, ANG003 Dose B, Creon
- Where
- 28 sites in United States
- Volunteers
- 113 people
- Sponsor
- Anagram Therapeutics, Inc.
Shwachman-Diamond Syndrome, Shwachman-Diamond Syndrome-Like
Shwachman Diamond Syndrome Registry and Study
Shwachman-Diamond syndrome (SDS) is a genetic condition characterized by bone marrow failure, medical co-morbidities, and leukemia predisposition.
- Trial name
- SDSR (NCT06056908)
- Where
- 4 sites in United States
- Volunteers
- 5,000 people
- Sponsor
- Boston Children's Hospital
Exocrine Pancreatic Insuficiency
Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Syndrome After Liver Transplantation
The goal of this clinical trial is to determine the prevalence of exocrine pancreatic insufficiency (EPI) in a population of pediatric and adult participants with Alagille Syndrome (ALGS) post liver transplant.
- Trial name
- NCT07335523
- Testing
- Fecal Fat
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- Digestive Care, Inc.
Neutropenia
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are…
- Trial name
- NCT06056297
- Testing
- Mavorixafor
- Where
- 114 sites in United States, Argentina and 23 more
- Volunteers
- 176 people
- Sponsor
- X4 Pharmaceuticals