Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform

Part of paid clinical trials in Woburn, Massachusetts.

Sponsor
Shwachman-Diamond Syndrome Alliance Inc
Study ID
NCT06999954
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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, secure, and convenient online platform, to * expand the understanding of SDS * improve the lives of people with SDS, and * accelerate the development of new therapies and cures for SDS. By joining, participants will receive early access to relevant information about new clinical trials and other research opportunities (such as clinical registries) based on their profile, accelerating research and increasing clinical trial impact and recruitment success. The platform, consent forms, and surveys are available in five languages: English, Spanish, French, German, and Italian. More languages to come.

Key Dates

Start date
Feb 7, 2024
Status verified
May 2025
Primary completion
Dec 31, 2088
Completion
Dec 31, 2088

Study Design

Enrollment
8,000 participants (estimated)

Primary Outcome Measure

Patient (or caregiver) reported symptoms over time [ Time Frame: At baseline and every 12 months, prospectively. ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Shwachman-Diamond Syndrome Alliance Inc.WoburnMassachusetts01888
Eszter Hars, Ph.D.
[email protected]
6173291838
SDS-GPS Program Team
[email protected]
+16173291838
Eszter Hars, Ph.D. (PRINCIPAL_INVESTIGATOR)

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Shwachman-Diamond Syndrome Alliance Inc.· Woburn, MA

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