Growth delay due to insulin-like growth factor type 1 deficiency

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ORPHA:73272OMIM:608747E34.3
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1Active trials1Specialists8Treatment centers

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Overview

Growth delay due to insulin-like growth factor type 1 deficiency, also known as IGF-1 deficiency or primary IGF-1 deficiency (PIGFD), is a rare condition where the body does not produce enough of a hormone called insulin-like growth factor 1 (IGF-1). IGF-1 is made mainly in the liver and plays a key role in helping children grow normally. Without enough IGF-1, the body cannot respond properly to growth hormone signals, even when growth hormone levels are normal or high. This is different from classic growth hormone deficiency, where the problem starts with too little growth hormone. The main effect of this condition is significantly short stature — children grow much more slowly than their peers and end up much shorter than expected for their family. Other features can include a small head size, delayed bone development, and sometimes mild intellectual or developmental differences. Some children are also born smaller than expected for their gestational age and do not catch up in growth after birth. Treatment with recombinant human IGF-1 (mecasermin, brand name Increlex) is FDA-approved and can help improve growth rates in children with this condition. Early diagnosis and treatment are important to help children reach a better final adult height. Regular monitoring by a pediatric endocrinologist is essential throughout childhood and adolescence.

Also known as:

Key symptoms:

Very short stature compared to peers and family membersSlow growth rate throughout childhoodSmall head size (microcephaly)Born small for gestational age with failure to catch up in growthDelayed bone age (bones develop more slowly than expected)Low birth weight and lengthMild intellectual or learning difficulties in some casesDelayed puberty in some individualsThin or underdeveloped musclesSmall jaw or facial features in some cases

Clinical phenotype terms (42)— hover any for plain English
Prelingual sensorineural hearing impairmentHP:0000399Congenital sensorineural hearing impairmentHP:0008527Bilateral sensorineural hearing impairmentHP:0008619Severe intrauterine growth retardationHP:0008846Severe postnatal growth retardationHP:0008850
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

3 events
Jun 2026The AgRP and GH/IGF-1 Axis in Children

Columbia University

TrialNOT YET RECRUITING
Mar 2026Evaluation of (Cardio)Metabolic and Auxological Outcomes of GHD Patients Under rhGH or LAGH With Stratification According to IGF-1 Levels

Cliniques universitaires Saint-Luc- Université Catholique de Louvain — NA

TrialNOT YET RECRUITING
Dec 2008Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).

Esteve Pharmaceuticals, S.A.

TrialRECRUITING

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 type 1 deficiency.

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
Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).
Actively Recruiting
PI: Global Program Clinical Lead (Esteve) · Sites: Orange, California; Miami, Florida +56 more · Age: 218 yrs

Specialists

1 foundView all specialists →
MR
Manuel Jesus Jimenez Roldan
TAMPA, FL
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

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Latest news about Growth delay due to insulin-like growth factor type 1 deficiency

Disease timeline:

New recruiting trial: Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).

A new clinical trial is recruiting patients for Growth delay due to insulin-like growth factor type 1 deficiency

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

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.How low is my child's IGF-1 level, and how does that compare to normal ranges for their age?,Is my child a candidate for mecasermin (Increlex) treatment, and how soon should we start?,What final adult height can we realistically expect with and without treatment?,What are the signs of low blood sugar I should watch for after giving the injection?,Should other family members be tested for this condition?,Are there any clinical trials or newer treatments we should consider?,What support services — such as developmental therapy or school accommodations — would you recommend?

Common questions about Growth delay due to insulin-like growth factor type 1 deficiency

What is Growth delay due to insulin-like growth factor type 1 deficiency?

Growth delay due to insulin-like growth factor type 1 deficiency, also known as IGF-1 deficiency or primary IGF-1 deficiency (PIGFD), is a rare condition where the body does not produce enough of a hormone called insulin-like growth factor 1 (IGF-1). IGF-1 is made mainly in the liver and plays a key role in helping children grow normally. Without enough IGF-1, the body cannot respond properly to growth hormone signals, even when growth hormone levels are normal or high. This is different from classic growth hormone deficiency, where the problem starts with too little growth hormone. The main

How is Growth delay due to insulin-like growth factor type 1 deficiency inherited?

Growth delay due to insulin-like growth factor type 1 deficiency follows a autosomal recessive 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 type 1 deficiency typically begin?

Typical onset of Growth delay due to insulin-like growth factor type 1 deficiency is childhood. Age of onset can vary across affected individuals.

Are there clinical trials for Growth delay due to insulin-like growth factor type 1 deficiency?

Yes — 1 recruiting clinical trial is currently listed for Growth delay due to insulin-like growth factor type 1 deficiency 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 type 1 deficiency?

1 specialists and care centers treating Growth delay due to insulin-like growth factor type 1 deficiency are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.