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NCT ID: NCT04623398 Active, not recruiting - Clinical trials for Autism Spectrum Disorder

Effect of Lithium in Patients With Autism Spectrum Disorder and Phelan-McDermid Syndrome (SHANK3 Haploinsufficiency)

Lisphem
Start date: February 21, 2022
Phase: Phase 3
Study type: Interventional

There is currently no treatment for the body symptoms of Autism Spectrum Disorders (ASD). However, basic research suggests that some forms of ASD may be alleviated, even in the adult stage. The genes involved in ASDs particularly impact synaptic homeostasis. Specific clinical trials in patients with synaptic mutations need to be carried out. In this spirit, patients with deleterious mutations in SHANK3 represent a paradigm. The induced pluripotent stem cells (iPSc) carrying SHANK3 mutations and derived in neurons, can be used for high-throughput screening of pharmacological substances and allow the identification of compounds that can restore the expression level of SHANK3. The objective of this proposed project is to test one of the compounds identified by research on these iPSc as a novel treatment for social communication deficit in patients with deleterious mutations in SHANK3. Its effect on the symptoms of the social deficit could represent a new perspective for other forms of idiopathic autism.

NCT ID: NCT04622553 Active, not recruiting - Myotonic Disorders Clinical Trials

Open-label Extension Study in Paediatric Patients Who Have Completed the MEX-NM-301 Study.

Start date: November 5, 2021
Phase: N/A
Study type: Interventional

Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed the MEX-NM-301 study.

NCT ID: NCT04622319 Active, not recruiting - Clinical trials for Residual Invasive Breast Cancer

A Study of Trastuzumab Deruxtecan (T-DXd) Versus Trastuzumab Emtansine (T-DM1) in High-risk HER2-positive Participants With Residual Invasive Breast Cancer Following Neoadjuvant Therapy (DESTINY-Breast05)

Start date: December 4, 2020
Phase: Phase 3
Study type: Interventional

Patients with HER2-positive primary breast cancer (BC) who do not achieve complete response after appropriate neoadjuvant therapy are at higher risk of disease recurrence. More effective treatment options are needed for this patient population. This study will examine the efficacy and safety of trastuzumab deruxtecan (T-DXd) compared with trastuzumab emtansine (T-DM1) in high-risk patients with residual invasive breast cancer following neoadjuvant therapy.

NCT ID: NCT04619797 Active, not recruiting - Clinical trials for Non-small Cell Lung Cancer (NSCLC)

A Study of Tiragolumab in Combination With Atezolizumab Plus Pemetrexed and Carboplatin/Cisplatin Versus Pembrolizumab Plus Pemetrexed and Carboplatin/Cisplatin in Participants With Previously Untreated Advanced Non-Squamous Non-Small Cell Lung Cancer

SKYSCRAPER-06
Start date: December 14, 2020
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of tiragolumab in combination with atezolizumab plus pemetrexed and carboplatin/cisplatin (Arm A) compared with placebo in combination with pembrolizumab plus pemetrexed and carboplatin/cisplatin (Arm B) in participants with previously untreated, locally advanced unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC). Eligible participants will be randomized in a 1:1 ratio to receive one of the following treatment regimens during the induction phase: - Arm A: Tiragolumab plus atezolizumab plus pemetrexed and carboplatin or cisplatin - Arm B: Placebo plus pembrolizumab plus pemetrexed and carboplatin or cisplatin Following the induction phase, participants will continue maintenance therapy with either tiragolumab in combination with atezolizumab and pemetrexed (Arm A) or placebo in combination with pembrolizumab and pemetrexed (Arm B).

NCT ID: NCT04619420 Active, not recruiting - Alzheimer Disease Clinical Trials

A Study of JNJ-63733657 in Participants With Early Alzheimer's Disease

Autonomy
Start date: January 6, 2021
Phase: Phase 2
Study type: Interventional

The primary purpose of this study is to evaluate the effect of JNJ-63733657 versus placebo on clinical decline as measured by the Integrated Alzheimer's Disease Rating Scale (iADRS), a composite of cognition and function.

NCT ID: NCT04619004 Active, not recruiting - Clinical trials for Non-Small Cell Lung Cancer Metastatic

HERTHENA-Lung01: Patritumab Deruxtecan in Subjects With Metastatic or Locally Advanced EGFR-mutated Non-Small Cell Lung Cancer

Start date: February 2, 2021
Phase: Phase 2
Study type: Interventional

This study is designed to evaluate the antitumor activity of patritumab deruxtecan in participants with metastatic or locally advanced NSCLC with an activating EGFR mutation (exon 19 deletion or L858R) who have received and progressed on or after at least 1 EGFR TKI and 1 platinum-based chemotherapy-containing regimen.

NCT ID: NCT04616807 Active, not recruiting - Myotonic Dystrophy Clinical Trials

An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders

Start date: December 17, 2020
Phase:
Study type: Observational

This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM.

NCT ID: NCT04612413 Active, not recruiting - Sepsis Clinical Trials

A Phase 2 Study Evaluating Efficacy, Safety and Tolerability of Different Doses and Regimens of Allocetra-OTS for the Treatment of Organ Failure in Adult Sepsis Patients

Start date: November 30, 2020
Phase: Phase 2
Study type: Interventional

A Phase 2, Multi-Center, Randomized, Placebo-Controlled, Dose-Finding Study Evaluating Efficacy, Safety and Tolerability of Different Doses and Regimens of Allocetra-OTS for the Treatment of Organ Failure in Adult Sepsis Patients

NCT ID: NCT04610736 Active, not recruiting - Pediatric Cancer Clinical Trials

TEMOkids Study : A Phase I Pediatric Study for KIMOZO, Oral Suspension of Temozolomide

Start date: March 16, 2021
Phase: Phase 1
Study type: Interventional

Non-randomized, international, multi-centre, open-label, single arm study to determine the pharmacokinetic (PK) parameters of a single dose of an oral suspension of temozolomide (KIMOZO) in the pediatric population aged 1 year and over.

NCT ID: NCT04609826 Active, not recruiting - Clinical trials for Acute Myeloid Leukemia

A Study of JNJ-74856665 in Participants With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

DHODH
Start date: November 26, 2020
Phase: Phase 1
Study type: Interventional

The purpose of this study is to determine the safety, tolerability, maximum tolerated doses (MTDs) and recommended Phase 2 doses (RP2Ds) of JNJ-74856665 as monotherapy and/or in combinations.