Hereditary endocrine growth disease
MONDO:0015514Also known as: genetic endocrine growth disease, growth disorder
196 clinical trials for this condition and its sub-types, 26 tagged with Hereditary endocrine growth disease itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Hereditary endocrine growth disease
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Non-acquired pituitary hormone deficiency 0 trials · 85 incl. sub-types
5 sub-types
- Congenital hypogonadotropic hypogonadism 0 trials · 83 incl. sub-types Sub-types →
- Non-acquired combined pituitary hormone deficiency 1 trial · 3 incl. sub-types Sub-types →
- Isolated thyroid-stimulating hormone deficiency 1 trial
- Pituitary stalk interruption syndrome 1 trial
- Short stature due to GHSR deficiency 0 trials
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Inherited primary ovarian failure 2 trials · 48 incl. sub-types
41 sub-types
- Turner syndrome 29 trials Sub-types →
- Ataxia telangiectasia 11 trials Sub-types →
- Classic galactosemia 3 trials
- Trisomy X 3 trials
- 46 XX gonadal dysgenesis 1 trial Sub-types →
- Congenital lipoid adrenal hyperplasia due to STAR deficency 1 trial Sub-types →
- Premature ovarian failure 1 1 trial Sub-types →
- 46,XX ovarian dysgenesis-short stature syndrome 0 trials
- Perrault syndrome 0 trials Sub-types →
- Satoyoshi syndrome 0 trials
- X small rings 0 trials
- Aromatase deficiency 0 trials
- Blepharophimosis, ptosis, and epicanthus inversus syndrome 0 trials Sub-types →
- Osteosclerosis-ichthyosis-premature ovarian failure syndrome 0 trials
- Premature ovarian failure 10 0 trials
- Premature ovarian failure 11 0 trials
- Premature ovarian failure 12 0 trials
- Premature ovarian failure 13 0 trials
- Premature ovarian failure 14 0 trials
- Premature ovarian failure 15 0 trials
- Premature ovarian failure 16 0 trials
- Premature ovarian failure 17 0 trials
- Premature ovarian failure 18 0 trials
- Premature ovarian failure 19 0 trials
- Premature ovarian failure 20 0 trials
- Premature ovarian failure 21 0 trials
- Premature ovarian failure 22 0 trials
- Premature ovarian failure 23 0 trials
- Premature ovarian failure 24 0 trials
- Premature ovarian failure 25 0 trials
- Premature ovarian failure 26 0 trials
- Premature ovarian failure 2A 0 trials
- Premature ovarian failure 2B 0 trials
- Premature ovarian failure 3 0 trials
- Premature ovarian failure 4 0 trials
- Premature ovarian failure 5 0 trials
- Premature ovarian failure 6 0 trials
- Premature ovarian failure 7 0 trials
- Premature ovarian failure 8 0 trials
- Premature ovarian failure 9 0 trials
- Tetrasomy X 0 trials
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Congenital adrenal hyperplasia 36 trials · 38 incl. sub-types
8 sub-types
- Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency 20 trials Sub-types →
- Classic congenital adrenal hyperplasia 5 trials
- Congenital adrenal hyperplasia due to 11-beta-hydroxylase deficiency 1 trial
- Congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiency 1 trial
- Congenital adrenal hyperplasia due to 3-beta-hydroxysteroid dehydrogenase deficiency 1 trial
- Congenital adrenal hyperplasia due to cytochrome P450 oxidoreductase deficiency 1 trial
- Congenital lipoid adrenal hyperplasia due to STAR deficency 1 trial Sub-types →
- Non-classic congenital adrenal hyperplasia 0 trials
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Growth hormone insensitivity syndrome 0 trials · 6 incl. sub-types
6 sub-types
- Growth delay due to insulin-like growth factor I resistance 4 trials
- Growth delay due to insulin-like growth factor type 1 deficiency 1 trial
- Growth hormone insensitivity syndrome with immune dysregulation 0 trials · 1 incl. sub-types Sub-types →
- Laron syndrome 0 trials
- Short stature due to partial GHR deficiency 0 trials
- Short stature due to primary acid-labile subunit deficiency 0 trials
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Permanent congenital hypothyroidism 1 trial · 3 incl. sub-types
2 sub-types
- Central congenital hypothyroidism 1 trial · 2 incl. sub-types Sub-types →
- Peripheral hypothyroidism 0 trials Sub-types →
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Zerres Rietschel Majewski syndrome 0 trials
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Microdontia hypodontia short stature 0 trials
Most studied deeper sub-types
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New nasal spray aims to curb relentless hunger in rare genetic disorder
Disease control Stopped earlyThis study tests the long-term safety of a nasal spray called carbetocin for people with Prader-Willi syndrome who experience severe, constant hunger (hyperphagia). About 160 participants who completed a previous study will receive the spray three times daily. The goal is to see …
Phase 3 • Sponsor: ACADIA Pharmaceuticals Inc. • Aim: Disease control
Last updated Aug 19, 2026 00:00 UTC
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New hope for rare hunger disorder: drug shows promise in Long-Term trial
Disease control Stopped earlyThis study tests whether ARD-101 can safely reduce extreme hunger and food-related behaviors in people with Prader-Willi syndrome over 12 months. About 90 participants who completed a prior study will take the drug daily and visit the clinic regularly. The goal is to improve qual…
Phase 3 • Sponsor: Aardvark Therapeutics, Inc. • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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Can a daily injection reshape bodies of HIV patients?
Disease control Stopped earlyThis study looked at whether tesamorelin (Egrifta), a daily injection that boosts growth hormone, can improve body composition in people with HIV who have excess belly fat (lipodystrophy). Six participants received the drug for up to 12 months, with researchers measuring liver fa…
Phase 4 • Sponsor: Columbia University • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC
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Continued EryDex treatment studied in rare neurological disorder
Disease control Stopped earlyThis study offered continued treatment with EryDex to 101 people with ataxia telangiectasia (A-T) who had finished a previous trial. The main goal was to monitor safety, including side effects and serious events. The study was terminated early, and it did not aim to cure the dise…
Phase 3 • Sponsor: Quince Therapeutics S.p.A. • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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Experimental drug shows promise for rare genetic disorder
Disease control Stopped earlyThis Phase II trial tested a drug called N-Acetyl-L-Leucine (IB1001) in 17 people with Ataxia-Telangiectasia, a rare genetic disease that affects movement and immunity. The study aimed to see if the drug could improve symptoms and slow the disease over time. The trial was termina…
Phase 2 • Sponsor: IntraBio Inc • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New hope for kids with rare hormone disorder: drug may cut steroid doses
Disease control Stopped earlyThis study tested an experimental drug called tildacerfont in 67 children aged 2 to 17 with congenital adrenal hyperplasia (CAH), a genetic condition that disrupts hormone production. The goal was to see if adding this once-daily pill to standard steroid treatment could improve d…
Phase 2 • Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC
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Prostate cancer drug trialed to reduce steroids in kids with rare hormone disorder
Disease control Stopped earlyThis early-phase study tested a drug called abiraterone acetate (normally used for prostate cancer) in 4 children with congenital adrenal hyperplasia (CAH). The goal was to see if it could lower high male hormone levels and reduce the need for strong steroid medications that can …
Phase 1 • Sponsor: University of Texas Southwestern Medical Center • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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New drug aims to cut steroid use in rare hormone disorder
Disease control Stopped earlyThis study tested a daily tablet called Tildacerfont in 100 adults with classic congenital adrenal hyperplasia, a condition where the body can't make certain hormones properly. The goal was to see if the drug could safely reduce the high doses of steroids patients need to take. T…
Phase 2 • Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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New oral test could replace painful insulin injections for hormone diagnosis
Diagnosis Stopped earlyThis study tested a new oral drink (GS3-007a) to diagnose adult growth hormone deficiency (AGHD). It compared the drink to the standard insulin tolerance test in 120 adults suspected of having AGHD and healthy volunteers. The goal was to see if the oral test works as well as the …
Phase 2 • Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd. • Aim: Diagnosis
Last updated Jun 27, 2026 12:37 UTC
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Hope for rare hunger disorder: new drug enters final testing phase
Symptom relief Stopped earlyThis phase 3 trial tests whether ARD-101 can reduce the intense, constant hunger (hyperphagia) seen in Prader-Willi syndrome. About 90 participants will take either ARD-101 or a placebo daily for 12 weeks. Caregivers will track changes in hunger-related behaviors using a standard…
Phase 3 • Sponsor: Aardvark Therapeutics, Inc. • Aim: Symptom relief
Last updated Jul 02, 2026 00:00 UTC
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CBDV study for Prader-Willi syndrome halted early
Symptom relief Stopped earlyThis study tested whether a cannabis-derived compound called CBDV could safely reduce irritability in children and young adults with Prader-Willi syndrome. Only 6 people enrolled before the study was stopped early. The goal was to see if CBDV helped with mood and behavior problem…
Phase 2 • Sponsor: Eric Hollander • Aim: Symptom relief
Last updated Jun 27, 2026 12:30 UTC
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Ultrasound could uncover hidden vascular risks in turner syndrome
Knowledge-focused Stopped earlyThis study is looking at whether yearly ultrasound scans can detect blood vessel abnormalities throughout the body in women with Turner syndrome, not just in the heart area. Turner syndrome is a genetic condition that increases the risk of serious vascular problems, which can sho…
Sponsor: University Hospital, Strasbourg, France • Aim: Knowledge-focused
Last updated Aug 23, 2026 00:00 UTC
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Brain and eye clues to emotion recognition in autism and psychosis
Knowledge-focused Stopped earlyThis study aimed to understand why people with autism or schizophrenia sometimes struggle to recognize emotions on faces. Researchers used brain wave recordings (EEG) and eye-tracking to see how participants processed facial expressions. The study included people with autism, sch…
Sponsor: Hôpital le Vinatier • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:07 UTC