Disorder of melanin metabolism

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ORPHA:352728
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5Active trials1Specialists8Treatment centers

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

Disorder of melanin metabolism is a broad term that covers a group of rare conditions where the body has trouble making, distributing, or breaking down melanin. Melanin is the pigment that gives color to your skin, hair, and eyes. It also plays a role in protecting your skin from the sun and in the normal function of certain parts of the brain and eyes. When melanin metabolism does not work properly, it can lead to areas of skin that are too light (hypopigmentation) or too dark (hyperpigmentation), or to a complete lack of pigment throughout the body. Some forms of this disorder are present from birth, while others may develop later in life. The symptoms and severity vary widely depending on the specific type of melanin metabolism problem. Some people may only have cosmetic changes in skin or hair color, while others may experience vision problems, increased sensitivity to sunlight, or a higher risk of skin damage and skin cancer. In some cases, the disorder can be part of a larger syndrome that affects other organs as well. Treatment depends on the specific underlying cause. There is currently no single cure that applies to all forms of this disorder. Management typically focuses on protecting the skin from sun damage, monitoring for skin cancer, addressing vision problems, and treating any associated conditions. Genetic counseling is often recommended for affected families to understand the inheritance pattern and risks for future children.

Key symptoms:

Unusually light or white skinWhite or very light hairLight-colored eyesPatches of skin with too much or too little colorSensitivity to sunlight and easy sunburningVision problems such as poor visual acuityInvoluntary eye movements (nystagmus)Increased risk of skin cancerFreckling or uneven skin pigmentationDifficulty seeing in bright light (photophobia)Darkened patches of skin in unusual areasDelayed or abnormal pigment development in infancy

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

7 events
Jan 2026Clinical Study to Assess Safety, Effectiveness and In-Use Tolerability of Saffron Extract Capsules for Improving Skin Health and Reducing Signs of Aging on Healthy Adult Male and Female Subjects.

NovoBliss Research Pvt Ltd — NA

TrialNOT YET RECRUITING
Nov 2025Clinical Effectiveness Of Polishing Brush Compared To Ceramic Soft Tissue Trimming Bur In Gingival Depigmentation

Cairo University — NA

TrialRECRUITING
Sep 2025Efficacy and Safety of a Combined Serum Containing Melasyl™, 10% Niacinamide, Hyaluronic Acid, and HEPES

Dr.dr.Irma Bernadette, SpKK (K) — NA

TrialNOT YET RECRUITING
Jun 2025CO2 Fractional Laser With Tranexamic Acid As An Effective Tool For Post Burn Hyperpigmentation.

King Edward Medical University — NA

TrialENROLLING BY INVITATION
Jul 2024National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases

National Eye Institute (NEI) — NA

TrialRECRUITING
Mar 2024Efficacy and Tolerability of the Tested Formula After 3 Months in Treatment of Facial Hyperpigmentation of 3 Origins

Cosmetique Active International — NA

TrialRECRUITING
Feb 2024Peripheral Serotonin and Albinism

Assistance Publique - Hôpitaux de Paris

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Disorder of melanin metabolism.

5 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

5 recruitingView all trials with filters →
N/A4 trials
Efficacy and Tolerability of the Tested Formula After 3 Months in Treatment of Facial Hyperpigmentation of 3 Origins
N/A
Actively Recruiting
PI: Sergio Schalka (Medcin Instituto da Pele Ltda) · Sites: Osasco · Age: 1899 yrs
National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases
N/A
Actively Recruiting
PI: Brian P Brooks, M.D. (National Eye Institute (NEI)) · Sites: Bethesda, Maryland; Bethesda, Maryland · Age: 1120 yrs
Clinical Effectiveness Of Polishing Brush Compared To Ceramic Soft Tissue Trimming Bur In Gingival Depigmentation
N/A
Actively Recruiting
PI: Mona Darhous, Professor (Cairo Univeristy) · Sites: Cairo · Age: 1840 yrs
CO2 Fractional Laser With Tranexamic Acid As An Effective Tool For Post Burn Hyperpigmentation.
N/A
Enrolling by Invitation
· Sites: Lahore, Punjab Province · Age: 1860 yrs
Other1 trial
Peripheral Serotonin and Albinism
Actively Recruiting
PI: Smail HADJ-RABIA, MD, PhD (Assistance Publique - Hôpitaux de Paris) · Sites: Paris · Age: 217 yrs

Specialists

1 foundView all specialists →
BM
Brian P Brooks, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 8 active trials

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 Disorder of melanin metabolism.

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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.What specific type of melanin metabolism disorder do I or my child have?,Is genetic testing recommended, and what genes should be tested?,How often should skin cancer screening and eye exams be done?,Are there any associated health problems I should watch for beyond skin and eyes?,What is the inheritance pattern, and should other family members be tested?,What accommodations might be helpful at school or work for vision problems?,Are there any clinical trials or new treatments being studied for this condition?

Common questions about Disorder of melanin metabolism

What is Disorder of melanin metabolism?

Disorder of melanin metabolism is a broad term that covers a group of rare conditions where the body has trouble making, distributing, or breaking down melanin. Melanin is the pigment that gives color to your skin, hair, and eyes. It also plays a role in protecting your skin from the sun and in the normal function of certain parts of the brain and eyes. When melanin metabolism does not work properly, it can lead to areas of skin that are too light (hypopigmentation) or too dark (hyperpigmentation), or to a complete lack of pigment throughout the body. Some forms of this disorder are present fr

Are there clinical trials for Disorder of melanin metabolism?

Yes — 5 recruiting clinical trials are currently listed for Disorder of melanin metabolism on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Disorder of melanin metabolism?

1 specialists and care centers treating Disorder of melanin metabolism are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.