Medical Research

Hannahs Hope for Giant Axonal Neuropathy Inc

  • Rexford, NY
  • www.hannahshopefund.org

Mission Statement

DEDICATED TO FUNDING RESEARCH FOR A TREATMENT AND/OR A CURE FOR GIANT AXONAL NEUROPATHY
ABA: Hannah's Hope Fund for GAN,  Hannah's Hope Fund

Main Programs

  1. Natural History Study

service areas

International

Self-reported by organization

Areas Served Narrative

Giant Axonal Neuropathy is effecting children globally

Self-reported by organization

ruling year

2008

Principal Officer

Mrs. Lori Sames

Self-reported by organization

Keywords

Hannah's Hope Fund

Self-reported by organization

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EIN

45-0593226

 Number

5948883249

Also Known As

Hannah's Hope Fund

Contact

Cause Area (NTEE Code)

(H)

(G)

IRS Filing Requirement

This organization is required to file an IRS Form 990 or 990-EZ.

Programs + Results

How does this organization make a difference?

Impact statement

Hannah’s Hope Fund is a 501c3 public charity dedicated to finding a treatment and cure of Giant Axonal Neuropathy, (GAN), a rare, fatal disorder. Eight year old Hannah Sames, of Rexford, NY is the inspiration of Hannah’s Hope Fund, the only organization in the world focused on a treatment for this ravaging disorder.
Children with GAN die of progressive nerve death. They eventually lose the ability to walk, move, speak and swallow. They die in the second of third decade, typically from pneumonia.
Since August of 2008, the collaborative team of scientists working with Hannah’s Hope Fund have developed gene therapy to treat the nerve cells in the spinal cord and brain with a healthy copy of the GAN gene. Scientists use a benign viral vector as a delivery truck to transport a healthy copy of the GAN gene to nerve cells, providing a life sustaining protein that is missing in cells of children with GAN.
The scientists working on this gene therapy approach for GAN feel it is ready for a human clinical trial. We met with the FDA on Jan. 26, 2012 and now have a clear path forward. If the FDA required safety studies go as planned, we will be ready to begin a trial this Fall of 2012. It is estimated that we need to raise another $500,000 to fund the Phase 1 clinical trial. 
Since GAN is very rare, it doesn’t attract the attention of pharmaceutical or biotechnology companies due to smaller profits. Therefore, it is reality that the families of loved ones suffering from rare disorders must raise the funds for therapy development and clinical trials in order to saved loved ones. It is our hope that a Biotech will take over the project and bring it through Phase 2 and Phase 3 trials if the Phase 1 study reveals GAN gene therapy is safe, and the data indicate it either stops or slows progression of the disease.
Please choose to get involved in our mission.

Programs

What are the organization's current programs, how do they measure success, and who do the programs serve?

Self-reported by organization

Program 1

Natural History Study

Giant Axonal Neuropathy Natural History Study
Verified by: Columbia University, December 2011
First Received: December 29, 2011 | Last Updated: December 30, 2011 | Phase: N/A | Start Date: December 2011
Overall Status: Recruiting | Estimated Enrollment: 15
…Tell a FriendPrint
Giant
Axonal Neuropathy (GAN) is a devastating and rare childhood disease.
Children with GAN develop increasing muscle weakness, impaired
sensation, and at times mental retardation. GAN starts in infancy,
leads to significant disability, and typically leads to death within
the first 30 years of life. GAN is caused by a defect in the
"gigaxonin" (GAN) gene, resulting in pathologically enlarged and...
Brief Summary
Official Title: “Clinical Study of Giant Axonal Neuropathy”
Giant
Axonal Neuropathy (GAN) is a devastating and rare childhood disease.
Children with GAN develop increasing muscle weakness, impaired
sensation, and at times mental retardation. GAN starts in infancy,
leads to significant disability, and typically leads to death within
the first 30 years of life. GAN is caused by a defect in the
"gigaxonin" (GAN) gene, resulting in pathologically enlarged and
dysfunctional nerves. Currently, there is no effective therapy. To find
out what medications can help patients with GAN, the investigators have
to conduct clinical trials. In this study, the investigators propose to
prepare for future clinical trials and will invite GAN patients to join
our research effort.
The investigators will examine them
regularly to better understand their disease. The visits will include
questions, a physical exam, blood drawing, a lumbar puncture, and a
skin biopsy. The visits will also include tests that assess the
electrical conductivity of the patients' nerves as well as a test to
measure the patients' brain wave activity. In addition, the
investigators will be performing tests to evaluate the patients' motor
function, their vision, and thinking ability. Identifying an effective
GAN treatment is very important because there is currently none.
Clinical trials are the only way to decide whether a new treatment
works in GAN patients or not.
With the future objective of
conducting clinical trials in GAN, the proposed project has three
specific aims. The first is to plan for clinical trials by developing
reliable outcome measures, and establishing the infrastructure needed
to carry out efficient clinical trials.
The second is to further
characterize the patient population from a clinical and molecular point
of view, and the third aim is to utilize the information gathered in
this study to further pre-clinical GAN drug development to select
candidate drugs.
Study Type: ObservationalStudy Design: Observational Model: Cohort, Time Perspective: ProspectiveStudy Primary Completion Date: December 2014Outcome Measures for this Clinical Trial
Primary MeasuresGross Motor Function Measure (GMFM)Time Frame: Up to 24 months
Safety Issue?: No
Secondary MeasuresNerve Conduction Study (NCS)/Motor Unit Number Estimation (MUNE)Time Frame: Up to 24 months
Safety Issue?: NoSomatosensory Evoked Potential (SSEP)Time Frame: Up to 24 months
Safety Issue?: NoBrainstem Auditory Evoked Response (BAER)Time Frame: Up to 24 months
Safety Issue?: NoPulmonary Function Testing (PFT)/Forced Vital Capacity (FVC)Time Frame: Up to 24 months
Safety Issue?: No
Criteria for Participation in this Clinical Trial
Inclusion Criteria:
1. Clinical diagnosis of Giant Axonal Neuropathy.2. Documentation of the presence of a mutation in the GAN gene as
determined by gene sequencing from a CAP/CLIA certified laboratory or
an equivalent organization.3. Parents or if applicable subjects must give informed consent must be capable of complying with the study procedures.4. Willing and able to comply with all protocol requirements and procedures.
Exclusion Criteria:
1. Unwilling or unable to travel to Columbia University Medical Center.2. Unstable medical condition precluding participation.3. Significant respiratory compromise that would interfere with safe travel to site of evaluation.4. Having a contraindication to the MRI safety requirements,
including pacemaker or other implanted electrical device, brain
stimulator, some types of dental implants, aneurysm clips (metal clips
on the wall of a large artery), metallic prostheses (including metal
pins and rods, heart valves, and cochlear implants), implanted delivery
pump, shrapnel fragments, or history of claustrophobia.
Gender Eligibility for this Clinical Trial: Both
Minimum Age for this Clinical Trial: N/A
Maximum Age for this Clinical Trial: N/A
Are Healthy Volunteers Accepted for this Clinical Trial?: No
Clinical Trial Investigator Information
Lead Investigator: Columbia University Other
Overall Clinical Trial Officials and Contacts
Douglas M. Sproule, MD, MSc Principal Investigator Columbia University
Overall Contact: Jonathan D. Marra, M.A. 212-305-2461 jdm2132@columbia.edu
Additional Information
Information obtained from ClinicalTrials.gov on March 15, 2012
Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT01503125
Study ID Number: AAAI4500
ClinicalTrials.gov Identifier: NCT01503125
Health Authority: United States: Institutional Review Board

Category

Medical Research

Budget

$300,000.00

Population Served

Children and Youth (infants - 19 years.)

Adults

service areas

International

Self-reported by organization

Areas Served Narrative

Giant Axonal Neuropathy is effecting children globally

Self-reported by organization

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Financials

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HANNAHS HOPE FOR GIANT AXONAL NEUROPATHY INC
Fiscal year: Jan 01-Dec 31
Yes, financials were audited by an independent accountant.

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Operations

The people, governance practices, and partners that make the organization tick.

Hannahs Hope for Giant Axonal Neuropathy Inc

Leadership

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Free: Gain immediate access to the following:
  • Address, phone, website and contact information
  • Forms 990 for 2014, 2013 and 2012
  • Board Chair and Board Members
  • Access to the GuideStar Community
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Principal Officer

Mrs. Lori Sames

Governance

BOARD CHAIR

Mrs. Carrie Favaro

No Affiliation

BOARD LEADERSHIP PRACTICES

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BOARD ORIENTATION & EDUCATION

Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations?


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CEO OVERSIGHT

Has the board conducted a formal, written assessment of the chief executive within the past year?


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ETHICS & TRANSPARENCY

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BOARD COMPOSITION

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BOARD PERFORMANCE

Has the board conducted a formal, written self-assessment of its performance within the past three years?