Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

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ORPHA:88924OMIM:600273Q61.2
Who is this for?
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3Active trials13Specialists8Treatment centers

Where are you in your journey?

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

Also known as:

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

6 events
Sep 2026Assessing Changes in Multi-parametric MRI in Patients With Acute Demyelinating Lesions Taking Clemastine Fumarate as a Myelin Repair Therapy

University of California, San Francisco — PHASE1, PHASE2

TrialNOT YET RECRUITING
Mar 2026Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)

Sovargen — PHASE1, PHASE2

TrialNOT YET RECRUITING
Mar 2026Sleep and Circadian Interventions for College Students at High Risk of Suicide

University of Pittsburgh — NA

TrialNOT YET RECRUITING
Dec 2025Sapu003 in Advanced mTOR-sensitive Solid Tumors

SAPU NANO (US) LLC — PHASE1

TrialRECRUITING
Jul 2024Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

GRIN Therapeutics, Inc. — PHASE1, PHASE2

TrialRECRUITING
May 2015Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)

Boston Children's Hospital

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis.

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

View clinical trials →

Clinical Trials

3 recruitingView all trials with filters →
Phase 11 trial
Sapu003 in Advanced mTOR-sensitive Solid Tumors
Phase 1
Actively Recruiting
· Sites: Adelaide · Age: 1899 yrs
Other1 trial
Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
Active
PI: Darcy Krueger, MD, PhD (Children's Hospital Medical Center, Cincinnati) · Sites: Birmingham, Alabama; Los Angeles, California +4 more

Specialists

13 foundView all specialists →
DP
Darcy Krueger, MD, PhD
CINCINNATI, OH
Specialist
PI on 2 active trials
MP
Mustafa Sahin, MD, PhD
BOSTON, MA
Specialist
PI on 5 active trials
DF
David N Franz
Specialist
PI on 1 active trial
JS
Julian Sampson
Specialist
PI on 1 active trial
NM
Nicole McDonald
Specialist
PI on 1 active trial
SP
Shoufeng Li, Ph.D
Specialist
PI on 1 active trial
SA
Sam Amin
Specialist
PI on 1 active trial
YM
Yannick LE MEUR
Specialist
PI on 1 active trial
OR
Owen M Rennert
Specialist
PI on 1 active trial
TG
Tanjala Gipson
MEMPHIS, TN
Specialist
PI on 1 active trial
IS
Ioana Stanescu
GLASTONBURY, CT
Specialist
PI on 1 active trial
CD
Claire RIGOTHIER, Dr
Specialist
PI on 1 active trial
MW
M.C.Y. de Wit
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 Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

Disease timeline:

New recruiting trial: Assessing Changes in Multi-parametric MRI in MS Patients Taking Clemastine Fumarate as a Myelin Repair Therapy

A new clinical trial is recruiting patients for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

New recruiting trial: Study of Skin Tumors in Tuberous Sclerosis

A new clinical trial is recruiting patients for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

New recruiting trial: Sapu003 in Advanced mTOR-sensitive Solid Tumors

A new clinical trial is recruiting patients for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

New recruiting trial: Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

A new clinical trial is recruiting patients for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

New recruiting trial: Regulating Together in Tuberous Sclerosis Complex

A new clinical trial is recruiting patients for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

New recruiting trial: Observational, Retrospective, Multicenter, Nonprofit Study on the Prevalence of Renal Involvement in Pediatric Patients With Tuberous Sclerosis

A new clinical trial is recruiting patients for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

New recruiting trial: Motor-cognitive Performance in People With Multiple Sclerosis

A new clinical trial is recruiting patients for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

New recruiting trial: Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling

A new clinical trial is recruiting patients for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

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.

Common questions about Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis

Are there clinical trials for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis?

Yes — 3 recruiting clinical trials are currently listed for Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis?

13 specialists and care centers treating Autosomal dominant polycystic kidney disease type 1 with tuberous sclerosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.