How to Join This Clinical Trial - NCTNCT06999954

Learn how to participate in this Not Specified trial studying an investigational therapy for Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia, Heme Malignancy, Shwachman Syndrome, Inherited Bone Marrow Failure, Exocrine Pancreatic Insufficiency, WHIM, ELANE, SBDS Gene Mutation, EFL1 Gene Mutation, DNAJC21 Gene Mutation, SRP54 Gene Mutation, Inherited Cancer Syndrome, Inherited Cancer-Predisposing Syndrome, Neutropenia, Severe Chronic, Neutropenia Other, Neutropenia Chronic Benign, Ribosome Alteration, Ribosomopathy, Immune Deficiency, Inherited BMF Syndrome, Inherited Immunodeficiency Diseases, Cognitive Delay, Mild, Myelodysplastic Syndromes, Pancytopenia. This study is currently enrolling participants.

Am I Eligible for This Clinical Trial?

This clinical research study is looking for participants with Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia, Heme Malignancy, Shwachman Syndrome, Inherited Bone Marrow Failure, Exocrine Pancreatic Insufficiency, WHIM, ELANE, SBDS Gene Mutation, EFL1 Gene Mutation, DNAJC21 Gene Mutation, SRP54 Gene Mutation, Inherited Cancer Syndrome, Inherited Cancer-Predisposing Syndrome, Neutropenia, Severe Chronic, Neutropenia Other, Neutropenia Chronic Benign, Ribosome Alteration, Ribosomopathy, Immune Deficiency, Inherited BMF Syndrome, Inherited Immunodeficiency Diseases, Cognitive Delay, Mild, Myelodysplastic Syndromes, Pancytopenia. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.

Condition Being Studied

Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia, Heme Malignancy, Shwachman Syndrome, Inherited Bone Marrow Failure, Exocrine Pancreatic Insufficiency, WHIM, ELANE, SBDS Gene Mutation, EFL1 Gene Mutation, DNAJC21 Gene Mutation, SRP54 Gene Mutation, Inherited Cancer Syndrome, Inherited Cancer-Predisposing Syndrome, Neutropenia, Severe Chronic, Neutropenia Other, Neutropenia Chronic Benign, Ribosome Alteration, Ribosomopathy, Immune Deficiency, Inherited BMF Syndrome, Inherited Immunodeficiency Diseases, Cognitive Delay, Mild, Myelodysplastic Syndromes, Pancytopenia

Treatment Being Tested

Investigational treatment

Study Phase

Not Specified - Research study

Enrollment Status

Currently enrolling participants

Study Identifier

NCTNCT06999954 - ClinicalTrials.gov Identifier

Sponsored By

Study sponsor

What to Expect as a Participant

Participating in this clinical trial involves:

• Initial screening to determine eligibility
• Regular study visits and health assessments
• Receiving the study treatment or placebo
• Medical monitoring and follow-up care
• Contributing to medical research that may help others
• Potential access to new treatments before they're widely available

Clinical Trial Benefits and Compensation

Participants in this clinical research study may receive:

• Close medical monitoring by healthcare professionals
• Access to potential new treatments
• Compensation for time and travel (varies by study)
• No-cost study-related medical care
• The opportunity to help advance medical knowledge

Where Is This Clinical Trial Located?

This study is enrolling participants at 1 locations. Find a study site near you:

How to Enroll in This Study

To learn more about participating in this Not Specified clinical trial for Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia, Heme Malignancy, Shwachman Syndrome, Inherited Bone Marrow Failure, Exocrine Pancreatic Insufficiency, WHIM, ELANE, SBDS Gene Mutation, EFL1 Gene Mutation, DNAJC21 Gene Mutation, SRP54 Gene Mutation, Inherited Cancer Syndrome, Inherited Cancer-Predisposing Syndrome, Neutropenia, Severe Chronic, Neutropenia Other, Neutropenia Chronic Benign, Ribosome Alteration, Ribosomopathy, Immune Deficiency, Inherited BMF Syndrome, Inherited Immunodeficiency Diseases, Cognitive Delay, Mild, Myelodysplastic Syndromes, Pancytopenia:

1. Review the eligibility criteria with your healthcare provider
2. Contact the study team for a pre-screening interview
3. Schedule an in-person screening visit if eligible
4. Review and sign the informed consent form
5. Begin participation in the clinical trial

Why Choose Quri.ai as Your Clinical Trial Matching Service

Quri.ai is a free clinical trial matching service that helps patients connect with research sites. We make it easier to:

• Find clinical trials that match your condition
• Get matched to the right study location near you
• Connect directly with study coordinators
• Understand if you qualify with simplified eligibility screening
• Navigate the enrollment process with personalized support
• Access new treatments through clinical research participation