Inherited thrombocytopenia
MONDO:0100241An instance of thrombocytopenia that is inherited.
Also known as: hereditary thrombocytopenia
30 clinical trials for this condition and its sub-types, 2 tagged with Inherited thrombocytopenia itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Inherited thrombocytopenia
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Thrombocytopenia 1 9 trials
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Syndromic constitutional thrombocytopenia 0 trials · 8 incl. sub-types
12 sub-types
- GNE myopathy 3 trials
- Platelet storage pool deficiency 3 trials
- Macrothrombocytopenia and granulocyte inclusions with or without nephritis or sensorineural hearing loss 1 trial
- Thrombocytopenia-absent radius syndrome 1 trial
- ACTB-associated syndromic thrombocytopenia 0 trials
- DIAPH1-related sensorineural hearing loss-thrombocytopenia syndrome 0 trials
- Jacobsen syndrome 0 trials
- Stormorken syndrome 0 trials
- Macrothrombocytopenia-lymphedema-developmental delay-facial dysmorphism-camptodactyly syndrome 0 trials
- Marcothrombocytopenia with mitral valve insufficiency 0 trials
- Radio-ulnar synostosis-amegakaryocytic thrombocytopenia syndrome 0 trials Sub-types →
- Thrombocytopenia 6 0 trials
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Congenital amegakaryocytic thrombocytopenia 4 trials · 6 incl. sub-types
1 sub-type
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Alpha granule disease 0 trials
5 sub-types
- Medich giant platelet syndrome 0 trials
- Paris-Trousseau thrombocytopenia 0 trials
- Quebec platelet disorder 0 trials
- Gray platelet syndrome 0 trials
- White platelet syndrome 0 trials
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3 sub-types
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Isolated delta-storage pool disease 0 trials
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Macrothrombocytopenia, isolated 0 trials
2 sub-types
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Thrombocytopenia 10 0 trials
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Thrombocytopenia 13, syndromic 0 trials
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Thrombocytopenia 2 0 trials
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Thrombocytopenia 3 0 trials
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Thrombocytopenia 4 0 trials
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Thrombocytopenia 5 0 trials
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Thrombocytopenia 7 0 trials
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Thrombocytopenia 9 0 trials
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1 sub-type
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Thrombocytopenia, cyclic 0 trials
Most studied deeper sub-types
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New drug combo aims to make bone marrow transplants safer for kids
Disease control OngoingThis pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…
Early phase 1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:39 UTC
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New enzyme replacement therapy aims to stop dangerous blood clots in rare disease
Disease control OngoingThis study tests a medicine called TAK-755 in people with a rare inherited blood disorder called congenital thrombotic thrombocytopenic purpura (cTTP). The condition causes dangerous blood clots and bleeding. TAK-755 replaces a missing enzyme to help prevent or treat these episod…
Phase 3 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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Can a puff of cannabis stop a migraine? small study investigates
Symptom relief PausedThis pilot study tested three different strengths of inhaled cannabis (2.5%, 5%, and 10% THC) against a placebo to see if they could quickly relieve migraine headaches. Twenty adults with migraine took part in a crossover design, meaning each person tried all options. The main go…
Phase 2 • Sponsor: University of California, San Diego • Aim: Symptom relief
Last updated Jun 27, 2026 11:01 UTC
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Can less potent weed make you sharper? new study investigates
Knowledge-focused PausedThis study looks at whether young adults (ages 21-25) who use high-potency cannabis concentrates can improve their memory and thinking by switching to less potent products. Researchers will test participants' cognitive performance and collect blood and urine samples. The goal is …
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:06 UTC