Growth delay due to insulin-like growth factor I resistance

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ORPHA:73273OMIM:270450E34.3
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1Active trials16Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Growth delay due to insulin-like growth factor I resistance, also called IGF-1 resistance or primary IGF-1 deficiency due to IGF1 receptor defect, is a rare condition where the body does not respond properly to a hormone called insulin-like growth factor 1 (IGF-1). IGF-1 is one of the most important signals the body uses to grow, especially during childhood. Even when the body makes enough IGF-1, the cells cannot 'hear' the signal because the receptor — the docking station on the cell surface — does not work correctly. This leads to poor growth before and after birth. Children with this condition are often born smaller than expected and continue to grow more slowly than their peers throughout childhood. They may have a small head size, subtle facial differences, and some may have mild learning or developmental differences. Blood tests typically show normal or even high levels of IGF-1, which is an important clue that the problem is resistance rather than a lack of the hormone itself. Treatment is challenging because the usual approach of giving extra growth hormone or IGF-1 may have limited benefit when the receptor is not working well. Management is largely supportive and focused on monitoring growth, addressing developmental needs, and improving quality of life. Research into better treatments is ongoing, and care is best provided by a team of specialists.

Also known as:

Key symptoms:

Slow growth before birth, resulting in a smaller-than-normal babyContinued slow growth after birth and short stature throughout childhoodSmall head size (microcephaly)Low birth weight despite a full-term pregnancyMild intellectual disability or learning difficulties in some casesDelayed developmental milestones such as sitting, walking, or talkingSubtle facial differencesHigh levels of IGF-1 in the blood despite poor growthPoor response to growth hormone treatment

Clinical phenotype terms (16)— hover any for plain English
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
Apr 2025Serum IGF-1 and IGF-1Ec in Malignancy Assessment

Çanakkale Onsekiz Mart University

TrialACTIVE NOT RECRUITING
Mar 2025Safety and Efficacy Evaluation of Anti IGF-1R Monoclonal Antibody Combined With Anti-PD-1 Monoclonal Antibody in Treatment in Patients With Metastatic Castration-Resistant Prostate Cancer

Shanghai Changzheng Hospital — EARLY_PHASE1

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Growth delay due to insulin-like growth factor I resistance.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
Serum IGF-1 and IGF-1Ec in Malignancy Assessment
Active
· Sites: Çanakkale · Age: 1899 yrs

Specialists

16 foundView all specialists →
JY
James Yao
Specialist
PI on 2 active trials
SM
Shancheng Ren, MD,PhD
Specialist
PI on 2 active trials
RO
Robert O'Donnell
Specialist
PI on 2 active trials
AF
Andrew B Hassan, BMBCh FRCP
Specialist
PI on 1 active trial
AC
Alberto Chiappori
TAMPA, FL
Specialist
PI on 1 active trial
KM
Kevin Judy, MD
PHILADELPHIA, PA
Specialist
PI on 1 active trial
EP
Eric Burak, PhD
Specialist
PI on 1 active trial
MP
Michael Bergqvist, M.D., Ph.D
Specialist
PI on 1 active trial
DC
David Camidge
AURORA, CO
Specialist
PI on 1 active trial
SP
Simon Ekman, M.D., Ph.D.
Specialist
PI on 1 active trial
PR
Principal Investigator - Czech Republic
Specialist
PI on 1 active trial
PI
Principal Investigator - Italy
Specialist
PI on 1 active trial
NM
Noura MS Shehabeldin, M.B.B.Ch
Specialist
PI on 1 active trial
MM
Michele Marinò, MD
Specialist
PI on 1 active trial
MM
Mohamed AA Zahran, M.D.
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Growth delay due to insulin-like growth factor I resistance.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

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Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What genetic test should my child have, and how long will results take?,Is my child a candidate for IGF-1 therapy, and what results can we realistically expect?,How often should we monitor my child's growth and hormone levels?,Should other family members be tested for this condition?,What developmental or educational support should we put in place now?,Are there any clinical trials or new treatments we should know about?,What signs should prompt us to seek urgent medical attention?

Common questions about Growth delay due to insulin-like growth factor I resistance

What is Growth delay due to insulin-like growth factor I resistance?

Growth delay due to insulin-like growth factor I resistance, also called IGF-1 resistance or primary IGF-1 deficiency due to IGF1 receptor defect, is a rare condition where the body does not respond properly to a hormone called insulin-like growth factor 1 (IGF-1). IGF-1 is one of the most important signals the body uses to grow, especially during childhood. Even when the body makes enough IGF-1, the cells cannot 'hear' the signal because the receptor — the docking station on the cell surface — does not work correctly. This leads to poor growth before and after birth. Children with this condi

How is Growth delay due to insulin-like growth factor I resistance inherited?

Growth delay due to insulin-like growth factor I resistance follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Growth delay due to insulin-like growth factor I resistance typically begin?

Typical onset of Growth delay due to insulin-like growth factor I resistance is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for Growth delay due to insulin-like growth factor I resistance?

Yes — 1 recruiting clinical trial is currently listed for Growth delay due to insulin-like growth factor I resistance on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Growth delay due to insulin-like growth factor I resistance?

16 specialists and care centers treating Growth delay due to insulin-like growth factor I resistance are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.